A Phase II, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Pharmacodynamics, Safety, Tolerability, Pharmacokinetics, and Efficacy of RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 27
- 试验地点
- 3
- 主要终点
- 1. Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles, as assessed by MRI bilaterally
研究概览
简要总结
To evaluate the pharmacodynamic (PD) effects of RO7204239 compared with placebo, using magnetic resonance imaging (MRI) and to evaluate the safety of RO7204239 compared with placebo
研究设计
- 分配方式
- Randomized
- 主要目的
- Overall design
- 盲法
- Double (Subject, Investigator)
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Age ≥ 18 and ≤ 65 years at the time of signing the Informed Consent Form
- •Genetic confirmation of FSHD1 or FSHD2
- •Clinical findings consistent with FSHD according to the investigator’s clinical judgment
- •Ability to walk unassisted 10 meters (Timed 10-Meter Walk Test [10MWT])
- •Ricci Clinical Severity Scale score ≥ 2.5 and ≤ 4
- •Agreement to maintain the same frequency and intensity of physiotherapy, occupational therapy, and other forms of exercise during the clinical study
排除标准
- •Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 17 months after the final dose of RO7204239
- •Current or previous treatment (or receipt) of anti-myostatin therapies
- •Treatment with any investigational therapy within 90 days prior to screening, or 5 drug-elimination half-lives of the drug, whichever is longer
- •Contraindications to MRI scans, difficulties maintaining a prolonged supine position, or any other clinical history or examination finding that would pose a potential hazard in combination with MRI
- •Presence of clinically significant ECG abnormalities from average of triplicate measurement at screening indicating a safety risk for participants
- •Presence of clinically significant cardiovascular disease indicating a safety risk for participants. •Presence of clinically significant abnormal findings in echocardiography at screening, with the exception of mitral valve prolapse, which does not exclude participants from the study
结局指标
主要结局
1. Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles, as assessed by MRI bilaterally
1. Percent change from baseline in contractile muscle volume (CMV) of quadriceps femoris muscles, as assessed by MRI bilaterally
2. Incidence, severity, and causal relationship of adverse events, with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events v5.0
2. Incidence, severity, and causal relationship of adverse events, with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events v5.0
3. Change from baseline in vital signs, physical findings, ECG, echocardiogram, and clinical laboratory results
3. Change from baseline in vital signs, physical findings, ECG, echocardiogram, and clinical laboratory results
4. Incidence of local and systemic injection reactions
4. Incidence of local and systemic injection reactions
5. Incidence of abnormal laboratory findings
5. Incidence of abnormal laboratory findings
6. Incidence of abnormal ECG parameters
6. Incidence of abnormal ECG parameters
7. Incidence of abnormal echocardiographic parameters
7. Incidence of abnormal echocardiographic parameters
8. Incidence of abnormal vital signs
8. Incidence of abnormal vital signs
次要结局
- 2. Percent change from baseline in CMV of 36 muscles based on whole body MRI (excluding muscles with severe fat replacement)
- 3. Change from baseline in fat fraction of 36 muscles based on whole body MRI (excluding muscles with severe fat replacement)
- 4. Percent change from baseline in CMV of quadriceps femoris muscles, as assessed by MRI bilaterally
- 5. Change from baseline in fat fraction of quadriceps femoris muscles, as assessed by MRI bilaterally
- 6. Percent change from baseline in CMV of tibialis anterior muscles, as assessed by MRI bilaterally
- 7. Change from baseline in fat fraction of tibialis anterior muscles, as assessed by MRI bilaterally
- 8. Percent change from baseline in CMV of biceps brachii muscles, as assessed by MRI bilaterally
- 9. Change from baseline in fat fraction of biceps brachii muscles, as assessed by MRI bilaterally
- 10. Percent change from baseline in the contractile cross-sectional area (CSA) of skeletal muscle in the proximal lower limb muscles, as assessed by MRI bilaterally
- 11. Change from baseline in the fat fraction of proximal lower limb muscles, as assessed at a single mid femur slice by MRI bilaterally
- 12. Serum concentrations of RO7204239 at specified timepoints
- 13. Cmax of RO7204239 at specified timepoints
- 14. Area under the concentration-time curve (AUC) of RO7204239 at specified timepoints
- 15. Ctrough of RO7204239 at specified timepoints
- 16. Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study
- 1. Change from baseline in serum concentrations of total and free latent myostatin, and mature myostatin
研究者
Trial Information System - TISL
Scientific
F. Hoffmann-La Roche AG