NCT04535609已完成2 期
A Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of 24 Weeks Treatment With REN001 in Patients With Primary Mitochondrial Myopathy
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 213
- 试验地点
- 41
- 主要终点
- Change in Distance Walked During a 12 Minute Walk Test
研究概览
简要总结
This is a randomized, double-blind, placebo-controlled, parallel group, multi-centre, study designed to investigate the efficacy and safety of REN001 administered once daily over a 24-week period to patients with PMM.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects age 18 years or older with PMM as defined by the International Workshop: Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adult (Mancuso et al 2017).
- •A confirmed PMM diagnosis due to known pathogenic gene mutation or deletion of the mitochondrial genome. The Sponsor may authorize local genetic testing at Screening, if required, but results must be available prior to randomization of the subject.
- •Documented PMM primarily characterized by exercise intolerance or active muscle pain.
- •Subjects must be ambulatory and able to perform the walking tests independently (walking aids are allowed).
- •Have no changes to any therapeutic exercise regimen within 30 days prior to Day 1 and be willing to remain on the same therapeutic exercise regimen for the duration of the study.
- •Females should be either of non-child-bearing potential or must agree to use highly effective methods of contraception from Screening through to 30 days after last dose in the study. Males with partners who are WOCBP must also use contraception.
- •Concomitant medications (including supplements) must be stable for at least 1 month prior to enrolment and throughout participation in the study.
- •Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
- •Participation in a prior REN001 (previously known as HPP-593) study.
- •Currently taking or anticipated to need a PPAR agonist during the study.
- •Subjects with bone deformities or motor abnormalities other than related to the mitochondrial myopathy that may interfere with the outcome measures.
- •Clinically significant kidney disease or impairment calculated as eGFR Grade 2 or above <60ml/min/1.73m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation at Screening.
- •Clinically significant liver disease or impairment of AST or ALT Grade 2 or above (>2.5 x ULN), or Total bilirubin > 1.6 x ULN or >ULN with other signs and symptoms of hepatotoxicity at Screening.
- •Subjects with uncontrolled diabetes and/or a Screening HbA1c of ≥11%.
- •Evidence of significant concomitant clinical disease that may need a change in management during the study or could interfere with the conduct or safety of this study. (Stable well-controlled chronic conditions such hypercholesterolemia, gastroesophageal reflux, or depression under control with medication (other than tricyclic antidepressants), are acceptable provided the symptoms and medications would not be predicted to compromise safety or interfere with the tests and interpretations of this study.)
- •Subjects with a history of cancer. A history of in situ basal cell carcinoma in the skin is allowed.
- •Clinically significant cardiac disease and/or clinically significant ECG abnormalities such as 2nd degree heart block, symptomatic tachyarrhythmia or unstable arrythmia (right bundle branch block, left fascicular block and long PR interval are not excluded) that in the opinion of the Investigator should exclude the subject from completing exercise tests.
- •Evidence of hospitalization for rhabdomyolysis within the year prior to enrolment.
- •Pregnant or nursing females.
- •History of sensitivity to PPAR agonists.
排除标准
- 未提供
研究组 & 干预措施
Mavodelpar
Experimental
Once daily
干预措施: Mavodelpar (Drug)
Matched placebo
Placebo Comparator
Once daily
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Distance Walked During a 12 Minute Walk Test
时间窗: Baseline to Week 24
Distance walked in meters
次要结局
- Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores(Baseline to Week 24)
研究者
研究点 (41)
Loading locations...
相似试验
已完成
2 期
A Study to Evaluate the Safety of 12 Weeks of Dosing With GW856553 and Its Effects on Inflammatory Markers, Infarct Size, and Cardiac Function in Subjects With Myocardial Infarction Without ST-segment ElevationAcute Coronary SyndromeNCT00910962GlaxoSmithKline526
终止
3 期
A Multicentre Study to Evaluate the Efficacy and Safety of ENIA11 in Patients With Ankylosing SpondylitisAnkylosing SpondylitisNCT02685904Mycenax Biotech Inc.10
尚未招募
3 期
A Study to Investigate Arbaclofen ER Tablets for the Treatment of Spasticity in Patients With Multiple SclerosisSpasticityNCT05179577RVL Pharmaceuticals, Inc.442
已完成
3 期
Efficacy and Safety Study of Benralizumab in Patients With Uncontrolled Asthma on Medium to High Dose Inhaled Corticosteroid Plus LABA (MIRACLE)AsthmaNCT03186209AstraZeneca695
已完成
3 期
Efficacy and Safety Study of Mepolizumab Adjunctive Therapy in Participants With Severe Eosinophilic Asthma on Markers of Asthma ControlAsthmaNCT02281318GlaxoSmithKline556