A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients
- Conditions
- Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 SkippingMedDRA version: 19.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2014-002008-25-FR
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 48
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
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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method