2023-506737-30-00招募中2 期
A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants with Duchenne Muscular Dystrophy
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 6
- 主要终点
- 1. Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiography
研究概览
简要总结
To assess the safety and tolerability of BMN 351 at different dose levels in participants with DMD
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Is male and age 4 through 10 years at Screening.
- •Clinical diagnosis of Duchenne muscular dystrophy in the opinion of the investigator resulting from a documented dystrophin mutation in the DMD gene amenable to exon 51 skipping as reviewed by a central genetic counselor.
- •Ambulatory at Screening, defined as able to walk independently without assistive devices and complete the timed 10 meter walk/run test in 8 seconds or less.
- •Not currently daytime ventilator dependent and not expected to need daytime mechanical or noninvasive ventilation within the next year in the opinion of the investigator.
- •Currently receiving treatment with oral corticosteroids or vamorolone, on a stable dose for at least 12 weeks prior to Baseline, and must remain on a consistent dose/dose regimen throughout the study except for modifications to accommodate changes in weight.
- •Normal urinalysis at Screening (trace protein allowed).
排除标准
- •For children 7 years of age or older, forced expiratory volume (FEV1) < 60% of predicted.
- •Current or history of liver or renal disease.
- •Left ventricular ejection fraction (LVEF) < 55% based on an ECHO performed within 3 months prior to the Baseline (Day 1) visit.
- •Mean QT interval corrected with Fridericia’s method (QTcF) ≥ 450 msec on the Screening electrocardiogram (ECG) conducted in triplicate.
- •Platelet count of < 150 x 10^9/L at Screening.
- •Renal function laboratory parameters outside of prespecified values as defined per protocol.
- •Treatment with any exon skipping therapy within 12 weeks prior to Baseline (Day 1) or with any gene therapy for the treatment of DMD at any time.
研究组 & 干预措施
Exon 51 specific phosphorothioate oligonucleotide
Test
干预措施: Exon 51 specific phosphorothioate oligonucleotide (Drug)
结局指标
主要结局
1. Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiography
1. Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiography
2. Physical examination
2. Physical examination
3. Safety laboratory test parameters
3. Safety laboratory test parameters
4. ECG parameters
4. ECG parameters
次要结局
- 1. BMN 351 plasma PK, urine PK and concentration in muscle
研究者
Clinical Trial Information Desk
Scientific
Biomarin Pharmaceutical Inc.
研究点 (6)
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