EUCTR2014-002008-25-IT进行中（未招募）1 期
A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 39
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1. Male aged 6 to 15, inclusive.
- •2. For treated patients (all patients in Part 1 + 12 additional treated patients in Part 2), established clinical diagnosis of DMD amenable to exon 53 skipping (e.g. deletions of exons such as 42-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52, or 54-58) as documented by a genetic report from an accredited laboratory confirming deletion endpoints by multiplex ligation-dependent probe amplification (MLPA) or sequencing.
- •3. For Part 2 untreated control patients, established clinical diagnosis of DMD with confirmed genomic deletion of exon(s) not amenable to exon 53 skipping as documented by an accredited laboratory and genomic methodology.
- •4. Have intact right and left biceps muscles or an alternative upper arm muscle group.
- •5. Have stable cardiac and pulmonary function that, in the Investigator’s opinion, is unlikely to decompensate over the duration of the study.
- •6. Achieve a mean distance of two separate assessments on two consecutive days at screening and again at baseline (prior to investigational drug product administration) =250 meters on the 6MWT with the two means being ±15% of each other. (Personal assistance or use of any assistive devices for ambulation is not permitted during the 6MWT.)
- •7. Patients must meet one of the following two criteria:
- •NSAA (North Star Ambulatory Assessment) total score >17; or
- •Rise (Gowers) time < 7 seconds
- •8. Have been on a stable dose of oral corticosteroids for at least 24 weeks prior to Week 1 and the dose is expected to remain constant throughout the study. Note: patients may be allowed to take other (non-RNA antisense or gene therapy) medication including angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), ß blockers, and potassium provided they have been on a stable dose for 12 weeks prior to Week 1 and the dose is expected to remain constant throughout the study.
- •9. Have a parent(s) or legal guardian(s) who is able to understand and comply with the study procedure requirements.
- •10. Be willing to provide informed assent and have a parent(s) or legal guardian(s) who is willing to provide written informed consent for the patient to participate in the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 48
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Use of any pharmacologic treatment, other than corticosteroids, that might have an effect on muscle strength or function within 12 weeks prior to study entry (e.g., growth hormone, anabolic steroids).
- •2. Previous treatment with the experimental agents BMN-195 (SMT C1100) or PRO053.
- •3. Previous or current treatment with any other experimental treatments within 12 weeks prior to study entry or participation in any other clinical trial within 6 months prior to study entry.
- •4. Have a left ventricular ejection fraction (LVEF) of <50% (or equivalent fractional shortening) based on the screening ECHO and QTc (Fredericia’s correction) >450 msec.
- •5. Have a forced vital capacity [FVC] <50% of predicted value or require nocturnal ventilation.
- •6. Major surgery within 3 months prior to Week 1 or planned orthopedic surgery for any time during this study which would interfere with the ability to perform outcome measures.
- •7. Use of any aminoglycoside antibiotic or statin within 12 weeks of Week 1 or need for use of an aminoglycoside antibiotic or statin during the study.
- •8. Presence of other clinically significant illness including significant cardiac, pulmonary, hepatic, renal, hematologic, immunologic, behavioral disease or malignancy.
- •9. Loss =30 degrees of plantar flexion from the normal range of movement at the ankle joint due to contracture (i.e. fixed loss of >10 degrees plantar flexion from plantagrade assuming normal range of dorsiflexion of 20 degrees).
- •10. Change in contracture treatment such as serial casting, contracture control devices, night splints, stretching exercises (passive, active, self) within 3 months prior to enrollment, or expected need for such intervention during the study.
- •11. Prior or ongoing medical condition that, in the Investigator’s opinion, could interfere with the patient’s participation in the study.
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