Over the past decades, healthcare systems face significant challenges to meet the needs of an aging population due to progressive debility, functional decline and chronic diseases development. While there is a growing appreciation of the potential impact of mTOR inhibitors on slowing aging processes, preventing chronic disease and prolonging healthy lifespan, a major challenge in developing clinical trials to establish the clinical efficacy of mTOR inhibitors is the absence of pharmacokinetics (PK) and pharmacodynamics (PD) data in older adults. The proposed study will provide the foundation for future clinical trials assessing the role of mTOR inhibitors on aging related indications

Start Date01 May 2025

Sponsor / Collaborator

The University of Texas Southwestern Medical Center

[+2]

NCT06752694

/ Not yet recruitingPhase 2

Ruxolitinib Based GVHD Prophylaxis Regimen for Older Adults Receiving Non-ATG Containing Non-Myeloablative Hematopoietic Cell Transplantation for Acquired Aplastic Anemia

This phase II trial tests how well a ruxolitinib-based graft versus host disease (GVHD) prevention (prophylaxis) regimen works before, during, and after bone marrow/stem cell transplantation (hematopoietic cell transplantation [HCT]) in patients with acquired aplastic anemia. Acquired aplastic anemia (AA) is a condition in which the bone marrow is unable to produce blood cells. Affected patients typically present with infections due to abnormally low number of neutrophils, bleeding due to low platelet count, and/or fatigue due to a lower-than-normal number of red blood cells (anemia). Its incidence varies with age, occurring most frequently in patients aged 2-5 years, 20-25 years, and 55 years and older. Treatment of AA includes either immunosuppressive therapy (IST) or bone marrow/stem cell transplantation (HCT) with first-line therapy in younger adults often being HCT, while adults over 40 still frequently trial IST first due to the morbidity and mortality concerns with HCT. GVHD is a common complication after donor stem cell transplantation, resulting from donor immune cells recognizing recipients' cells and attacking them. Ruxolitinib, a drug in a class of oral medications called JAK inhibitors has been approved for the treatment of acute and chronic GVHD. It has also been shown to decrease GVHD when used in the prevention setting in patients with myelofibrosis. The current study aims to assess whether adding ruxolitinib to a standard GVHD prevention regimen may reduce the risk of Grade II-IV acute and chronic GVHD after bone marrow/stem cell transplantation in older patients with acquired aplastic anemia.

Start Date15 Feb 2025

Sponsor / Collaborator

Fred Hutchinson Cancer Center

[+1]

NCT04370795

/ Enrolling by invitationPhase 1/2IIT

Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus

Background:
Severe combined immune deficiency (SCID) is a group of conditions where the immune system does not work properly. The only cure for most SCIDs is a stem cell transplant (getting cells from a donor). These transplants can have serious complications. Before the transplant, people often get high doses of drugs and radiation to prepare the body to accept the cells from the donor. Researchers want to see if low doses of drugs alone without radiation work just as well as low doses of drugs with radiation for SCID patients getting stem cell transplants.
Objective:
To test a set of drugs with or without radiation given before a stem cell transplant.
Eligibility:
People ages 3-40 who have SCID and who have a stem cell donor - either related or unrelated.
Design:
Participants will be admitted to the hospital 10 days before transplant. They will undergo:
medical history
medication review
physical exam
blood and urine tests (may include a 24-hour urine collection)
heart, lung, and breathing tests
imaging scans
bone marrow sample
nutrition assessment
dental exam
eye exam
meeting with a social worker.
Participants will get a plastic port called a central line. It is a hollow tube that is placed in the upper chest. It will be used to give medicines and take blood.
All participants will take chemotherapy drugs. Some will get radiation.
Participants will have a stem cell transplant. They will get the cells as an infusion through their central line. They will stay in the hospital for 30 days after transplant.
Participants must stay within 1 hour of NIH for 3 months after transplant. During this time, they will have follow-up visits at NIH at least once a week. Then they will have follow-up visits once or twice a year for 5-6 years.

Start Date22 Jan 2025

Sponsor / Collaborator

National Institute of Allergy & Infectious Diseases

100 Clinical Results associated with Sirolimus

100 Translational Medicine associated with Sirolimus

100 Patents (Medical) associated with Sirolimus

25,765

Literatures (Medical) associated with Sirolimus

01 Dec 2025·Journal of Clinical Immunology

Report of the Italian Cohort with Activated Phosphoinositide 3-Kinase δ Syndrome in the Target Therapy Era

Article

Author: Montin, Davide ; De Rosa, Antonio ; Ricci, Silvia ; Marinoni, Maddalena ; Trizzino, Antonino ; Moratti, Mattia ; Rivalta, Beatrice ; Zecca, Marco ; Tommasini, Alberto ; Marzollo, Antonio ; Barzaghi, Federica ; Baselli, Lucia Augusta ; Badolato, Raffaele ; Cancrini, Caterina ; Conti, Francesca ; Lougaris, Vassilios ; Lodi, Lorenzo ; Giardino, Giuliana ; Pignata, Claudio ; Panza, Giuseppina ; Costagliola, Giorgio ; Martire, Baldassarre ; Chinello, Matteo ; Milito, Cinzia

BACKGROUNDActivated Phosphoinositide 3-Kinase (PI3K) δ Syndrome (APDS), an inborn error of immunity due to upregulation of the PI3K pathway, leads to recurrent infections and immune dysregulation (lymphoproliferation and autoimmunity).METHODSClinical and genetic data of 28 APDS patients from 25 unrelated families were collected from fifteen Italian centers.RESULTSPatients were genetically confirmed with APDS-1 (n = 20) or APDS-2 (n = 8), with pathogenic mutations in the PIK3CD or PIK3R1 genes. The median age at diagnosis was 15.5 years, with a median follow-up of 74 months (range 6-384). The main presenting symptoms were respiratory tract infections alone (57%) or associated with lymphoproliferation (17%). Later, non-clonal lymphoproliferation was the leading clinical sign (86%), followed by respiratory infections (79%) and gastrointestinal complications (43%). Malignant lymphoproliferative disorders, all EBV-encoding RNA (EBER)-positive at the histological analysis, occurred in 14% of patients aged 17-19 years, highlighting the role of EBV in lymphomagenesis in this disorder. Diffuse large B-cell lymphoma was the most frequent. Immunological work-up revealed combined T/B cell abnormalities in most patients. Treatment strategies included immunosuppression and PI3K/Akt/mTOR inhibitor therapy. Rapamycin, employed in 36% of patients, showed efficacy in controlling lymphoproliferation, while selective PI3Kδ inhibitor leniolisib, administered in 32% of patients, was beneficial on both infections and immune dysregulation. Additionally, three patients underwent successful HSCT due to recurrent infections despite ongoing prophylaxis or lymphoproliferation poorly responsive to Rapamycin.CONCLUSIONSThis study underscores the clinical heterogeneity and challenging diagnosis of APDS, highlighting the importance of multidisciplinary management tailored to individual needs and further supporting leniolisib efficacy.

01 Dec 2025·Journal of Clinical Immunology

Novel Compound Heterozygous Variants in the FAS Gene Lead to Fetal Onset of Autoimmune Lymphoproliferative Syndrome (ALPS)

Article

Author: Yu, Meiping ; Wu, Bingbing ; Zhu, Li ; Chen, Yao ; Li, Qifan ; Wang, Chenghao ; Qian, Feng ; Zhou, Weitao ; Liu, Luyao ; Wu, Qi ; Hui, Xiaoying ; Zhou, Qinhua ; Hou, Jia ; Wang, Xiaochuan ; Sun, Bijun ; Wang, Ying ; Sun, Jinqiao ; Ying, Wenjing ; Wang, Wenjie

OBJECTIVEFAS gene defects lead to autoimmune lymphoproliferative syndrome (ALPS), which is often inherited in an autosomal dominant and rarely in an autosomal recessive manner. We report a case of a newborn girl with novel compound heterozygous variants in FAS and reveal the underlying mechanism.METHODSWhole-exome sequencing (WES) was used to identify pathogenic variants. Multiparametric flow cytometry analysis, phosflow analysis, and FAS-induced apoptosis assays were used to explore the effects of the variants on FAS expression, apoptosis, and immunophenotype. The HEK293T cells were used to assess the impact of the variants on protein expression and FAS-induced apoptosis.RESULTSThe patient was born with hepatosplenomegaly, anemia, and thrombocytopenia. She also experienced COVID-19, rotavirus infection, herpes simplex virus infection, and severe pneumonia. The proportion of double-negative T cells (DNTs) was significantly elevated. Novel FAS compound heterozygous variants c.310T > A (p.C104S) and c.702_704del (p.T235del) were identified. The apoptotic ability of T cells was defective, and FAS expression on the surface of T cells was deficient. The T235del variant decreased FAS expression, and the C104S protein remained in the endoplasmic reticulum (ER) and could not translocate to the cell surface. Both mutations resulted in loss-of-function in terms of FAS-induced apoptosis in HEK293T cells. The DNTs were mainly terminally differentiated T (TEMRA) and CD45RA⁺HLA-DR⁺, with high expression of CD85j, PD-1, and CD57. The percentage of Th1, Tfh, and autoreactive B cells were significantly increased in the patient. The abnormal immunophenotyping was partially attenuated by sirolimus treatment.CONCLUSIONSWe identified two variants that significantly affect FAS expression or localization, leading to early disease onset of in the fetus. Abnormalities in the mTOR pathway are associated with a favorable response to sirolimus.

01 Dec 2025·Molecular Biology Reports

Rapamycin and Niacin combination induces apoptosis and cell cycle arrest through autophagy activation on acute myeloid leukemia cells

Article

Author: Akçok, İsmail ; Gencer Akçok, Emel Başak ; Subay, Lale Beril

BACKGROUNDAcute myeloid leukemia (AML) is a heterogeneous hematological malignancy caused by disorders in stem cell differentiation and excessive proliferation resulting in clonal expansion of dysfunctional cells called myeloid blasts. The combination of chemotherapeutic agents with natural product-based molecules is promising in the treatment of AML. In this study, we aim to investigate the anti-cancer effect of Rapamycin and Niacin combination on THP-1 and NB4 AML cell lines.METHODS AND RESULTSThe anti-proliferative effects of Rapamycin and Niacin were determined by MTT cell viability assay in a dose- and time-dependent manner. The combination indexes were calculated by isobologram analysis. Furthermore, apoptosis was investigated by Annexin-V/Propidium Iodide(PI) double staining and cell cycle distribution was measured by PI staining. The expression levels of autophagy-related proteins were detected by western blotting. The combination of Rapamycin and Niacin synergistically decreased cell viability of AML cell lines. The combination treatment induced the apoptotic cell population of THP-1 and NB4 by 4.9-fold and 7.3-fold, respectively. In THP-1 cells, the cell cycle was arrested at the G2/M phase by 10% whereas the NB4 cells were accumulated at the G0/G1 phase. The combination treatment decreased Akt and p-Akt expression. Besides, the ATG7 expression was reduced by combination treatment on THP-1 cells. Similarly, the ATG5 level was downregulated in NB4 cells. The level of LC3B-II/LC3B-I, which is an indicator of autophagy flux, was upregulated in THP-1 and NB4 cells.CONCLUSIONAlthough further studies are required, the combination of Rapamycin and Niacin combats cell proliferation by inducing cellular apoptosis, cell cycle arrest and autophagy activation.

337

News (Medical) associated with Sirolimus

20 Jan 2025

·www.prnewswire.com

SIRPAD - WORLD'S LARGEST RCT INVESTIGATING MAGICTOUCH PTA SIROLIMUS COATED BALLOON COMPLETES PATIENT ENROLMENT

TAMPA, Fla., Jan. 20, 2025 /PRNewswire/ -- Concept Medical Inc. (CMI), a global leader in innovative drug-delivery technology across vascular interventions, proudly announces the successful completion of patient enrolment in the SirPAD Trial, with over 1,250 patients now enrolled. Continue Reading Concept Medical proudly announces that over 1,250 patients have successfully enrolled in the SirPAD Trial. The SirPAD (Major adverse limb events in patients with femoro-popliteal and below-the-knee peripheral arterial disease treated with either sirolimus-coated or uncoated balloon) randomised controlled trial is the world's largest study evaluating the treatment of Peripheral Artery Disease (PAD) using the MagicTouch PTA Sirolimus Coated Balloon (Concept Medical Inc.) versus uncoated balloons and one of the largest device studies ever performed for PAD. More than 1,250 patients have been enrolled and will be followed up at 12 months to assess the primary outcome of major adverse limb events (MALE). Final results are expected by Q1-Q2 2026. SirPAD is an investigator-initiated, multi-centre, randomized, open-label trial that aims to determine whether the MagicTouch PTA sirolimus-coated balloon is non-inferior to plain old balloon angioplasty (POBA). The trial will also explore the potential for superiority. Principal Investigators Prof. Dr. med. Nils Kucher and Prof. Dr. med. Stefano Barco, from the University Hospital Zurich, who leads this ambitious study, said, "Sirolimus-coated balloons represent a promising technology in treating symptomatic PAD, supported by evidence primarily from studies with surrogate endpoints. While trials like SIRONA and ongoing studies such as SirPAD are expanding knowledge, robust data from randomized controlled trials with clinical outcomes are needed to guide treatment and shape future guidelines. This result, having been able to randomize so many patients in a PAD device study, is unprecedented and we are very thankful to all investigators and patients for having supported the trial" on achieving the significant milestone of enrolling 1,250+ patients and successfully completing this phase of the study. With sirolimus-coated balloons gathering global momentum and MagicTouch PTA at the forefront, SirPAD is a crucial milestone in expanding the evidence for PAD treatment options. While patients with PAD have limited therapeutic avenues, SirPAD aims to bolster data supporting sirolimus-coated balloon technology. This large RCT was critical to evaluating treatment alternatives, and its year-long follow-up will provide much-anticipated answers. "At Concept Medical, we are committed to generating robust clinical evidence to support our innovative technologies. Following the successful completion of SIRONA—the largest superficial femoral artery (SFA) trial comparing our device against paclitaxel drug-coated balloons (DCBs), which demonstrated non-inferiority—we are proud to announce the completion of enrollment for another historical milestone: the largest randomised clinical trial for peripheral all-comers patients, completed enrolment of 1250+ patients. With this achievement, Concept Medical continues to lead the way with the most extensive and advanced clinical trial program in the field, reinforcing our position as a global pioneer in patient-centric vascular innovations," said Dr. Manish Doshi, Founder & Managing Director of Concept Medical. About Concept Medical Concept Medical Inc., headquartered in Tampa, Florida, has a global presence and is dedicated to enhancing patient care through cutting-edge research and development of drug-delivery technologies. Its proprietary platforms are designed to deliver pharmaceutical agents across vascular luminal surfaces with unparalleled precision. Concept Medical is the developer of the MagicTouch family of Sirolimus Coated Balloons (SCBs)—the world's first and most utilized SCB technology—well recognized for its versatility and efficacy in treating coronary and peripheral artery disease. The revolutionary MagicTouch and Abluminus product lines have been used to treat over a million patients globally, setting a new standard for vascular therapy. For more information, please visit Photo: Logo: SOURCE Concept Medical WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical Study

16 Jan 2025

·www.prnewswire.com

Concept Medical Announces First Patient Enrollment in the MAGICAL BTK IDE Trial, Launching its PAD Clinical Program in the United States

TAMPA, Fla., Jan. 16, 2025 /PRNewswire/ -- Concept Medical Inc., a global pioneer in innovative drug delivery technology, is proud to announce the successful enrolment of the first patient in the "MAGICAL BTK" (Randomized Controlled Trial of MagicTouch PTA – Sirolimus Coated Balloon Versus Standard Balloon Angioplasty in the Treatment of Below The Knee arterial disease) U.S. Investigational Device Exemption (IDE) pivotal trial. The first patient was enrolled by Dr. Prakash Krishnan, MD, and his team at the Icahn School of Medicine at Mount Sinai, marking a crucial milestone in the advancement of treatment options for patients suffering from peripheral artery disease (PAD) below the knee. Continue Reading First patient enrollment in MAGICAL-BTK IDE Study in the United States. Following the recent IDE Approval by the U.S. Food and Drug Administration (FDA) for MagicTouch PTA, this enrolment heralds the beginning of Concept Medical's peripheral clinical trial program in the United States. With the ongoing IDE study for MagicTouch SCB in coronary artery disease, Concept Medical is now actively enrolling in two U.S. clinical trials in both coronary and peripheral interventions—an unprecedented achievement aimed at improving patient outcomes and expanding therapeutic choices. "The enrolment of the first patient in the MAGICAL BTK trial represents an important stride toward improving the standard of care for below-the-knee disease in patients with peripheral artery disease," said Dr. Prakash Krishnan, MD, Icahn School of Medicine at Mount Sinai. "Sirolimus-coated balloon technology has the potential to significantly enhance limb salvage and patient quality of life. I am honored to be part of this landmark study and look forward to the meaningful clinical evidence it will generate." The MAGICAL BTK pivotal trial is designed to evaluate the safety and effectiveness of MagicTouch PTA Sirolimus Coated Balloon compared to standard percutaneous transluminal angioplasty (PTA) for the treatment of below-the-knee arterial disease. The primary endpoint, measured at 12 months, is primary patency, ensuring that the trial's findings focus on meaningful clinical outcomes. This global, multi-center study is led by Prof. Sahil Parikh (Chairperson), with a distinguished panel of Principal Investigators including Dr. Eric Secemsky (Beth Israel Deaconess Hospital and Harvard Medical School), Prof. Brian DeRubertis (NY Presbyterian Weill Cornell Medical Center and Cornell University Medical College), Prof. Edward Choke (Northern Heart Hospital), and Prof. Osamu Iida (Osaka Keisatsu Hospital). "The initiation of the MAGICAL BTK Trial heralds a new era in the treatment of patients with chronic limb-threatening ischemia and offers hope to the millions of patients with this condition who are at risk for limb loss because of insufficient arterial circulation in their legs," stated Prof. Sahil Parikh. Sirolimus-coated balloon technology has already reshaped coronary and peripheral artery disease management, with MagicTouch achieving positive results in large-scale trials across Asia and Europe.. Now, with the MAGICAL BTK trial, U.S. patients have the opportunity to benefit from this breakthrough approach. Concurrently, patient enrolment from Asia will broaden the global patient cohort, offering a more comprehensive understanding of the therapy's benefits across diverse populations. "At Concept Medical, our unwavering commitment to innovation and patient care drives us to push the boundaries of what is possible in vascular interventions," said Dr. Manish Doshi (Founder & Managing Director). "The start of MAGICAL BTK further validates our dedication to bringing next-generation, evidence-based solutions to clinicians and patients worldwide. Our goal is to redefine current standards, ensuring better outcomes and improved quality of life for those affected by PAD." With the MAGICAL BTK trial and the MAGICAL SFA IDE trial for superficial femoral artery (SFA) treatment set to begin, Concept Medical is poised to revolutionise endovascular care. The company's distinctive technology platform has already elevated the management of coronary artery disease, and now, by expanding its reach into peripheral interventions, Concept Medical reinforces its leading position in advancing patient-focused, drug-delivery solutions. About Concept Medical Concept Medical Inc., headquartered in Tampa, Florida, has a global presence and is dedicated to enhancing patient care through cutting-edge research and development of drug-delivery technologies. Its proprietary platforms are designed to deliver pharmaceutical agents across vascular luminal surfaces with unparalleled precision. Concept Medical is the developer of the MagicTouch family of Sirolimus Coated Balloons (SCBs)—the world's first and most utilized SCB technology—well recognized for its versatility and efficacy in treating coronary and peripheral artery disease. The revolutionary MagicTouch and Abluminus product lines have been used to treat over a million patients globally, setting a new standard for vascular therapy. For more information, please visit . Photo - Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical Study

13 Jan 2025

·www.globenewswire.com

R3 Vascular Secures WCG IRB Approval and CMS Category B Medicare Coverage for the ELITE-BTK Pivotal Trial of its MAGNITUDE® Drug Eluting Next Generation Bioresorbable Scaffold for Below-the-Knee Peripheral Arterial Disease

MOUNTAIN VIEW, Calif., Jan. 13, 2025 (GLOBE NEWSWIRE) -- R3 Vascular Inc., a medical device company dedicated to developing and providing novel, best-in-class bioresorbable scaffolds for treating peripheral arterial disease (PAD), is pleased to announce that it received WCG Institutional Review Board (IRB) approval for the ELITE-BTK Pivotal Trial of its MAGNITUDE Drug Eluting Next Generation Bioresorbable Scaffold for Below-the-Knee Peripheral Arterial Disease (PAD). This approval allows the trial to take place at institutions that utilize WCG IRB as their central IRB in the United States. Additionally, the company announced that the Centers for Medicare & Medicaid Services (CMS) has granted Category B approval for the investigational device exemption (IDE) study, ensuring Medicare coverage for the device, related and routine items, and services for the ELITE-BTK trial. Commenting on the announcements, Christopher M. Owens, President and Chief Executive Officer of R3 Vascular said, “We are very pleased to have received IRB and CMS Category B approval so quickly. These important milestones validate the potential of R3 Vascular’s break-through technology and help accelerate our path to first enrollment of our ELITE-BTK Pivotal Trial to measure the impact of MAGNITUDE on patient outcomes and its ability to meet this critical and growing clinical need.” Josh Smale, Vice President of Global Clinical and Scientific Affairs of R3 Vascular added, “Securing WCG and CMS approvals enables us to move forward quickly with investigational site activation activities, which brings us one step closer to first subject enrollment. I’m extremely proud of the great work our team has done to continue to accelerate and maintain our momentum between these milestones, as well as our continued urgency to get this next generation product delivered to patients.” Eric A. Secemsky, M.D., MSc, Director of Vascular Intervention at Beth Israel Deaconess Medical Center in Boston, Massachusetts and lead investigator for the ELITE-BTK pivotal trial said, “R3 Vascular’s novel approach in the design of its next generation bioresorbable drug eluting scaffold is of great interest to the physician community and has the potential to transform the field of peripheral interventions. We are eager to assess the impact of the MAGNITUDE scaffold on patient outcomes, which is now even closer to reality with the completion of these latest important milestones.” The National Institutes of Health estimates PAD affects 8-12 million people in the U.S. and one in 20 Americans over the age of 50. The global peripheral vascular device market was valued at $12.9 billion in 2023 and is expected to reach $17.4 billion by 2030 with significant growth coming from the below the knee segment. In May of 2024, R3 Vascular announced the closing of its $87 million Series B financing round to support the ELITE-BTK IDE pivotal trial as well as additional research and development, global regulatory submissions, scale up of manufacturing processes, and initial commercialization. About R3 Vascular Inc.R3 Vascular is a privately-held medical device company that develops a novel technology platform for the next generation of fully bioresorbable drug eluting sirolimus coated vascular scaffolds. These are designed to deliver the ‘stent-like’ support of a scaffold along with the anti-inflammatory and anti-proliferative result of sirolimus, but ‘disappearing’ over time as the vessel heals. R3 Vascular is headquartered in Mountain View, California. More information can be found at www.r3vascular.com. Media Contact:David Gutierrez, Dresner Corporate Services, (312) 780-7204, dgutierrez@dresnerco.com

Clinical Study

100 Deals associated with Sirolimus

External Link

KEGG	Wiki	ATC	Drug Bank
D00753	Sirolimus	L04AA10 S01XA23	DB00877

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Vascular Malformations	JP	Nobelpharma Co., Ltd.	18 Jan 2024
Vascular Neoplasms	JP	Nobelpharma Co., Ltd.	18 Jan 2024
Angiofibroma	EU	Pfizer Inc.	15 May 2023
Angiofibroma	IS	Pfizer Inc.	15 May 2023
Angiofibroma	LI	Pfizer Inc.	15 May 2023
Angiofibroma	NO	Pfizer Inc.	15 May 2023
Tuberous Sclerosis	CN	Nobelpharma Co., Ltd.	28 Mar 2023
Angiofibroma associated with tuberous sclerosis	US	Nobelpharma Co., Ltd.	22 Mar 2022
Lymphangiectasis	JP	Nobelpharma Co., Ltd.	27 Sep 2021
Lymphangioma	JP	Nobelpharma Co., Ltd.	27 Sep 2021
Osteolysis, Essential	JP	Nobelpharma Co., Ltd.	27 Sep 2021
Lymphangioleiomyomatosis	IS	Pfizer Europe MA EEIG	13 Mar 2001
Lymphangioleiomyomatosis	EU	Pfizer Europe MA EEIG	13 Mar 2001
Lymphangioleiomyomatosis	NO	Pfizer Europe MA EEIG	13 Mar 2001
Lymphangioleiomyomatosis	LI	Pfizer Europe MA EEIG	13 Mar 2001
Renal transplant rejection	US	PF Prism CV	15 Sep 1999

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Focal Cortical Dysplasia of Taylor	Phase 3	JP	Nobelpharma Co., Ltd.	-
Liver transplant rejection	Preclinical	FR	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	BE	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	PT	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	NL	Wyeth AB	01 Dec 2002
Liver transplant rejection	Preclinical	CH	Wyeth AB	01 Dec 2002
Allograft Rejection	Discovery	-	Wyeth AB	01 Aug 2002
Graft vs Host Disease	Discovery	US	Wyeth AB	20 May 2002
Graft vs Host Disease	Discovery	CA	Wyeth AB	20 May 2002
Kidney Diseases	Discovery	-	Wyeth AB	01 Jul 2000

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05050149 (CTgov)	Phase 2	Lymphatic Abnormalities	12	PTX-022	wrsiyfdqgs(fnaxbjxqco) = cjclavxogd lepaedyhfb (dxdkpnirwq, vwkdaadsxe - baxuqnoywt) View more	-	17 Jan 2025
SIRONA (PRNewswire) Manual	Not Applicable	Peripheral Arterial Disease	482	MagicTouch PTA	fdwrjdshzn(rsejdzxemc) = yirbffaqvs mmkozukwfr (hjpseaxlme ) Met View more	Similar	08 Jan 2025
				紫杉醇涂层球囊	fdwrjdshzn(rsejdzxemc) = bmjwbmybzl mmkozukwfr (hjpseaxlme ) Met View more
NCT04339101 (1043, ASH2024)	Phase 2	Myelofibrosis \| acute leukemia \| Myelodysplastic Syndromes HGF \| IL17 \| TNFaR ... View more	59	Itacitinib + Tacrolimus/Sirolimus	hnxgemkmnv(sdvmkowycy) = eyvsriabem ybwsjfreqs (ypgqejzjut, 41 - 66) View more	Positive	09 Dec 2024
				Tacrolimus/Sirolimus	hnxgemkmnv(sdvmkowycy) = nkmvaikfub ybwsjfreqs (ypgqejzjut )
ChiCTR1900020657 (1187Sirolimus, ASH2024)	Phase 3	Purpura, Thrombocytopenic, Idiopathic First line	-	HD-DXM plus sirolimus	ruwferdfte(yevjowmdcf) = zqjrprjgjd thvrfundbw (vuxpbybper ) View more	Positive	07 Dec 2024
				HD-DXM monotherapy	ruwferdfte(yevjowmdcf) = zuvbpbvbdn thvrfundbw (vuxpbybper ) View more
NCT02105766 (CTgov)	Phase 2	Beta-Thalassemia \| Hematopoietic stem cell transplantation \| Anemia, Sickle Cell \| Graft vs Host Disease	56	Radiotherapy+cyclophosphamide+Sirolimus+Pentostatin+Alemtuzumab (Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor)	gbikrgadqh(hvgvcpatgu) = ymajoiioyk ybzwtvuvjv (lupncrkfda, mkriipixhc - ngcfjxjtzh) View more	-	29 Nov 2024
				Radiotherapy+cyclophosphamide+Sirolimus+Pentostatin+Alemtuzumab (Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant)	shqqzmbubh(ufspkskxni) = zajbwpawsm uwdbwdobzl (iiftteqida, apndikaqpz - yenfglowwa) View more
NCT03095703 (CTgov)	Phase 2	Adenomatous Polyposis Coli	4	Sirolimus	teqaeljezs(rfsnlgcwhe) = uqfzmpdlsa oyduxywexf (haixhjdtuu, xkkhaoxjss - vvetgwsoab) View more	-	24 Oct 2024
NCT04348292 (CTgov)	Phase 1	Resectable Lung Non-Small Cell Carcinoma \| Non-small cell lung cancer stage IIIA	3	Sirolimus+Durvalumab	qxwwtechmw(svbhwqmbvw) = ckrctovzcb qkoemubbqa (ooekfmvsdo, hssilvzawl - ifkfjxoswu) View more	-	22 Aug 2024
NCT03077542 (CTgov)	Phase 1/2	Anemia, Sickle Cell	57	Filgrastim	ganuyebizb(haqaqwfwie) = rqnsjavrur seuuhgxouw (rqfhnzsfcb, zszrgkwicr - kfqlnpxgah) View more	-	07 Aug 2024
NCT04393454 (CTgov)	Phase 2	Metastatic Solid Tumor \| Mismatch repair-deficient Solid Tumors \| Microsatellite instability-high cancer	6	Sirolimus 2mg Tablet	(yxxddcvbvs) = odzrfgflym pvlzcoxnfi (nwgktsxlde, agkxysyleq - gcyfxlgevq) View more	-	25 Jul 2024
NCT01467986 (RIST-rNB-2011, Pubmed) Manual	Phase 2	Neuroblastoma	129	dasatinib-rapamycin	(wlbmjlbdlo) = uwqvnqeqqc aguiesurnn (yrmxaivioo, 7 - 17) View more	Positive	01 Jul 2024
				irinotecan-temozolomide	(wlbmjlbdlo) = xjrdxxgzxo aguiesurnn (yrmxaivioo, 2 - 8) View more

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