An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
研究概览
简要总结
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). This is an open label study to determine the safety and tolerability of ascending dose levels of BBP-418 in the treatment of ambulatory and non-ambulatory patients with LGMD2I for which no approved therapy currently exists.
详细描述
This is an open label study in ambulatory and non-ambulatory subjects with LGMD2I (also known as LGMD R9) previously enrolled in the natural history Study MLB-01-001. This is a study to determine the safety and tolerability of ascending dose levels of BBP-418 in those subjects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have a body weight >30 kg
- •Have a genetically confirmed diagnosis of LGMD2I and be clinically affected (defined as demonstrating clinical weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity)
- •Able to complete the 10-meter walk test in ≤ 12 seconds unaided ("moderate disease") or are with "severe disease"/non-ambulatory as defined by being unable to complete the 10-meter walk unaided in >12 seconds
- •Willing to use an adequate method of contraception from time of consent through 12 weeks after last dose
- •Previous enrolment in the Natural History study MLB-01-001
排除标准
- •Evidence of clinically significant concomitant disease, including:
- •Any history of a gastrointestinal condition, including surgeries, which may affect absorption after oral administration
- •Any significant concomitant medical condition, including cardiac, pulmonary, renal, hepatic or endocrine disease other than that associated with LGMD2I
- •Any condition other than LGMD2I requiring therapy with prescription medicine (medication for common and mild concomitant conditions may be permitted after consultation with the PI)
- •Any other laboratory, vital sign, ECG abnormality, or clinical history or finding that, in the investigator's opinion, is likely to unfavorably alter the risk-benefit of study participation, confound study results, or interfere with study conduct or compliance
- •If pregnant and/or breastfeeding or planning to conceive children within the projected duration of the study through 12 weeks after the last dose of study treatment.
- •History of drug abuse including alcoholism within 2 years prior to consenting
- •Use of ribose or other sugar alcohol-containing supplement within 60 days of Day 1
- •Use of a corticosteroid within 60 days of Day 1
- •Presence of a platelet disorder, bleeding disorder or other contraindication to muscle biopsy
- •Actively on an experimental therapy or device or was on an experimental therapy or device within 60 days prior to Day 1.
研究组 & 干预措施
Cohort 1
Subjects will receive 6 grams of BBP-418 once daily x 90 days, then 12 grams twice daily (BID, a least 8 hours apart) of BBP-418 daily until study completion.
干预措施: BBP-418 (Drug)
Cohort 2
Subjects will receive 6 grams of BBP-418 twice daily (BID, at least 8 hours apart) x 90 days, then 12 grams BID of BBP-418 daily until study completion.
干预措施: BBP-418 (Drug)
Cohort 3
Subjects will receive 12 grams of BBP-418 twice daily (BID, at least 8 hours apart) x 90 days, then 12 grams BID of BBP-418 daily until study completion.
干预措施: BBP-418 (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
时间窗: 60 months
次要结局
- Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior(24 months)
- Pharmacokinetic profile of BBP-418 by assessment of maximum concentration (Cmax)(24 months)
- Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (α-DG)(24 months)
- Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC)(24 months)