A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients
- Conditions
- Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53SkippingMedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2017-004625-32-BE
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 150
1. Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
2. If sexually active, agrees to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose of study drug. The sexual partner must also use a medically acceptable form of contraceptive (eg, female oral contraceptives) during this time frame.
3. Is able to understand and comply with all the study requirements and, if under 18 years of age, had as (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements.
4. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.
5. Is between 7 and 23 years of age, inclusive, at randomization.
Are the trial subjects under 18? yes
Number of subjects for this age range: 135
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. A medical condition that could, in the Investigator’sopinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
2. Any patient who, in the Investigator’s opinion, seems unable/unwilling to comply with the study procedures.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of the study is to evaluate the safety and tolerability of long-term treatment with 30 mg/kg of casimersen or golodirsen.;Secondary Objective: • To evaluate changes in physical functioning with long-term treatment with 30 mg/kg of casimersen or golodirsen.<br>• To evaluate changes in pulmonary function with long-term treatment with 30 mg/kg of casimersen or golodirsen.;Primary end point(s): Patient incidence of serious adverse events (SAEs);Timepoint(s) of evaluation of this end point: continuous
- Secondary Outcome Measures
Name Time Method Secondary end point(s): • 6-minute walk test (6MWT)<br>• Performance of the Upper Limb (PUL)<br>• North Star Ambulatory Assessment (NSAA)<br>• Loss of ambulation (LOA)<br>• Pulmonary function tests (PFTs)<br>• Need for assisted ventilation<br>• Percentage of patients who develop anti-dystrophin, anti-casimersen, or anti-golodirsen antibodies;Timepoint(s) of evaluation of this end point: 6MWT, PUL, NSAA, PFTs: Screening, Week 24, 48, 72, 96, 120, 144<br>LOA: Week 60, 72, 84, 96, 108, 120, 132, 144<br>Need for assisted ventilation: continuous<br>Immunogenicity*: Screening, Week 1, 4, 8, 12, 24, 36, 48, 72, 96, 120, 144<br><br>*Patients who previously participated in 4045-101 or 4053-101 do not need to have immunogenicity testing at Weeks 1, 4 and 8