2025-524371-23-00招募中3 期
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of BBP-418 (Ribitol) in Participants with Limb-Girdle Muscular Dystrophy 2I/R9 (LGMD2I/R9)
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 35
- 试验地点
- 5
研究概览
简要总结
To assess the long-term safety of BBP-418 in participants with LGMD2I/R9 To assess the long-term clinical efficacy of BBP-418 in participants with LGMD2I/R9
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Completed Study MLB-01-005 on study drug through the final clinic visit (Month 36 or another qualifying end-of-study visit as determined by the Sponsor).
- •The participant (or parent/guardian) who signs the ICF understands the study procedures and agrees to participate in the study by giving informed consent (or assent, if <18 years of age).
- •Is willing and able to complete all study procedures according to the Schedule of Assessments.
- •A WOCBP or a nonsterile male participant must be willing to use an acceptable method of contraception (see Section 5.3) from the time of consent through 30 days after the last dose of study drug in this study.
排除标准
- •Has developed clinically significant concomitant disease that would, in the Investigator’s opinion, be likely to unfavorably impact study participation, including: a. Any significant concomitant medical condition, including psychiatric, cardiac, renal, pulmonary, hepatic, or endocrine disease other than that associated with LGMD2I/R
- •b. Any other significant laboratory, vital sign, ECG abnormality, clinical history, or finding.
- •Is pregnant (based on the Baseline / Day 1 pregnancy test result) and/or breastfeeding or planning to conceive children within the projected duration of the study through 30 days after the last dose of study drug in this study.
- •Has active suicidal ideation, defined as having a suicide ideation score of 4 (Active Suicidal Ideation with Some Intent to Act, without Specific Plan) or 5 (Active Suicidal Ideation with Specific Plan and Intent) on the C-SSRS at Baseline / Day 1.
研究组 & 干预措施
Ribitol
Test
干预措施: Ribitol (Drug)
研究者
Chief Medical Officer
Scientific
ML Bio Solutions Inc.
研究点 (5)
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