E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping |
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E.1.1.1 | Medical condition in easily understood language |
Duchenne muscular dystrophy |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10013801 |
E.1.2 | Term | Duchenne muscular dystrophy |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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. |
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E.2.2 | Secondary objectives of the trial |
• To evaluate changes in physical functioning with long-term treatment with 30 mg/kg of casimersen or golodirsen. • To evaluate changes in pulmonary function with long-term treatment with 30 mg/kg of casimersen or golodirsen. • To evaluate immunogenicity of long-term treatment with 30 mg/kg casimersen or golodirsen. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
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E.4 | Principal exclusion criteria |
1. Any 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. 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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Patient incidence of serious adverse events (SAEs) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
• 6-minute walk test (6MWT) • Performance of the Upper Limb (PUL) • North Star Ambulatory Assessment (NSAA) • Loss of ambulation (LOA) by continued wheelchair use (CWU) • Pulmonary function tests (PFTs) • Need for assisted ventilation • Percentage of patients who develop anti-dystrophin, anti-casimersen, or anti-golodirsen antibodies |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
6MWT, PUL, NSAA, PFTs, CWU: Screening, Week 24, 48, 72, 96, 120, 144 Need for assisted ventilation: continuous Immunogenicity: Screening, Week 12, 24, 36, 48, 72, 96, 120, 144 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 28 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Belgium |
Canada |
Czech Republic |
Finland |
France |
Germany |
Ireland |
Israel |
Italy |
Poland |
Spain |
Sweden |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |