A Phase 1/2 Safety Study in Subjects With CHM (Choroideremia) Gene Mutations Using an Adeno-Associated Virus Serotype 2 Vector to Deliver the Normal Human CHM Gene [AAV2-hCHM] to the Retina

This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations.

Start Date15 Jan 2015

Sponsor / Collaborator

Spark Therapeutics, Inc.

100 Clinical Results associated with SPK-7001

100 Translational Medicine associated with SPK-7001

100 Patents (Medical) associated with SPK-7001

637

Literatures (Medical) associated with SPK-7001

01 Nov 2024·Chinese Journal of Integrative Medicine

Patient-Reported Outcomes of Postoperative NSCLC Patients with or without Staged Chinese Herb Medicine Therapy during Adjuvant Chemotherapy (NALLC 2): A Randomized, Double-Blind, Placebo-Controlled Trial

Article

Author: Qian, Cheng-Xin ; Yao, Jia-Lin ; Yao, Yi-qin ; Xu, Ling ; Chen, Zhi-Wei ; Su, Ling-zi ; Xu, Jian-fang ; Jiao, Li-jing ; Wang, Yi-chao ; Zhang, Jie ; Zhang, Yuan-Hui ; Wang, Yi-Chao ; Zhang, Yi-lu ; Li, Jia-Qi ; Ni, Jian ; Gong, Ya-Bin ; Shen, Xiao-Yong ; Yao, Yi-Qin ; Wang, Li-Yu ; Yang, Wen-xiao ; Chen, Zhi-wei ; Yang, Wen-Xiao ; Li, Jia-qi ; Xu, Jian-Fang ; Gong, Ya-bin ; Wang, Li-yu ; Qian, Cheng-xin ; Zhou, Di ; Zhang, Yuan-hui ; Su, Ling-Zi ; Zhang, Yi-Lu ; Yao, Jia-lin ; Shen, Xiao-yong ; Jiao, Li-Jing ; Wang, Qin

OBJECTIVE:

To investigate whether the combination of chemotherapy with staged Chinese herbal medicine (CHM) therapy could enhance health-related quality of life (QoL) in non-small-cell lung cancer (NSCLC) patients and prolong the time before deterioration of lung cancer symptoms, in comparison to chemotherapy alone.

METHODS:

A prospective, double-blind, randomized, controlled trial was conducted from December 14, 2017 to August 28, 2020. A total of 180 patients with stage I B-IIIA NSCLC from 5 hospitals in Shanghai were randomly divided into chemotherapy combined with CHM (chemo+CHM) group (120 cases) or chemotherapy combined with placebo (chemo+placebo) group (60 cases) using stratified blocking randomization. The European Organization for Research and Treatment of Cancer (EORTC) Quality-of-Life-Core 30 Scale (QLQ-C30) was used to evaluate the patient-reported outcomes (PROs) during postoperative adjuvant chemotherapy in patients with early-stage NSCLC. Adverse events (AEs) were assessed in the safety analysis.

RESULTS:

Out of the total 180 patients, 173 patients (116 in the chemo+CHM group and 57 in the chemo+placebo group) were included in the PRO analyses. The initial mean QLQ-C30 Global Health Status (GHS)/QoL scores at baseline were 57.16 ± 1.64 and 57.67 ± 2.25 for the two respective groups (P>0.05). Compared with baseline, the chemo+CHM group had an improvement in EORTC QLQ-C30 GHS/QoL score at week 18 [least squares mean (LSM) change 17.83, 95% confidence interval (CI) 14.29 to 21.38]. Conversely, the chemo+placebo group had a decrease in the score (LSM change -13.67, 95% CI -22.70 to -4.63). A significant between-group difference in the LSM GHS/QoL score was observed, amounting to 31.63 points (95% CI 25.61 to 37.64, P<0.001). The similar trends were observed in physical functioning, fatigue and appetite loss. At week 18, patients in the chemo+CHM group had a higher proportion of improvement or stabilization in GHS/QoL functional and symptom scores compared to chemo+placebo group (P<0.001). The median time to deterioration was longer in the chemo+CHM group for GHS/QoL score [hazard ratio (HR)=0.33, 95% CI 0.23 to 0.48, P<0.0010], physical functioning (HR=0.43, 95% CI 0.25 to 0.75, P=0.0005), fatigue (HR=0.47, 95% CI 0.30 to 0.72, P<0.0001) and appetite loss (HR=0.65, 95% CI 0.42 to 1.00, P=0.0215). The incidence of AEs was lower in the chemo+CHM group than in the chemo+placebo group (9.83% vs. 15.79%, P=0.52).

CONCLUSION:

The staged CHM therapy could help improve the PROs of postoperative patients with early-stage NSCLC during adjuvant chemotherapy, which is worthy of further clinical research. (Registry No. NCT03372694).

10 Sep 2024·Pharmaceuticals

Adjunctive Therapy with Chinese Herbal Medicine Lowers Risk of Hearing Loss in Type 2 Diabetes Patients: Results from a Cohort-Based Case-Control Study.

Article

Author: Livneh, Hanoch ; Chen, Wei-Jen ; Tsai, Tzung-Yi ; Lu, Ming-Chi ; Huang, Hui-Ju ; Yen, Chieh-Tsung

Hearing loss is a frequently observed complication of type 2 diabetes (T2D). Emerging evidence has found that Chinese herbal medicine (CHM) can effectively treat chronic disease; nevertheless, it is unclear if adding CHM to the routine management of T2D would modify sequent risk of hearing loss. This cohort-based case-control study was conducted to address this issue. First, a total of 64,418 subjects aged 20-70 years, diagnosed with T2D between 2002 and 2011, were extracted from a nationwide health claims database. Among them, we identified 4516 cases of hearing loss after T2D by the end of 2013. They were then randomly matched to 9032 controls without hearing loss at a 1:2 ratio. Following conditional logistic regression, we found the addition of CHM to conventional care reduced the risk of developing hearing loss, with an adjusted odds ratio of 0.75 (95% confidence interval: 0.70-0.83). Specifically, taking CHM products for at least two years benefits T2D patients in lowering sequent risk of hearing loss. The findings herein implicated that integrating CHM into conventional care substantially correlated to lower risk of hearing loss for T2D patients, but further basic research is needed to secure the application of finished herbal products.

01 Sep 2024·Journal of Traditional and Complementary Medicine

Chinese herbal medicine may reduce major adverse cardiovascular events in patients with dialysis hypotension: A taiwan nationwide cohort study

Article

Author: Lee, Wen-Chin ; Cheng, Ben-Chung ; Huang, Po-Yu ; Liu, Chun-Ting ; Tsai, Fuu-Jen ; Tsai, Ming-Yen

Background:

The association between Chinese herbal medicine (CHM) and the risk of developing major adverse cardiovascular events (MACEs) in patients with dialysis hypotension is unclear and has not yet been investigated. This study aimed to determine whether CMH intervention could reduce the risk of MACEs in patients with dialysis hypotension.

Methods:

The study data from the Taiwan National Health Insurance Research Database were analyzed to clarify this association. For this study, a case-control design with a cohort of patients who received hemodialysis (HD) from 2008 to 2018, 20 295 HD patients who had received blood pressure (BP) raising drugs were identified. After 1:1 frequency-matching, 730 patients were identified as CHM users and CHM non-users. Vascular access revision/reconstruction and MACEs were observed as the main outcomes during the follow-up period.

Results:

The occurrence of vascular access revision/reconstruction in HD patients receiving BP raising drugs was associated with a 0.34-fold lower risk in CHM users than in CHM non-users [adjusted hazard ratio (aHR) = 0.34, 95% confidence interval (CI) = 0.26, 0.45]. The occurrences of MACEs in HD patients receiving BP raising drugs was associated with a 0.41-fold lower risk in CHM users than in CHM non-users (aHR = 0.41, 95% CI = 0.33, 0.51). A markedly predominant effect was observed in those receiving CHM for more than 180 days (aHR = 0.32; 95% CI = 0.22, 0.45).

Conclusion:

The findings revealed lower vascular access dysfunction and MACEs risk correlated with the use of CHM treatment among HD patients who received BP raising drugs.

News (Medical) associated with SPK-7001

16 Nov 2022

·www.businesswire.com

Choroideremia Drug Pipeline Insights Report 2022 Featuring Spark Therapeutics, Molecular Therapeutics, Curative Biotechnology, & Ray Therapeutics - ResearchAndMarkets.com

DUBLIN--( BUSINESS WIRE )--The "Choroideremia - Pipeline Insight, 2022" report has been added to ResearchAndMarkets.com's offering. This report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Choroideremia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. The report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Choroideremia pipeline landscape is provided which includes the disease overview and Choroideremia treatment guidelines. The assessment part of the report embraces, in depth Choroideremia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Choroideremia collaborations, licensing, mergers and acquisition, funding, designations and other product related details. Report Highlights The companies and academics are working to assess challenges and seek opportunities that could influence R&D Choroideremia. The therapies under development are focused on novel approaches to treat/improve Choroideremia. In February 2019, Roche entered into a definitive merger agreement to acquire Spark Therapeutics and its clinical assets, including SPK-7001 for choroideremia in Phase I/II. 4D therapeutics is developing and investigating its lead product candidate 4D-110 in an ongoing Phase I dose-escalation clinical trial in patients with choroideremia related to mutations in the CHM gene. Choroideremia Emerging Drugs AAV2-hCHM: Spark Therapeutics Spark Therapeutics is advancing an open-label, dose-escalating Phase I/II trial designed to assess the safety and preliminary efficacy of subretinal administration of investigational SPK-7001. Each of the investigational research programs uses an adeno-associated viral (AAV) vector developed and manufactured by the Spark team and their collaborators. Choroideremia: Therapeutic Assessment This segment of the report provides insights about the Choroideremia drugs segregated based on following parameters that define the scope of the report, such as: Major Players in Choroideremia There are approx. 5+ key companies which are developing the therapies Choroideremia. The companies which have their Choroideremia drug candidates in the most advanced stage, i.e phase I/II include Spark Therapeutics Phases This report covers around 5+ products under different phases of clinical development like Late stage products (Phase III) Mid-stage products (Phase II) Early-stage product (Phase I) along with the details of Pre-clinical and Discovery stage candidates Discontinued & Inactive candidates Route of Administration Choroideremia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Molecule Type Products have been categorized under various Molecule types such as Small molecule Cell Therapy Peptides Polymer Small molecule Gene therapy Product Type Drugs have been categorized under various product types like Mono, Combination and Mono/Combination. Choroideremia: Pipeline Development Activities The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Choroideremia therapeutic drugs key players involved in developing key drugs. Pipeline Development Activities The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Choroideremia drugs. Choroideremia Report Insights Choroideremia Analysis Therapeutic Assessment Unmet Needs Impact of Drugs Choroideremia Report Assessment Pipeline Product Profiles Therapeutic Assessment Pipeline Assessment Inactive drugs assessment Unmet Needs Key Questions Current Treatment Scenario and Emerging Therapies: How many companies are developing Choroideremia drugs? How many Choroideremia drugs are developed by each company? How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Choroideremia? What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Choroideremia therapeutics? What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies? What are the clinical studies going on for Choroideremia and their status? What are the key designations that have been granted to the emerging drugs? Key Players Spark Therapeutics Molecular Therapeutics Curative Biotechnology Ray Therapeutics Key Products AAV2-hCHM 4D 110 Metformin RAY 001 For more information about this report visit https://www.researchandmarkets.com/r/suln3y

Phase 1Gene Therapy

03 Feb 2022

·www.biospace.com

Roche Abandons Crohn’s Program Following Mixed Topline Verdict

In Roche’s 2021 annual report, the company detailed that group sales rose 9%, pharmaceutical sales were up 3%, and diagnostics up a whopping 29%. The company noted that it had halted a Phase III development program for etrolizumab for Crohn’s disease, as well as a few others in the middle of all financial news. Roche's big approvals in the fourth quarter included Susvimo and Vabysmo for age-related blindness in the U.S.; in Europe, Gavreto for a specific form of advanced lung cancer, Actemra/RoActemra for severe COVID-19 and Ronapreve for non-hospitalized COVID-19 patients and prophylaxis. In the diagnostics arena, in the fourth quarter, the company noted approvals for its cobas 5800 molecular laboratory system, Avenio Edge sequencing sample preparation system, cobas pulse and cobas infinity edge digital ecosystem for clinical decision support and additional SARS-CoV-2 tests. The company expects sales for 2022 to be stable or to grow in the low-single digits. And in what is a hopeful sign for the world but not necessarily for Roche’s bottom line, the company said it expects sales of COVID-19-related drugs and diagnostics to fall by approximately CHF 2 billion ($2.17 billion) with sales drops for biosimilars of around CHF 2.5 billion ($2.7 billion). Otherwise, group sales are projected to grow in the high-single digit range. “We achieved good results in 2021,” stated Severin Schwan, Roche’s Chief Executive Officer. “The demand for our new medicines and diagnostics remains very high. I am particularly pleased with the progress of our product pipeline across several areas, including oncology, vision loss and neurological diseases. Based on our strong product portfolio in both divisions and the promising product pipeline, we are well-positioned for future growth.” In August 2020, Roche announced mixed topline results for its Phase III program of etrolizumab in patients with moderately to severely active ulcerative colitis, an inflammatory bowel disease similar to Crohn’s disease. The drug hit the primary endpoint of inducing remission compared to placebo in two of three studies, but the drug missed the primary endpoint versus placebo as maintenance therapy in ulcerative colitis. At the time, Dr. Levi Garraway, Roche’s chief medical officer and head of global product development, said, “We are disappointed with these results, because we know that people with ulcerative colitis need new treatment options. We are fully analyzing these data to learn more about how we might address the needs of people with this devastating disease. These studies were part of the largest clinical trial program ever undertaken in inflammatory bowel diseases, and we thank all the patients, investigators and healthcare professionals for their participations.” The studies in Crohn’s disease with and without previous anti-TNF treatment was continuing in the Phase III BERGAMOT trial and open-label extension and safety monitoring study (JUNIPER). Etrolizumab is a dual anti-integrin designed to selectively inhibit alpha4beta7 and alphaEbeta7 to control the trafficking of immune cells into the gut and their inflammatory effects on the lining of the gut. Whatever data on these studies Roche observed, it wasn’t strong enough to throw more time and money at. Roche also announced another Phase III program it was axing, RG6422, an oral COVID-19 antiviral it was developing with Atea Pharmaceuticals. In Phase II, Roche was halting SPK-7001, a choroideremia gene therapy it was developing with Spark Therapeutics, and RG7992 for nonalcoholic steatohepatitis (NASH), and RG7835 for autoimmune disorders.

Gene TherapyBiosimilar

09 Sep 2021

·www.globenewswire.com

Drug Development of Choroideremia Pipeline Report Analysis

Los Angeles, USA, Sept. 08, 2021 (GLOBE NEWSWIRE) -- Drug Development of Choroideremia Pipeline Report Analysis | DelveInsight The need for a new therapeutic option is acute. Clinical research is underway investigating the gene therapies for the effective treatment of Choroideremia. DelveInsight’s “Choroideremia Pipeline Insight” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in the Choroideremia pipeline landscapes. It comprises Choroideremia pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Choroideremia therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Choroideremia pipeline products. Some of the key takeaways from the Choroideremia Pipeline Report Get an overview of pipeline landscape @ Choroideremia Clinical Trials Analysis Choroideremia is a rare genetic disorder of vision that is more common in men. It is an X-linked recessive condition that gradually degrades the choroid, retinal pigment epithelium (RPE), and photoreceptors. Choroideremia Emerging Drugs NightstaRx Ltd initiated the development of BIIB111 for the treatment of Choroideremia. BIIB111 is a gene therapy-based treatment consisting of AAV2 vectors with recombinant human DNA designed to deliver REP1 protein to the retina. Spark Therapeutics is advancing an open-label, dose-escalating Phase I/II trial designed to assess the safety and preliminary efficacy of subretinal administration of investigational SPK-7001. Each of the investigational research programs uses an adeno-associated viral (AAV) vector developed and manufactured by the Spark team and their collaborators. For further information, refer to the detailed report @ Choroideremia Pipeline Therapeutics Scope of Choroideremia Pipeline Drug Insight · Choroideremia Therapies Late-stage (Phase III) · Choroideremia Therapies Mid-stage (Phase II)· Choroideremia Therapies Early-stage (Phase I) · Choroideremia Preclinical stage and Discovery candidates · Discontinued and Inactive candidates · Gene transference· CHM protein expression stimulants; Gene transference· Gluconeogenesis inhibitors· CHM protein expression stimulants; Gene transference · Peptides· Monoclonal antibodies· Small molecules · Polymer · Gene therapy · Parenteral · Intravitreal· Oral· Subretinal · Monotherapy· Combination· Mono/Combination Key Questions regarding Current Choroideremia Treatment Landscape and Emerging Therapies Answered in the Pipeline Report Table of Contents Get a customized pipeline report @ Choroideremia Drugs Pipeline Report Other Reports Browse Blog Posts About DelveInsightDelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also provides Healthcare Consulting services comprising credible market analysis that will help accelerate the business growth and overcome challenges with a practical approach.

Gene TherapyAntibodySmall molecular drug

100 Deals associated with SPK-7001

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Indication	Highest Phase	Country/Location	Organization	Date
Retinal Dystrophies	Phase 2	US	-	20 Jan 2015
Choroideremia	Phase 2	US	Spark Therapeutics, Inc.	15 Jan 2015
Retinal Dysplasia	Phase 2	-	The Children's Hospital of Philadelphia	-
Retinal Dysplasia	Phase 2	-	Spark Therapeutics, Inc.	-
Retinal Dysplasia	Phase 2	-	University of Pennsylvania	-

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02341807 (CTgov)	Phase 1/2	Choroideremia	15	AAV2-hCHM (Cohort 1: AAV2-hCHM Dose 1)	eohzewflie(gnxogzibbv) = yejdempouj bxglcuvfik (rdmpuicuns, huwgguunpr - aznkbhjdcc) View more	-	02 Nov 2023
NCT02341807 (CTgov)	Phase 1/2	Choroideremia	15	AAV2-hCHM (Cohort 2: AAV2-hCHM Dose 2)	eohzewflie(gnxogzibbv) = vqsvcaihwd bxglcuvfik (rdmpuicuns, ebvaavsnku - raocfmnxrd) View more	-	02 Nov 2023
Phase I/II trial of AAV2-hCHM (ARVO2019)	Phase 1/2	Choroideremia CHM variants	5	AAV2-hCHM	vugpmgyqng(caouozysjv) = in one patient in whom a macular hole developed intraoperatively resulting in a SAE due to persistent vision loss rsuxugkauo (tppyvgbmwd )	-	01 Jul 2019

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