A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: Male

Target Recruitment: 260

Inclusion Criteria

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, has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements.
4. Is willing and legally able to provide written informed assent and/or consent, or, if not legally able to provide written informed assent and/or consent, has (a) parent(s) or legal guardian(s) who is (are) willing and legally able to provide written informed assent and/or consent for the patient to participate in the study.
5. Is between 7 and 23 years of age, inclusive, at enrollment.
Are the trial subjects under 18? yes
Number of subjects for this age range: 260
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 26
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Any medical condition that could, in the Investigator's opinion: 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.
3. Treatment with any investigational therapies at the time of consent or within 6 months prior to dosing if there was an unexpected gap in treatment.

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.<br>• To evaluate immunogenicity of long-term treatment with 30 mg/kg 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): Exploratory endpoints:<br>• 6-minute walk test (6MWT)<br>• Performance of the Upper Limb (PUL)<br>• North Star Ambulatory Assessment (NSAA)<br>• Loss of ambulation (LOA) by continued wheelchair use (CWU)<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, CWU: Screening, Week 24, 48, 72, 96, 120, 144<br>Need for assisted ventilation: continuous<br>Immunogenicity: Screening, Week 12, 24, 36, 48, 72, 96, 120, 144

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