A Randomized Phase II Bayesian Pick-the-Winner Clinical Trial Investigating the Efficacy of Fibrosis-Modulating Agents (Paricalcitol and Pirfenidone) in Improving Surgical Resectability in Patients With Peritoneal Carcinomatosis Ineligible for Definitive Cytoreduction (Randy 1A)

The goal of this clinical trial is to learn if paricalcitol or pirfenidone can help make tumors removable by surgery in adults with peritoneal cancer who are currently unable to have their tumor removed.
The study aims to answer are whether paricalcitol or pirfenidone improve tumor response and tissue healing after 12 weeks of treatment, and which of these two drugs is more effective at helping participants become eligible for surgery. Researchers will compare paricalcitol to pirfenidone to see which drug works better and has a manageable safety profile.
Participants will take either paricalcitol or pirfenidone by mouth every day for 12 weeks while continuing their standard chemotherapy, visit the clinic regularly for checkups and lab tests, keep a daily diary to track their study medication, and undergo a surgical re-evaluation after treatment to see if their tumor can be removed.

Start Date01 Sep 2026

Sponsor / Collaborator

Allegheny-Singer Research Institute

ChiCTR2500113259

/ SuspendedPhase 2IIT

A Single-Center, Single-Arm, Open-Label Phase II Clinical Study of Recombination with Paricalcitol After First-Line PD-1 Inhibitor Regimen Failure in Unresectable Hepatocellular Carcinoma Patients with Serum Vitamin D Deficiency

Start Date01 Dec 2025

Sponsor / Collaborator-

NCT06209268

/ Not yet recruitingNot ApplicableIIT

The Impact of Selective Vitamin D Receptor Activation on Clinical Outcomes in Septic Patients -a Randomised Placebo Controlled Trial

Sufficient serum levels of vitamin D are important for immune system regulation with protective effect against severe infection and overactivated inflammatory response in sepsis. It is also not clear what level of vitamin D in the blood would be the trigger for vitamin D administration. A more selective approach to VDR activation than cholecalciferol could have a more significant role in the clinical outcomes of patients with sepsis. A study demonstrated that low baseline serum level of vitamin D receptor (VDR) was associated with a high incidence of 28-day mortality and negatively correlated with lactate, C-reactive protein, APACHE II SOFA scores, and disease severity among patients with sepsis in an ICU setting.
The role of selective vitamin D receptor activation agents (paricalcitol or maxacalcitol) was not studied in septic patients, despite its anti-inflammatory and immunomodulatory properties. Vitamin D analogs have different effects on nuclear VDRs than does calcitriol, through different response elements in various target genes, so it is possible that their effect on a patient with sepsis will be more effective than cholecalciferol. As distribution of VDRs is ubiquitous in many organs and tissues, selective VDR activation with paricalcitol may have beneficial effects in preserving organs functionality and modulating the immune response in sepsis.
Hypotheses
1. The immunoregulatory, anti-inflammatory, and anti-oxidative properties of selective vitamin D receptor activator paricalcitol would result in improvement of inflammatory, endothelial function, and antioxidative parameters and clinical outcomes in groups of septic patient admitted to ICU.
2. The baseline septic patient serum 25(OH) D3 levels at admission time in ICU have influence on clinical outcomes as well as on inflammatory, endothelial function, and antioxidative parameters.
3. The inflammatory, endothelial function, and antioxidative parameters measured at ICU admission time have significant impact on clinical outcomes in septic patients.
The aim
The main objective of study is to test hypothesis that that selective activator of vitamin D receptors paricalcitol will improve outcomes of septic patient admitted in ICU. The study aims to investigate the effects of paricalcitol on clinical outcomes, inflammatory markers, organ dysfunction, endothelial function, vascular morphology, coagulation markers, and haemodynamic parameters.
The additional objectives of the study are to test hypothesis that septic patient serum 25(OH)vitamin D3 have impact on inflammatory, endothelial function, and antioxidative parameters including protein carbonylation; and to test hypothesis that these markers and clinical outcomes are interconnected with significant impact on clinical outcomes.

Start Date01 May 2025

Sponsor / Collaborator-

100 Clinical Results associated with Paricalcitol

100 Translational Medicine associated with Paricalcitol

100 Patents (Medical) associated with Paricalcitol

684

Literatures (Medical) associated with Paricalcitol

01 Oct 2026·EXPERIMENTAL GERONTOLOGY

Serum 25(OH) vitamin D and SIRT1- VDR- miR-124 expression: A controlled interventional study in a vitamin D-deficient male rat model

Article

Author: Dhar, Poulami ; Kalladka, Shwetha Shetty ; Moodithaya, Shailaja ; Bhandary, Prajna

BACKGROUND:

Epigenetic aging is a new dimensional advancement in geroscience that continually addresses factors beyond genetic determinants of longevity. It occurs through three conventional pathways, one of which is non-coding RNA production. miR-124 is a newly introduced miRNA that was found in aged skin and several tumorigenic cells and is associated with aging. Additionally, SIRT1, a protein from the NAD⁺-dependent family, is found to have a protective mechanism in the pathways of aging. On the other hand, vitamin D works as an epigenetic factor and often targets pathways related to the aging process. In this study, the aim is to identify the expression of miR-124 in circulating blood to address the epigenetic-related biomarker and link the facets with anti-aging SIRT1 and vitamin D cognate receptor (VDR) to understand vitamin D deficiency interception.

METHODS:

A vitamin D deficiency middle-aged rat model was developed by assigning male Albino Wistar Rats. Paricalcitol was administered IP (intraperitoneal) to the experimental group (32 ng/day) to induce deficiency, along with a calcium-rich rescue diet. The study was conducted for 21 days following blood collection and storage. ELISA and RTqPCR were performed to estimate serum 25(OH)D and gene expression analysis, respectively.

RESULT:

25(OH)D comparison between the group p-value of 0.002. The log2 fold changes of SIRT1, VDR, and miR-124 were compared between the groups using boxplot visualisation. A linear regression model was developed to understand the explorative nature of aging index (R² = 0.561; Adjusted R² = 0.517; p = 0.005; n = 12).

CONCLUSION:

A putative epigenetic relation was found between miR-124 levels and their association with SIRT1. The investigated biomarkers of aging are associated with serum concentration of 25(OH) vitamin D in a middle-aged vitamin D-deficient rat model, which supports the need for mechanistic investigation in future studies.

01 Oct 2026·JACC. Advances

Vitamin D Pathway Activation Reduces Cardiomyocyte DNA Damage and Improves Cardiac Contractility in Preclinical Models

Article

Author: Toyoda, Masashi ; Kubota, Masayuki ; Nishijo, Daigo ; Takeda, Norihiko ; Yamada, Shintaro ; Abe, Ryo ; Umezawa, Akihiro ; Tsukihashi, Ayako ; Yamasaki, Masataka ; Komuro, Jin ; Ikeuchi, Masashi ; Nomura, Seitaro ; Onoue, Kenji ; Takada, Shuji ; Aburatani, Hiroyuki ; Hiruma, Takashi ; Dai, Zhehao ; Katagiri, Mikako ; Katoh, Manami ; Matsuura, Katsuhisa ; Ko, Toshiyuki ; Morita, Hiroyuki ; Komuro, Issei ; Zhang, Yanfei ; Inoue, Shunsuke ; Ito, Masamichi

BACKGROUND:

In heart failure (HF), DNA damage caused by various external stressors contributes to cardiac dysfunction through the activation of DNA damage response pathways. To date, no clinical strategies have been established to restore cardiac function by reducing accumulated DNA damage. We previously found that vitamin D improved contractility in lamin A/C (LMNA) p.Q353R-mutant induced pluripotent stem (iPS) cell-derived cardiomyocytes (iPSCMs), but whether this effect extends to other LMNA variants and in vivo models remained uncertain.

OBJECTIVES:

The objective of the study was to evaluate the association of vitamin D pathway activation with cardiomyocyte phosphorylated histone H2AX (γH2AX) foci and contractile phenotypes in patient-derived iPSCMs and mouse models of HF.

METHODS:

iPS cell lines were generated from dilated cardiomyopathy patients carrying the LMNA p.R225X mutation, and the effects of vitamin D treatment on γH2AX foci and cardiomyocyte contractility were evaluated. In addition, the effects of the vitamin D analog paricalcitol were evaluated in Lmna p.R225X mice and in a pressure overload mouse model of HF.

RESULTS:

Consistent with previous findings, vitamin D treatment reduced γH2AX foci in cardiomyocytes derived from LMNA p.R225X mutant iPS cells through upregulating the expression of DNA repair factors, and improved contractility in these iPSCMs. Furthermore, paricalcitol reduced γH2AX foci and attenuated cardiac dysfunction in both Lmna p.R225X mice and pressure overload HF model mice.

CONCLUSIONS:

Vitamin D pathway activation improved contractile phenotypes across complementary preclinical models and was accompanied by reduced γH2AX foci or related transcriptional changes. These findings support further mechanistic and preclinical investigation.

01 Aug 2026·Cancer research communications

Co-Clinical Evaluation of Synthetic Vitamin D and Chemotherapy in Pancreatic Cancer by Integrated Imaging and Molecular Analyses

Article

Author: Liu, Fang ; Choi, Hoon ; O’Dwyer, Peter J. ; Gee, James C. ; Rosen, Mark ; Cao, Quy ; Englander, Sarah ; Pickup, Stephen ; Duda, Jeffrey ; Karasic, Thomas ; Zhou, Rong ; Clendenin, Cynthia ; Fan, Yong ; Furth, Emma E. ; Shaffer, Sydney ; Gupta, Mamta ; Song, Hee Kwon

Abstract:

Purpose::

A pilot clinical trial (NCT03519308) was conducted to evaluate perioperative chemotherapy [nab-paclitaxel, gemcitabine, and cisplatin (NGC)] plus nivolumab, with or without synthetic vitamin D (paricalcitol) in resectable pancreatic ductal adenocarcinoma (PDAC). Preclinical studies were designed with statistical power to match the experimental regimen and compare NGC only with NGC plus synthetic vitamin D [calcipotriol (Cal)] or losartan (Losa; an antihypertensive medication).

Materials and Methods::

Molecular subtyping was applied to enroll patients with epithelial subtype PDAC. Genetically engineered PDAC models (KrasG12D;Trp53R172H;Pdx1-Cre mice, N = 140) were randomized to NGC only and combined therapies. Multiparametric MRI and molecular profiling were integrated to assess treatment-induced effects on stroma, including capillary perfusion and density, matrix collagen content, fibroblast activation protein (FAP) levels and transcriptome changes.

Results::

The clinical trial had limited enrollment (N = 3) but confirmed the safety and feasibility of weekly paricalcitol infusion in combination with multiagent chemotherapy. NGC chemotherapy had a profound impact on stroma, inducing a remarkable reduction in FAP levels accompanied by a significant increase in matrix collagen content; chemotherapy also activated epithelial–mesenchymal transition (EMT) and significantly reduced blood perfusion and capillary density, resulting in features of chemoresistance. Combining NGC with stromal-targeting agents—Cal or Losa—diminished EMT and partially restored perfusion through distinct mechanisms: Cal activates the vitamin D receptor to reprise a quiescent stroma, whereas Losa suppresses TGFβ to increase lymphocyte infiltration.

Conclusions::

These findings support further investigations of stromal-directed combination strategies to overcome chemotherapy-induced resistance of PDAC. Dynamic contrast-enhanced MRI may provide a valuable platform to monitor stromal adaptations to therapy in vivo.

Significance::

Results from co-clinical trials integrating MRI and molecular profiling provide a translational rationale for investigating stromal-directed combination strategies in PDAC. These approaches may help preserve chemosensitivity, counteract chemotherapy-induced stromal and vascular remodeling, and overcome adaptive resistance mechanisms that limit durable treatment response.

News (Medical) associated with Paricalcitol

14 Oct 2026

·www.globenewswire.com

Three-Year Real-World Study Compares Burosumab Treatment with Oral Phosphate and Active Vitamin D in Adults with XLH

Publication in the Journal of Bone and Mineral Research offers new insight into the long-term XLH treatment experience PRINCETON, N.J., Oct. 14, 2026 (GLOBE NEWSWIRE) -- Kyowa Kirin, Inc., a wholly owned subsidiary of Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin), today announced the publication of three-year real-world findings from an interim analysis of the XLH Disease Monitoring Program (DMP) in the Journal of Bone and Mineral Research. The analysis assessed serum phosphate levels and patient-reported pain, stiffness, and physical function at years 1 and 3 among adults with X-linked hypophosphatemia (XLH) treated with burosumab or oral phosphate and active vitamin D (Pi/D). Read the full study here. “Real-world evidence in rare diseases like XLH is invaluable, helping to deepen our understanding of the patient experience and treatment outcomes beyond clinical trials,” said lead author Steven W. Ing, MD, MSCE, Professor of Clinical Medicine, Division of Endocrinology, Diabetes and Metabolism, The Ohio State University Wexner Medical Center. “These findings add to the broader evidence base for burosumab and can help inform clinical decision-making and the ongoing evolution of care for adults living with XLH.” XLH is a rare, lifelong, progressive genetic disease that causes excessive phosphorus loss, weakening the bones and muscles in children and adults, leading to skeletal deformities.1 The XLH DMP is a prospective, 10-year, observational study of adults and children with XLH in the US, Canada, and Latin America. It is designed to characterize XLH disease presentation and progression, and to prospectively assess changes over time in biochemical, clinical, and patient/caregiver-reported outcomes in a representative population. No randomized studies have directly compared treatment with burosumab, a monoclonal antibody targeting FGF23, with oral phosphate and active vitamin D in adults with XLH. This three-year analysis included 109 adults enrolled in the XLH DMP: 54 who received burosumab and 55 who received Pi/D. “For people living with a rare, lifelong condition such as XLH, it is essential to understand how treatments perform over time,” said Angela Williams, PhD, Vice President, Global Head of Health Economics and Outcomes Research at Kyowa Kirin. “By following patients longitudinally and incorporating patient-reported measures, the XLH Disease Monitoring Program provides additional context for the clinical data supporting burosumab use in adults with XLH.” In addition to the XLH Disease Monitoring Program, Kyowa Kirin is advancing real-world insight generation through the International XLH Registry in Europe and the SUNFLOWER study in Japan and Korea. About CRYSVITA® (burosumab-twza) Injection CRYSVITA is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore phosphorus reabsorption in the kidneys and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium. U.S. CRYSVITA Indication CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Important Safety Information CONTRAINDICATIONS CRYSVITA is contraindicated: In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia. When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism. WARNINGS AND PRECAUTIONS Hypersensitivity Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment. Hyperphosphatemia and Risk of Nephrocalcinosis Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient’s serum phosphorus levels. Hypercalcemia Increases in serum calcium have been reported in patients treated with CRYSVITA. Patients with risk factors such as pre-existing hyperparathyroidism, prolonged immobilization, dehydration, hypervitaminosis D, or renal impairment, are at higher risk of hypercalcemia. Monitor these patients for serum calcium and parathyroid hormone levels before and during CRYSVITA treatment for moderate to severe hypercalcemia. In patients with moderate to severe hypercalcemia, CRYSVITA should not be administered until hypercalcemia is adequately managed. Injection Site Reactions Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment. ADVERSE REACTIONS Pediatric Patients Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%). Adult Patients Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%). Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression. USE IN SPECIFIC POPULATIONS There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544. There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition. PATIENT COUNSELING INFORMATION Advise patients not to use any oral phosphate and/or active vitamin D analog products. Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur. You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544. For important risk and use information, please see the full Prescribing Information for CRYSVITA. About X-linked hypophosphatemia (XLH) X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults. In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health. This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine. When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in adults (osteomalacia).1 In children, XLH typically appears as bowed legs or knock knees. Over time, bone weakening can also lead to bone abnormalities in the legs, delayed growth, and short stature. In adults, XLH may cause osteomalacia, fractures and pseudo-fractures, and hypophosphatemia.1 About the XLH Disease Monitoring Program The XLH DMP is an international, prospective, 10-year, longitudinal, observational study (NCT03651505) of adults and children with XLH in the US, Canada, and Latin America designed to characterize XLH disease presentation and progression, and prospectively assess changes over time in biochemical, clinical, and patient/caregiver-reported outcomes in a representative population. The program steering committee includes clinical experts in XLH, patient advocates from the XLH Network, and representatives from Kyowa Kirin and Ultragenyx Pharmaceutical. About Kyowa Kirin Kyowa Kirin aims to discover novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology, and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at: https://www.kyowakirin.com References: Dahir K, et al. X-linked hypophosphatemia: a new era in management. J Endocr Soc. 2020;4(12):bvaa151. COR-US-CRY-0082 Oct 2026 Contact: Susan Thiele Head of Therapeutic Communications, North America media.northamerica.bt@kyowakirin.com

Clinical ResultClinical StudyDrug Approval

03 Oct 2026

·www.sciencedaily.com

Vitamin D drug may help crack pancreatic cancer’s protective shield

A vitamin D analog may help break down pancreatic cancer’s protective defenses, potentially making tumors more vulnerable to chemotherapy and immune attack. A small clinical trial found encouraging responses, especially in patients whose tumors had high levels of the vitamin D receptor.A small clinical trial led by researchers at Dana Farber Cancer Institute has tested a treatment strategy that began with laboratory discoveries at the Salk Institute. The idea is to activate the vitamin D receptor and, in doing so, alter the protective environment surrounding pancreatic tumors. That change could potentially make this notoriously difficult cancer more vulnerable to existing treatments. The study, published in Nature Cancer, included patients with previously untreated metastatic pancreatic cancer. Participants received standard chemotherapy either alone or in combination with paricalcitol, a vitamin D analog already approved by the FDA for other medical uses. Whether paricalcitol was given by mouth or intravenously, researchers found that it could be combined safely with chemotherapy overall. The drug also reduced the activation of fibroblasts in the tumor microenvironment, providing evidence in patients that supported earlier findings from Salk laboratory studies. Although the trial was not designed to determine whether the treatment improved pancreatic cancer outcomes, researchers observed encouraging signals. Patients who received paricalcitol with chemotherapy had more responses to treatment and were more likely to remain free of disease progression after one year. Those whose tumors had high levels of the vitamin D receptor and who received paricalcitol also had the longest overall survival. "This study really takes a novel approach for cracking therapeutic resistance in pancreatic cancer," says study co-author Ronald Evans, PhD, professor and the March of Dimes Chair in Molecular and Developmental Biology at Salk. "By using vitamin D analogs to engage the body's own natural system for dampening fibrotic and inflammatory responses, we can enable other therapies to do their job." How Vitamin D Research Led to a Cancer Trial Evans discovered the nuclear receptor superfamily, a major class of molecules that includes the vitamin D receptor. Nuclear receptors respond to substances such as hormones, vitamins, and lipids, then influence cell behavior by switching genes "on" and "off." About 13% of all FDA approved drugs currently work by targeting nuclear receptors. Earlier research by Evans and his colleagues also showed that the vitamin D receptor helps regulate fibroblasts in the liver and pancreas in preclinical models. Fibroblasts are connective tissue cells that can contribute to the dense protective structure surrounding pancreatic tumors and some other cancers. Those studies found particularly high levels of the vitamin D receptor in rare groups of fibroblasts that live within tissues. The receptor also appeared to play an important role in maintaining normal tissue health and stability. The Salk researchers further showed that synthetic forms of vitamin D, including paricalcitol, could block liver fibrosis and pancreatitis in experimental models. These compounds are designed to resist the natural process that normally breaks down vitamin D. Reprogramming the Environment Around Pancreatic Tumors Pancreatic cancer is known for producing an especially strong fibrotic response, so Evans and his team investigated whether vitamin D analogs could change the environment surrounding pancreatic tumors. The results surprised them. In pancreatic cancer models, vitamin D analogs were able to reverse the activation of cancer associated fibroblasts. That change was accompanied by stronger responses to chemotherapy. The work helped establish a different way of thinking about pancreatic cancer treatment. Rather than focusing only on killing cancer cells directly, researchers could try to reprogram the surrounding tissue that supports and protects the tumor. Testing the Strategy in Patients Building on the Salk findings, Brian Wolpin, MD, MPH, and Kimberly Perez, MD, at Dana Farber Cancer Institute worked with the Evans team to lead a randomized clinical study focused primarily on safety. The trial enrolled 36 people with previously untreated metastatic pancreatic cancer. Participants received standard chemotherapy (gemcitabine plus nab-paclitaxel) together with either a placebo, intravenous paricalcitol, or oral paricalcitol. Paricalcitol is already approved by the FDA to prevent and treat secondary hyperparathyroidism in people with chronic kidney disease. The primary purpose of the study was to determine whether adding paricalcitol to chemotherapy was safe. Overall, the combination was safely administered. However, five of the 12 patients who received oral paricalcitol developed elevated levels of calcium in their blood. Researchers were able to manage this side effect using standard reductions in dosage. Vitamin D Analog Alters Tumor Supporting Cells Another major goal was to determine whether paricalcitol actually produced measurable changes inside the tumors. Researchers collected biopsies when patients entered the study and again after four to six weeks of treatment. They then used advanced multiplex immunofluorescence and spatial transcriptomic techniques to examine changes in cancer cells and other cells within the tumor environment. Paricalcitol reduced the activation of fibroblasts inside the tumors (but not their overall numbers). It also increased the infiltration of T cells, immune cells that are typically prevented from entering pancreatic tumors. These results supported the idea that paricalcitol can remodel the tumor microenvironment, potentially making conditions around the cancer less protective. Encouraging Treatment Signals in a Small Trial The study was not designed to compare the effectiveness of the treatments or determine whether paricalcitol extends survival. Even so, researchers observed notable differences between the groups. Among the 24 patients who received paricalcitol, 10 had a partial response to treatment, equal to 42%. Only one of the 12 patients receiving placebo had a partial response, equal to 9%. Five patients in the paricalcitol groups also remained free of disease progression at the one year mark. None of the patients in the placebo group did. Researchers found another potentially important clue when they examined vitamin D receptor levels within the tumors. These levels varied considerably among patients and appeared to be associated with treatment outcomes. Patients with high vitamin D receptor levels who received paricalcitol responded better to chemotherapy and had the longest overall survival following treatment. Why Pancreatic Cancer Is So Difficult to Treat Pancreatic cancer remains among the most challenging cancers to treat successfully. Chemotherapy can slow the disease in some people, but outcomes remain poor, and the environment surrounding the tumor is thought to play an important role in treatment resistance. Pancreatic tumors are often wrapped in dense connective tissue rich in fibroblasts. This tissue can form a physical barrier that makes it harder for cancer drugs to reach tumor cells. It can also create an immunosuppressive environment that limits the ability of the immune system to attack the cancer. By altering this protective environment rather than targeting only the cancer cells themselves, vitamin D analogs could offer a way to make other therapies more effective. Larger Pancreatic Cancer Trials Are Needed The researchers say these results provide a foundation for larger clinical trials that can directly test whether combining vitamin D analogs with chemotherapy or other cancer treatments improves survival. Future studies will also need to determine whether the amount of vitamin D receptor present in a tumor before treatment can reliably predict which patients are most likely to benefit from therapies involving vitamin D analogs. "This study is an important step forward for the use of a vitamin D analog as a stromal remodeling therapy that can overcome therapeutic resistance in pancreatic," says Perez. "It was built upon foundational basic research at the Salk Institute, validates those preclinical findings in patients, and provides a road map for future studies that could someday establish a new treatment standard."

Clinical ResultDrug Approval

14 May 2026

·www.biospace.com

FDA Approves New Dosing Option for CRYSVITA® (burosumab-twza) in Adults With XLH

Expanded dosing options support individualized treatment for adults with XLH PRINCETON, N.J., May 14, 2026 (GLOBE NEWSWIRE) -- Kyowa Kirin, Inc., a wholly owned subsidiary of Kyowa Kirin Co. Ltd., today announced the U.S. Food and Drug Administration (FDA) has approved a dosing update to the CRYSVITA ® (burosumab-twza) Prescribing Information for adults living with X-linked hypophosphatemia (XLH), a rare, progressive, genetic disease that impacts the bones and muscles in both children and adults. Healthcare providers now have the option to increase the dose and frequency of CRYSVITA for patients whose serum phosphorus is below the normal range after the initial treatment period. These patients may be transitioned to a dosage of 0.5 mg/kg, not to exceed 90 mg, once every 2 weeks. If further dosage increase is needed after 4 weeks of treatment with 0.5 mg/kg every 2 weeks, the dosage can be increased to 1 mg/kg, not to exceed 90 mg, administered every 2 weeks. “Kyowa Kirin is committed to delivering solutions that make a meaningful difference for people living with XLH. As the only FDA-approved treatment for XLH, CRYSVITA plays a critical role in care, and it’s essential that clinicians have the flexibility to support optimal patient outcomes,” said Richard Wilson, Senior Vice President, Rare Disease Franchise Head. “While most adults with XLH maintain normal serum phosphorus levels with CRYSVITA administered every four weeks, this update enables a more personalized approach for those who need additional support to reach and maintain serum phosphorus levels within normal range.” To review the new dosing guidance, please see the full Prescribing Information for CRYSVITA, Section 2.3 (Recommended Dosage for Adult Patients with X-linked Hypophosphatemia, 18 years of age and older). About X-linked hypophosphatemia X-linked hypophosphatemia is a rare, lifelong, genetic disease that can impact the bones and muscles in both children and adults. In individuals with XLH, the body doesn't hold on to enough phosphorus, which is an essential mineral for bone health. This is due to the production of excess fibroblast growth factor 23 (FGF23), causing the body to release too much phosphorus through the urine. When phosphorus levels are too low (hypophosphatemia), it can cause the softening and weakening of growing bones in children (rickets) and of mature bones in children and adults (osteomalacia). About CRYSVITA ® (burosumab-twza) Injection CRYSVITA is a recombinant, fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, which binds to and inhibits the biological activity of FGF23, the underlying cause of hypophosphatemia in XLH. By blocking FGF23, CRYSVITA helps to restore renal phosphorus reabsorption and increase the serum concentration of active vitamin D. U.S. CRYSVITA Indication CRYSVITA is a fibroblast growth factor (FGF23) blocking antibody indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older. Important Safety Information CONTRAINDICATIONS CRYSVITA is contraindicated: In concomitant use with oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol) due to the risk of hyperphosphatemia. When serum phosphorus is within or above the normal range for age. In patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism. WARNINGS AND PRECAUTIONS Hypersensitivity Hypersensitivity reactions (e.g., rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment. Hyperphosphatemia and Risk of Nephrocalcinosis Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient’s serum phosphorus levels. Hypercalcemia Increases in serum calcium have been reported in patients treated with CRYSVITA. Patients with risk factors such as pre-existing hyperparathyroidism, prolonged immobilization, dehydration, hypervitaminosis D, or renal impairment, are at higher risk of hypercalcemia. Monitor these patients for serum calcium and parathyroid hormone levels before and during CRYSVITA treatment for moderate to severe hypercalcemia. In patients with moderate to severe hypercalcemia, CRYSVITA should not be administered until hypercalcemia is adequately managed. Injection Site Reactions Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment. ADVERSE REACTIONS Pediatric Patients Adverse reactions reported in 10% or more of CRYSVITA-treated pediatric XLH patients across three studies are: pyrexia (55%, 44%, and 62%), injection site reaction (52%, 67%, and 23%), cough (52%), vomiting (41%, 48%, and 46%), pain in extremity (38%, 46%, and 23%), headache (34% and 73%), tooth abscess (34%, 15%, and 23%), dental caries (31%), diarrhea (24%), vitamin D decreased (24%, 37%, and 15%), toothache (23% and 15%), constipation (17%), myalgia (17%), rash (14% and 27%), dizziness (15%), and nausea (10%). Adult Patients Adverse reactions reported in more than 5% of CRYSVITA-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain (15%), headache (13%), tooth infection (13%), restless legs syndrome (12%), vitamin D decreased (12%), dizziness (10%), constipation (9%), muscle spasms (7%), and blood phosphorus increased (6%). Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression. USE IN SPECIFIC POPULATIONS There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-844-768-3544. There is no information regarding the presence of CRYSVITA in human milk or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition. PATIENT COUNSELING INFORMATION Advise patients not to use any oral phosphate and/or active vitamin D analog products. Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless legs syndrome induction or worsening of symptoms occur. You may report side effects to the FDA at (800) FDA-1088 or . You may also report side effects to Kyowa Kirin, Inc. at 1-844-768-3544. For important risk and use information, please see the full Prescribing Information for CRYSVITA. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, Kyowa Kirin has invested in drug discovery and biotechnology innovation for more than 70 years and is currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology and rare diseases. A shared commitment to Kyowa Kirin’s values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at . CONTACT: Contact: Susan Thiele Head of Product and Therapeutic Communications, North America susan.thiele.38@kyowakirin.com

100 Deals associated with Paricalcitol

External Link

KEGG	Wiki	ATC	Drug Bank
D00930	Paricalcitol	H05BX02	DB00910

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hyperparathyroidism, Secondary	United States	AbbVie, Inc.	17 Apr 1998

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Albuminuria	Phase 3	Slovenia	Abbott Laboratories	01 Jul 2012
Kidney Diseases	Phase 3	Slovenia	Abbott Laboratories	01 Jul 2012
Kidney Failure, Chronic	Phase 3	-	AbbVie, Inc.	01 Oct 2011
Chronic Kidney Diseases	Phase 3	Sweden	Abbott Laboratories	01 Sep 2010
Diabetic Nephropathies	Phase 3	Sweden	Abbott Laboratories	01 Sep 2010
Coronary Artery Disease	Phase 3	United States	Abbott Laboratories	01 Jan 2010
Vitamin D Deficiency	Phase 3	United States	Abbott Laboratories	01 Jan 2010
Hypertrophy, Left Ventricular	Phase 3	United States	AbbVie, Inc.	01 Feb 2008
Hypertrophy, Left Ventricular	Phase 3	Australia	AbbVie, Inc.	01 Feb 2008
Hypertrophy, Left Ventricular	Phase 3	Czechia	AbbVie, Inc.	01 Feb 2008

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04064827 (CTgov)	Phase 3	Hyperparathyroidism, Secondary \| Chronic Kidney Diseases	2	Paricalcitol	tohkemfvdt = fzclqwxhds xxghngylmu (lwxjjkdoyo, sygyempuwl - xcyykuvqjq) View more	-	15 Jun 2026
NCT03415854 (NABPLAGEMD, CTgov)	Phase 2	Neoplasm Metastasis \| Metastatic Pancreatic Ductal Adenocarcinoma	34	Paricalcitol	kojfkmcmrd(negjiirvnb) = udtkkdsake mdgeulobhc (vrxdevbkmp, labbnmwdyn - hdihooixyg) View more	-	12 May 2026
NCT04524702 (CTgov)	Phase 2	Pancreatic adenocarcinoma metastatic \| Advanced Pancreatic Adenocarcinoma	10	Hydroxychloroquine+Nab-paclitaxel+Gemcitabine+Paricalcitol	xcogaebexs = kokmjulkxv xburictbfr (aiwomvqvwx, aelurgiacs - evztlgauwm) View more	-	23 Jan 2026
NCT04054362 \| NCT03415854 (PINBALL, AACR2024) Manual	Phase 2	Metastatic Pancreatic Ductal Adenocarcinoma First line	16	Paricalcitol	psuyfvomwd(ffkvuourbe) = btxnaseptt uspbbkkowy (zjsvaadjtx ) View more	Negative	05 Apr 2024
ISN WCN2024 Manual	Not Applicable	Chronic Kidney Diseases FGF23	20	Paricalcitol	riugpujwvl(ptmjdimgex) = Initial diastolic function Z-scores (n=11) were significantly different from mean normal controls (all p<0.05) suggesting DD. Follow-up Z-scores of diastolic function during Pc treatment (15.6 ± 12.4 μg/week) were similar to baseline. rebwnefqxk (dyhojqhgum ) View more	Positive	01 Apr 2024
NCT03519308 (CTgov)	Early Phase 1	Pancreatic Cancer	3	Nivolumab+Nab-Paclitaxel+Gemcitabine+Paricalcitol (Arm A)	bniucadqqz(fqjlrrgqet) = pxrltutjdy onlysuleun (vynrzlflae, 4.2) View more	-	15 Mar 2024
NCT03519308 (CTgov)	Early Phase 1	Pancreatic Cancer	3	Nivolumab+Nab-Paclitaxel+Gemcitabine (Arm B)	bniucadqqz(fqjlrrgqet) = itkusmqqul onlysuleun (vynrzlflae, 0) View more	-	15 Mar 2024
NCT01725113 (ECRIP, CTgov)	Phase 4	Hypercalcemia \| Hyperphosphatemia \| Renal Insufficiency ... View more	16	Calcitriol (Calcitriol)	pqejuluiuz(oywzwwxrfm) = uefqpdycxj txrvcxsxtl (gsvwuzucqh, 0.332) View more	-	07 Nov 2023
NCT01725113 (ECRIP, CTgov)	Phase 4		16	Paricalcitol (Paricalcitol)	zfwmkzeyyq(ebsimoyyrz) = zboqcjwgrz hcwrqcjhyz (oeplrhuwxs, 0.3) View more	-	07 Nov 2023
NCT03883919 (ASCO_GI2022) Manual	Phase 1	Pancreatic Cancer	20	5FU+LV+liposomal irinotecan+paricalcitol 75mcg IV (dose level 1)	mgmjvjunjm(frvzqopoma) = ttfrminyuk kinqsoljwv (yobhafxkzn ) View more	Negative	19 Jan 2022
NCT03883919 (ASCO_GI2022) Manual	Phase 1	Pancreatic Cancer	20	5FU+LV+liposomal irinotecan+paricalcitol 7mcg/kg IV (dose level 2)	mgmjvjunjm(frvzqopoma) = qgsotuxeyz kinqsoljwv (yobhafxkzn ) View more	Negative	19 Jan 2022
NCT02754726 (AACR2021) Manual	Phase 2	Metastatic Pancreatic Ductal Adenocarcinoma First line	32	nivolumab+albumin bound paclitaxel+paricalcitol+cisplatin+gemcitabine	snjvleguie(pbanolgpvg) = cvkljuzwxa ptbehoutkr (fosrzewlct, 5 - 8) View more	Positive	05 Nov 2021
ChiCTR2000031420 (ASN2021)	Phase 4	Hyperparathyroidism, Secondary serum intact parathyroid hormone (iPTH) \| serum calcium \| serum phosphorus	271	Paricalcitol-dominated therapy	ocbtxljgar(hstlrnqohx) = ujjukffglp ulqfsjxqzn (gkirsbhqfv ) View more	Positive	27 Oct 2021
ChiCTR2000031420 (ASN2021)	Phase 4		271	Cinacalcet-dominated therapy	ocbtxljgar(hstlrnqohx) = utemkgjvvt ulqfsjxqzn (gkirsbhqfv ) View more	Positive	27 Oct 2021

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