更新于：2024-06-29

Vamorolone

更新于：2024-06-29

概要

研发状态

概要

基本信息

药物类型

小分子化药

别名

Vamorolone (USAN/INN)、8XP29XMB43、VB-15

+ [2]

靶点

作用机制

GR激动剂(糖皮质激素受体激动剂)

治疗领域

神经系统疾病遗传病与畸形皮肤和肌肉骨骼疾病+ [1]

在研适应症

杜氏肌营养不良症贝克型肌营养不良

非在研适应症

哮喘囊性纤维化炎症性肠病+ [3]

原研机构

ReveraGen BioPharma, Inc.Santhera Pharmaceuticals (USA), Inc.

在研机构

Santhera Pharmaceuticals Holding AG

Purna Pharmaceuticals NV

Catalyst Pharmaceuticals, Inc.

+ [3]

非在研机构

University of Pittsburgh

Newcastle University

最高研发阶段批准上市

首次获批日期

美国 (2023-10-26),

杜氏肌营养不良症

最高研发阶段(中国)申请上市

特殊审评快速通道 (美国)、孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、优先审评 (中国)、突破性疗法 (中国)、儿科研究计划 (欧盟)、潜力创新药 (英国)

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结构

分子式C22H28O4

InChIKeyZYTXTXAMMDTYDQ-DGEXFFLYSA-N

CAS号13209-41-1

关联

项与 Vamorolone 相关的临床试验

ChiCTR2300078541 / Recruiting临床1期IIT

A Randomized, Cross-over, Open-label Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Vamorolone Oral Suspension Given as A Single Dose in Healthy Chinese Adult Male Subjects

开始日期2023-12-12

申办/合作机构-

CTR20232865 / Completed临床1期

一项随机、交叉、开放标签，评价Vamorolone口服混悬液在中国健康成年男性受试者中单次给药后的药代动力学和安全性耐受性的研究

评价中国健康成年男性受试者在空腹/餐后状态下单次口服VBP15口服混悬液的安全性、耐受性和药代动力学特征。

开始日期2023-12-01

申办/合作机构

曙方（上海）医药科技有限公司

[+2]

NCT05166109 / Recruiting临床2期

A Phase II Pilot Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy

This Phase II pilot study is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of vamorolone 500mg (250mg for body weight <50 kg) daily administered orally compared to placebo over a treatment period of 24 weeks in males with BMD.
Funding Source - FDA OOPD

开始日期2022-07-07

申办/合作机构

ReveraGen BioPharma, Inc.

[+1]

100 项与 Vamorolone 相关的临床结果

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100 项与 Vamorolone 相关的转化医学

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100 项与 Vamorolone 相关的专利（医药）

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项与 Vamorolone 相关的文献（医药）

2024-01-20·Human molecular genetics

Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models

Article

作者: Braughton, Bruce ; Yeh, Shirley ; Narasimhan, Jana ; Liu, Grace ; Jung, Stephen ; Weetall, Marla ; Lennox, William ; Yurkow, Edward ; Voronin, Gregory ; Rice, Cari ; Li, Wencheng ; More, Vijay ; Huarte, Eduardo ; Moon, Young-Choon ; Lipari, Philip ; Gruszka, Barbara ; Mollin, Anna ; Buckendahl, Patricia ; Teplova, Irina ; Ying, Lanqing ; Welch, Ellen ; Ray, Balmiki ; Goodwin, Elizabeth ; McGann, Eric

Abstract:

Duchenne muscular dystrophy (DMD) is a progressive disabling X-linked recessive disorder that causes gradual and irreversible loss of muscle, resulting in early death. The corticosteroids prednisone/prednisolone and deflazacort are used to treat DMD as the standard of care; however, only deflazacort is FDA approved for DMD. The novel atypical corticosteroid vamorolone is being investigated for treatment of DMD. We compared the pharmaceutical properties as well as the efficacy and safety of the three corticosteroids across multiple doses in the B10-mdx DMD mouse model. Pharmacokinetic studies in the mouse and evaluation of p-glycoprotein (P-gP) efflux in a cellular system demonstrated that vamorolone is not a strong P-gp substrate resulting in measurable central nervous system (CNS) exposure in the mouse. In contrast, deflazacort and prednisolone are strong P-gp substrates. All three corticosteroids showed efficacy, but also side effects at efficacious doses. After dosing mdx mice for two weeks, all three corticosteroids induced changes in gene expression in the liver and the muscle, but prednisolone and vamorolone induced more changes in the brain than did deflazacort. Both prednisolone and vamorolone induced depression-like behavior. All three corticosteroids reduced endogenous corticosterone levels, increased glucose levels, and reduced osteocalcin levels. Using micro-computed tomography, femur bone density was decreased, reaching significance with prednisolone. The results of these studies indicate that efficacious doses of vamorolone, are associated with similar side effects as seen with other corticosteroids. Further, because vamorolone is not a strong P-gp substrate, vamorolone distributes into the CNS increasing the potential CNS side-effects.

2024-03-05·Journal of neuromuscular diseases

The Early Care (0–3 Years) In Duchenne Muscular Dystrophy Meeting Report

作者: Berggren, Kiera N ; Mathews, Katherine ; Braun, Catherine ; Scharf, Rebecca J ; Parad, Richard ; Connolly, Anne ; McGuire, Michelle ; Scavina, Mena ; Ciafaloni, Emma ; Waldrop, Megan ; Armstrong, Niki ; Apkon, Susan ; Kennedy, Annie ; Kuntz, Nancy

Objective: This report summarizes the key discussions from the “Early Care (0–3 years) in Duchenne Muscular Dystrophy” meeting, which aimed to address the challenges and opportunities in the diagnosis and care of Duchenne muscular dystrophy (DMD) and female carriers within the 0–3-year age group. Methods: The meeting brought together experts and healthcare providers who shared insights, discussed advancements in DMD care, and identified research needs. Presentations covered diagnostic challenges, approved therapies, clinical trials, identification of young female carriers, and the importance of clinical care and support for families. Results: The meeting highlighted the importance of timely diagnosis and the lack of evidence-based guidelines for the care of children with DMD aged 0–3 years. Diagnostic challenges were discussed, including delays in receiving a DMD diagnosis and disparities based on ethnicity. The potential benefits and process of newborn screening were addressed. Approved therapeutic interventions, such as corticosteroids and exon-skipping drugs, were explored, with studies indicating the potential benefits of early initiation of corticosteroid therapy and the safety of exon-skipping drugs in DMD. Clinical trials involving infants and young boys were discussed, focusing on drugs like ataluren, vamorolone, and gene therapies. The meeting emphasized the importance of clinical care and support for families, including comprehensive information provision, early intervention services, and individualized support. The identification and care of young female carriers were also addressed. Conclusion: The meeting provided a platform for experts and healthcare providers to discuss and identify key aspects of early care for children with DMD aged 0–3 years. The meeting emphasized the need for early diagnosis, evidence-based guidelines, and comprehensive care and support for affected children and their families. Further research, collaboration, and the development of consensus guidelines are needed to improve early diagnosis, treatment, and outcomes in this population.

2024-01-01·Drugs

Vamorolone: First Approval

Review

作者: Keam, Susan J

Vamorolone (AGAMREE^®) is an oral, selective, dissociative corticosteroid developed by ReveraGen BioPharma and Santhera Pharmaceuticals for the treatment of patients with muscular dystrophy. Vamorolone was approved in October 2023 for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older in the USA and received a positive opinion in the EU in October 2023 for the treatment of DMD in patients 4 years of age and older. This article summarizes the milestones in the development of vamorolone leading to this first approval for DMD.

167

项与 Vamorolone 相关的新闻（医药）

2024-06-24

·PRNewswire

Parent Project Muscular Dystrophy Hosts 30th Annual Conference in Orlando, Florida

Milestone event brings together record-breaking 1,300 plus families, physicians, researchers, caregivers, industry partners, and those living with Duchenne and Becker in person and virtually to connect, share information, and learn the latest progress in the fight for every future. ORLANDO, Fla., June 24, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will host its 30th Annual Conference in Orlando, Florida, June 27–29. Each year, the Duchenne and Becker community gathers to learn about the latest progress in the fight to end Duchenne and connect with leading experts. Now in its 30th year, PPMD's Annual Conference returns to Orlando, the location of the organization's very first Annual Conference in 1994, to engage more of the Duchenne and Becker community than ever before. PPMD expects a record-breaking 1,300 plus families, researchers, clinicians, and industry partners from all over the world for three days of informative and interactive sessions featuring the latest news in research, clinical trials, approved and emerging therapies, care initiatives, quality of life issues, and more. Before the Conference officially kicks off, PPMD will host a Becker Care Professional Meeting, June 26–27, aimed at initiating discussions within the healthcare professional community regarding the creation of consensus-based standards of care for Becker muscular dystrophy. Founded in 1994 by a group of parents and grandparents, and led by Founding President and CEO Pat Furlong, PPMD is the largest, most comprehensive nonprofit organization in the United States focused on ending Duchenne. Since its first meeting 30 years ago, PPMD has convened families with researchers, clinicians, industry partners, advocacy organizations, and most importantly, people with Duchenne and Becker and their families at the largest Duchenne-specific conference held in the U.S. Furlong is excited about this year's milestone Conference and looks forward to bringing the Duchenne and Becker community together: "From PPMD's first Annual Conference 30 years ago to our momentous return to Orlando this week, this community's unwavering commitment, passion, and resilience have been the driving force behind our fight for every future. We have faced challenges and celebrated victories and we have motivation to keep progressing. PPMD's 30th Annual Conference will be filled with a variety of interactive experiences to spark conversation, foster new and familiar relationships, learn from one another, and continue to drive change in the fight to end Duchenne." PPMD is grateful to all of our industry sponsors, whose support helps make the Annual Conference possible, especially our 30th Anniversary Star sponsor Sarepta Therapeutics, our Platinum sponsors Catalyst Pharmaceuticals and Pfizer, Inc., and our Gold sponsors Avidity Biosciences, Dyne Therapeutics, Edgewise Therapeutics, ITF Therapeutics, NS Pharma, Inc., Upsher-Smith Laboratories, and Wave Life Sciences. For sponsorship opportunities, please click here. PPMD's 30th Annual Conference agenda will feature stories from multiple keynote speakers, and interactive discussion panels addressing comprehensive, multi-dimensional approaches to care for Duchenne and Becker. Unique topics to this year's conference include: Approved therapies in Duchenne, including presentations on the recently expanded label for ELEVIDYS and newly-approved therapies AGAMREE and DUVYZAT, as well as a clinician panel discussion on the eight currently approved therapies. Becker-specific breakout sessions, including discussions on neuromuscular care, cardiology, genetic counseling, and physical therapy, as well as industry updates about emerging therapies for Becker. What we have learned over time about outcome measures, including Functional Reach, Stride Velocity, Dynamic Visual Acuity (DVA), and Northstar and Performance of Upper Limb (PUL). Cardiac, neuromuscular, and adolescent care for carriers, as well as physical therapy implications and research in progress in the field of Duchenne carriers. Unique experiences and perspectives on life, living, and hope, as well as daily life care such as sleep and behavior; decision making around steroids, weight, and nutrition; pain; exercise; social connection; and more. This year's Conference will also feature dedicated updates from more than 20 partners currently in clinical trials, with panels including: Strategies to restore dystrophin The current and evolving landscape of gene therapy Approaches to alter disease progression PPMD's role in moving the needle in clinical research A variety of breakout sessions and social gatherings will also be offered during PPMD's 30th Annual Conference, encouraging attendees to create their own, unique experiences, in-person or virtually, by participating in: Special programming for Newly Diagnosed Families A dedicated track for Teens and Adults with Duchenne/Becker Expanded Sibs Track sessions, offering a safe place for siblings to be together, talk, and open up about topics that they face Breakout sessions according to age, covering multiple aspects of care One-on-one private appointments with Physical Therapists, Genetic Counselors, and behavior, speech, and language experts to help provide guidance on every family's unique needs Small group Lighthouse Mini-Sessions focused on self-care, effective communication, asking for help, and staying connected and finding belonging A Build Your Own Care Binder station for families to create personalized binders filled with important information regarding Duchenne/Becker care to help navigate routine care and emergencies A robust Resource Fair enabling resource providers and the community to connect about practical services, research and more A meet and greet for diverse families in Duchenne to share their unique experiences and foster connection Social activities for moms, dads, grandparents, siblings, and, of course, tweens, teens, and adults with Duchenne and Becker Fun and interactive sessions for kids Ahead of the Conference, all registered attendees were given access to PPMD's online Conference Hub & App to stream sessions and personalize their agenda and Conference experience. This year's Hub & App offers an enhanced experience for both in-person and virtual attendees, allowing for engagement with exhibitors and increased interaction during live sessions. AI-powered live translation will also be available for main sessions in dozens of languages. It is not too late to attend this year's meeting, in-person or virtually. To register for PPMD's 30th Annual Conference, click here. To learn more about PPMD, please visit our website. SOURCE Parent Project Muscular Dystrophy (PPMD)

2024-06-21

·Science Daily

Lab-grown muscles reveal mysteries of rare muscle diseases

Biomedical engineers have grown muscles in a lab to better understand and test treatments for a group of extremely rare muscle disorders called dysferlinopathy or limb girdle muscular dystrophies 2B (LGMD2B). The research revealed the biological mechanisms underlying the disease and showed that a combination of existing treatments could alleviate its symptoms. Biomedical engineers at Duke University have developed a new technique to better understand and test treatments for a group of extremely rare muscle disorders called dysferlinopathy or limb girdle muscular dystrophies 2B (LGMD2B). The approach grows complex, functional 3D muscle tissue from stem cells in the laboratory, creating a platform that replicates patient symptoms and treatment responses. In its debut study, researchers reveal some of the biological mechanisms underlying the characteristic loss of mobility caused by LGMD2B. They also demonstrate that a combination of existing treatments may be able to alleviate some of the worst symptoms of the disease. The results appear online June 18 in the journal Advanced Science. LGMD2B affects only about eight people per million around the world. Unlike it's more well-known and more common cousin Duchenne muscular dystrophy, the disease affects both men and women, presents later in life -- in the late teens or early 20s -- and is rarely fatal. However, LGMD2B patients develop severe weakness in the legs and shoulders, typically requiring them to use wheelchairs for the rest of their lives. Caused by a genetic disorder, LGMD2B stops the body from creating a fully functional form of a protein called dysferlin. There are currently no approved treatments or cures. Part of the reason for this lack of options is the many functions of dysferlin, including sealing holes in muscle membranes, regulating calcium balances required for muscle contraction and controlling cellular metabolism. And for reasons unknown, affected muscles initially accumulate fat within the muscle fibers themselves before degenerating and ultimately being replaced by fat cells. "This phenomenon is very rare even for muscular dystrophies," said Nenad Bursac, professor of biomedical engineering at Duke. "It's a burning question within the community of why that happens." One of the challenges facing researchers trying to solve these problems is that the mouse model used to approximate LGMD2B shows very mild symptoms compared to patients. Mice with the disease are still able to walk, and it does not present until nearly a year into their two-year long lifespan, making studies of the disease extremely slow. Dysferlin is expressed in other cell types, and blood levels of metabolic fuels such as cholesterol are also altered in mice and patients. Together, this complicates LGMD2B research by making it difficult to assess what cell types are responsible for the disease and if metabolic changes are due to the loss of dysferlin itself or whole-body effects. To get around these challenges, Bursac and his research scientist Alastair Khodabukus turned to an engineered muscle platform that they have been developing for nearly a decade. The Bursac Lab was the first to grow contracting, functional human skeletal muscle in a Petri dish and has been improving its processes ever since to enable studies of muscle strength, metabolism and repair. This system allowed them to focus their study on only the effects of dysferlin on skeletal muscle without the complications of other cell types or altered blood metabolite pro In the study, the researchers began with induced pluripotent stem cells (IPSCs) derived from patients living with LGMD supplied by The Jain Foundation, a charity focused on finding a cure for LGMD2B. Using their muscle-growing techniques, the lab matured these stem cells into muscle fibers and ran them through a battery of tests over the course of six weeks. Like muscles found in patients themselves, the lab-grown versions displayed a wide variety of issues. "Overall, our model repeated a lot of the clinical manifestations of the disease and observations made in real patients, but it was all done in a Petri dish," said Bursac. "We've been able to gain new insights into the muscle-specific aspects of the disease." The researchers discovered that the loss in muscle strength was not the result of deficiencies in muscle structure or size, but in its handling of calcium. Muscle contractions are physically caused by reservoirs of calcium in muscle cells being released and binding to muscle proteins. Tests showed that the diseased muscle cells had sprung leaks in their calcium reserves, resulting in less calcium to release and weaker contractions. The researchers also showed that the lack of dysferlin caused damage to the muscle cells to go unrepaired, and that an inability to burn fatty acids for energy production was at least partially to blame for the accumulation of fat within muscle fibers that has long puzzled the medical community. "We replicated something seen in patients and showed that it's not due to environmental factors within the body, but issues within the muscle itself," Khodabukus said. The researchers then tested the effects of two drug candidates to potentially treat the disease that have been identified through mouse models but have not yet been tested in humans. One called dantrolene is supposed to stop calcium from leaking from muscle cells' reservoirs. The second, called vamorolone, was recently approved for use with Duchenne muscular dystrophy patients, although researchers do not fully understand how it works. Together, the drugs prevented the calcium leak and helped the cell membrane repair itself, restoring much of the muscles' strength. And while they also helped reduce the amount of fat accumulated within the muscles, they did not fully prevent it, nor did they help the muscles efficiently burn fats for fuel. Moving forward, the group is planning to add immune and fat cells to their experiments for greater complexity. They also are looking to further understand disruptions to cellular metabolism and to find new drugs that fully restore all strength, repair and metabolic deficit. "Right now, we have a basic muscle-only system, which is great to see the effects of the disease within the muscle itself, but what is also important is the crosstalk between immune cells, fat cells and muscle cells," Khodabukus said. "By building out our system more, we'll hopefully be able to fully figure out what is driving the loss of muscle and its replacement by fat in these patients." This research was supported by the National Institutes of Health (UG3TR002142, U01EB028901, R01AR070543, R01AR079223, R01ARO82979, R21AR078269, UH3TR002142) and the Jain Foundation.

上市批准

2024-06-18

·GlobeNewswire-Health Care

Santhera’s Shareholders Approve all Board Proposals at Today’s Annual General Meeting

Pratteln, Switzerland, June 18, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that its shareholders approved all motions by the Board of Directors at the Annual General Meeting (AGM) held today in Pratteln, Switzerland, with a large majority. Participating shareholders represented a total of 4,535,785 shares or 35.9% of the Company’s share capital. Approval of the 2023 Annual Report and discharge of corporate bodiesThe shareholders approved the Annual Report, the annual financial statements and the consolidated financial statements for 2023, as well as the appropriation of the annual result and its offset against free reserves. Furthermore, the shareholders granted discharge to the members of the Board of Directors and Executive Management for the financial year 2023. Approval of amendments to the Articles of IncorporationIn four separate votes, the shareholders approved various amendments to the Articles of Incorporation to bring them in line with the revised Swiss corporate law, which came into force on January 1, 2023. The revision of corporate law aimed, among other things, to improve corporate governance, to modernize corporate law and to integrate the rules on remuneration for listed companies in the statute. Members of the Board of Directors and the Compensation Committee re-electedShareholders re-elected Thomas Meier, PhD, Philipp Gutzwiller, Bradley C. Meyer and Otto Schwarz, PhD, to serve another one-year term on the Board of Directors. In addition, shareholders re-elected Thomas Meier, PhD, as Chairman of the Board of Directors. As members of the Compensation Committee, the shareholders confirmed Thomas Meier, PhD, and Bradley C. Meyer (Committee Chairman). Compensation Report and remuneration for Board and Executive Management approvedIn a consultative vote, the shareholders endorsed the Compensation Report 2023. In separate binding votes, Santhera’s shareholders agreed to the compensation for members of the Board of Directors (maximum total amount of fixed compensation covering the period from the 2024 AGM to the 2025 AGM) and for the members of the Executive Management (maximum total amount of fixed compensation for 2025, variable cash compensation for 2023 and variable compensation in the form of Share Appreciation Rights and Performance Share Units for 2024). AGM documentsDetails and accompanying explanations on all motions, which were all approved by the AGM, can be viewed in the “Invitation to the Annual General Meeting of Shareholders”, available on Santhera’s website at https://www.santhera.com/share-bondholder-meetings. From the end of next week, the minutes of the 2024 AGM will also be available. About SantheraSanthera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals, Inc. and for China to Sperogenix Therapeutics. For further information, please visit www.santhera.com. AGAMREE® is a trademark of Santhera Pharmaceuticals. For further information please contact: public-relations@santhera.com orEva Kalias, Head Investor Relations & CommunicationsPhone: +41 79 875 27 80eva.kalias@santhera.com Disclaimer / Forward-looking statements This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements. # # # Attachment 2024 06 18_AGM-resolutions_e_final

上市批准引进/卖出高管变更

100 项与 Vamorolone 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11000	-	-	DB15114

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
杜氏肌营养不良症	美国	Catalyst Pharmaceuticals, Inc.	2023-10-26

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
小儿溃疡性结肠炎	临床2期	-	ReveraGen BioPharma, Inc.	2020-09-01
贝克型肌营养不良	临床1期	瑞士	Santhera Pharmaceuticals Holding AG	2022-01-30
囊性纤维化	临床前	美国	ReveraGen BioPharma, Inc.	2016-05-25
多发性硬化症	临床前	美国	ReveraGen BioPharma, Inc.	2016-05-25
炎症性肠病	临床前	美国	ReveraGen BioPharma, Inc.	2016-05-04
类风湿关节炎	临床前	美国	ReveraGen BioPharma, Inc.	2016-04-22
哮喘	临床前	美国	ReveraGen BioPharma, Inc.	2016-04-10

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03439670 (VISION-DMD, MDA2024)	临床2期	杜氏肌营养不良症	118	Vamorolone 2 mg/kg/day	膚醖獵襯衊壓襯築夢蓋(鹹構築夢衊壓選膚窪繭) = 鏇蓋壓遞糧膚繭齋築衊製壓糧蓋廠構鏇窪廠構 (蓋鹹鹽淵鹽餘餘鬱獵衊 )	不佳	2024-03-03
				Prednisone 0.75 mg/kg/day	膚醖獵襯衊壓襯築夢蓋(鹹構築夢衊壓選膚窪繭) = 顧糧簾鑰網膚獵壓鬱築製壓糧蓋廠構鏇窪廠構 (蓋鹹鹽淵鹽餘餘鬱獵衊 )
NCT03439670 (FDA) 人工标引	临床2期	杜氏肌营养不良症	90	Placebo	願蓋膚廠獵簾夢鹹範鹽(鏇網鏇遞夢觸廠鑰網繭) = 構餘築鹽觸壓艱壓網襯鏇選積衊鹹膚積願願廠 (衊簾鏇襯鑰遞艱壓鏇鹹 ) 更多	积极	2023-10-26
				AGAMREE 2 mg/kg/d	願蓋膚廠獵簾夢鹹範鹽(鏇網鏇遞夢觸廠鑰網繭) = 鬱壓簾膚膚壓夢襯積襯鏇選積衊鹹膚積願願廠 (衊簾鏇襯鑰遞艱壓鏇鹹 ) 更多
NCT03439670 (Pubmed)	临床2期	杜氏肌营养不良症	133	Vamorolone 2 mg/kg per day	積蓋鏇範願憲齋衊繭糧(餘壓觸糧鑰鹹積簾廠鏇) = Bone turnover markers declined with prednisone but not with vamorolone 構鏇繭範遞選觸築窪選 (餘範糧襯齋積鏇願壓鹹 ) 更多	积极	2022-08-29
NCT03439670 (VISION-DMD \| VBP15-004, CTgov)	临床2期	杜氏肌营养不良症	121	Vamorolone (Treatment Group 1)	製淵範鑰鏇範選選獵鬱(餘艱網蓋艱廠艱艱鬱構) = 築齋築範淵築築襯糧獵製衊糧鏇鹹憲獵積簾選 (製蓋糧築築餘範範網醖, 鏇築願獵繭觸鬱顧簾築 ~ 獵遞繭艱顧齋鑰簾鑰選) 更多	-	2022-07-13
				Vamorolone (Treatment Group 2)	製淵範鑰鏇範選選獵鬱(餘艱網蓋艱廠艱艱鬱構) = 蓋襯衊網膚選觸窪衊憲製衊糧鏇鹹憲獵積簾選 (製蓋糧築築餘範範網醖, 築遞顧窪鏇願襯築鹹鑰 ~ 範醖築鬱獵鬱遞鹹製積) 更多
VBP15-004 (MDA2022)	临床2期	杜氏肌营养不良症	121	Vamorolone 60 mg/kg/day	淵築觸鏇淵簾壓夢窪夢(繭觸衊艱鏇觸齋淵築簾) = 廠繭範餘壓構獵醖醖蓋網遞構鬱鑰餘壓遞壓蓋 (構顧鬱鑰範繭蓋窪夢選 )	积极	2022-03-13
NCT03038399 (Pubmed \| JAMA Netw Open)	临床2期	杜氏肌营养不良症	46	Vamorolone	構衊遞蓋觸願構鹹襯鏇(鹽壓壓鏇鏇觸築願繭衊) = 積襯窪廠鏇積淵選範鏇鏇艱廠鹽憲夢餘餘範顧 (壓鑰夢鹽築醖築構範糧, -4.48 ~ 4.04) 更多	积极	2022-01-04
NCT03038399 (VBP15-LTE, CTgov)	临床2期	杜氏肌营养不良症	46	Vamorolone 0.25 mg/day/day (Dose Level Group 1)	願鹹網壓製構鬱餘襯淵(製遞襯餘遞選壓製膚遞) = 窪製齋餘選醖構鹹鏇醖襯範襯顧製鑰繭鹹築膚 (積鏇淵淵糧構遞構夢鹹, 膚壓鏇獵積淵窪齋製廠 ~ 積網蓋衊獵淵蓋網鑰繭) 更多	-	2021-05-20
				Vamorolone 0.75 mg/day/day (Dose Level Group 2)	願鹹網壓製構鬱餘襯淵(製遞襯餘遞選壓製膚遞) = 憲廠構積窪衊壓衊觸膚襯範襯顧製鑰繭鹹築膚 (積鏇淵淵糧構遞構夢鹹, 願醖鬱壓鹹夢顧願糧製 ~ 窪鑰鏇築醖壓淵範遞夢) 更多
NCT03038399 (VBP15-LTE, Pubmed \| PLoS Med) 人工标引	临床2期	杜氏肌营养不良症	65	vamorolone	範夢廠積選築遞衊膚餘(齋憲醖鏇繭構簾鏇蓋襯): P-Value = 0.001 更多	积极	2020-09-21
				group-matched participants
NCT02760277 (Pubmed \| Neurology) 人工标引	临床2期	杜氏肌营养不良症	48	Vamorolone	鬱範觸觸遞壓鏇糧壓簾(範鹽遞遞鬱艱夢願艱遞) = improved (2.0-mg/kg/d dose group) 鹽鑰夢餘鏇餘醖壓構襯 (齋鑰襯糧襯廠齋鬱鹹範 )	积极	2019-09-24
NCT02760277 (CTgov)	临床2期	杜氏肌营养不良症	48	Vamorolone 0.25 mg/day/day (Dose Level Group 1)	繭鹹積獵醖糧醖夢鬱簾(範鹽膚憲簾窪糧窪範鏇) = 憲鬱鏇醖壓遞餘醖襯齋壓淵艱獵積觸淵夢艱壓 (製夢衊憲觸鏇鏇襯顧艱, 積製築糧構艱鏇鑰夢製 ~ 壓艱築構製鑰積衊襯遞) 更多	-	2019-07-23
				Vamorolone 0.75 mg/day/day (Dose Level Group 2)	繭鹹積獵醖糧醖夢鬱簾(範鹽膚憲簾窪糧窪範鏇) = 網蓋窪鹽鹹齋鹽積鬱壓壓淵艱獵積觸淵夢艱壓 (製夢衊憲觸鏇鏇襯顧艱, 夢夢艱顧鏇鏇構網顧膚 ~ 構獵鹹獵繭顧觸鹽餘鹽) 更多