CTIS2022-500215-39-00进行中（未招募）1 期
A Phase 2 Randomized, Double-blind, Placebo-controlled Study of the Effect of EDG-5506 on Biomarker Response to Exercise in Adults with Becker Muscular Dystrophy, McArdle Disease, or Limb-Girdle Muscular Dystrophy - EDG-5506-202
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 21
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 65+（—）
- 性别
- All
入选标准
- •1. Participants with BMD or LGMD2I must be 18 to 65 years of age inclusive, at the time of signing the informed consent. Participants with McArdle disease may enroll up to 75 years of age inclusive, at the time of signing the informed consent form., 2. Participant has a molecular diagnosis of BMD (a documented dystrophin mutation and phenotype consistent with BMD, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids), McArdle disease, or LGMD2I with a consistent clinical phenotype., 3. Participant is able to comply with the cycle exercise protocol (in the opinion of the Principal Investigator)., 4. Participant has no concurrent medical condition that would, in the opinion of the Investigator, impair the ability to give informed consent or undertake any of the protocol-mandated visits/assessments., 5. Participant has no active muscle injury at Screening., 6. Participant is willing and able to comply with all protocol requirements., 7. Participant is able to converse and read fluently in Danish or English., 8. Participant is willing to comply with contraception requirements described in protocol., 9. Capable of giving signed informed consent as described in the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
排除标准
- •1. Medical history, clinically significant laboratory, or physical examination finding that, in the opinion of the Investigator, would render the participant unsuitable for the study. This includes anything that would prevent completion of the cycle ergometer test., 6. Moderate or severe renal impairment (estimated glomerular filtration rate [eGFR] <60 mL/min/1.73 m2 ). eGFR will be based on Cystatin C formula (eGFR = 133 x min(Scys/0.8, 1)^-0.499 x max (Scys/0.8, 1)^-1.328 X 0.996^Age x 0.932 [if female]., 7. Positive test for hepatitis C antibody (unless negative hepatitis C virus polymerase chain reaction), hepatitis B surface antigen, or human immunodeficiency virus antibody., 8. Receipt of oral corticosteroids for >10 days in the previous 6 months. Inhaled/intranasal steroids are permitted., 09. Current use of any strong or moderate cytochrome P450 (CYP)3A4 inhibitors or inducers., 10. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior, recent (within the past year) history of substance abuse or dependency or laboratory results or abnormality that may increase the risk of study participation or, in the Investigator’s judgment, make the participant inappropriate for the study., 11. Participation in a previous exercise challenge study if the challenge itself has taken place less than 10 weeks prior to screening., 12. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of dosing in the present study., 2. Left ventricular ejection fraction <45% or New York Heart Association (NYHA) Class III or Class IV, determined by local read., 3. 12-lead electrocardiogram (ECG) demonstrating clinically relevant abnormalities that may affect participant safety or interpretation of study results, determined by local read., 4. Forced vital capacity (FVC) predicted <60% or using daytime (mechanical or noninvasive) ventilatory support., 5. Moderate or severe hepatic impairment
研究者
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