E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Duchenne Muscular Dystrophy |
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MedDRA Classification |
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 |
To assess the safety and tolerability of PRO044 at different dose levels in patients with Duchenne muscular dystrophy
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E.2.2 | Secondary objectives of the trial |
To assess the effect of PRO044 at different dose levels in patients with Duchenne muscular dystrophy To determine the pharmacokinetics of PRO044 at different dose levels after subcutaneous administration in patients with Duchenne muscular dystrophy.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Age at least 5 and not older than 16 years on the day of first drug administration 2.Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO044 3.Life expectancy at least 6 months after inclusion in the trial
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E.4 | Principal exclusion criteria |
1. Ventilator dependency 2. Previous treatment with investigational medicinal treatment within 6 months prior to the start of the pre-screening for the study 3. Previous treatment with idebenone within 6 months prior to the start of the pre-screening for the study.
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E.5 End points |
E.5.1 | Primary end point(s) |
This is an exploratory study. The primary endpoints are safety and tolerability measures: - Adverse events - Local tolerability - Safety biochemistry parameters - Safety hematology parameters - Coagulation (aPTT) - Cystatin C - Urinalysis (dipstick, quantitative protein and creatinine, and creatinine, and α-1-microglobulin) - Complement split products (C3a, SC5b-9, Bb) - Cytokines (IL-6, TNF-α) and chemokine (MCP-1) - Antibodies to dystrophin - ECG parameters
Secondary endpoints (also exploratory) relating to efficacy are: - Presence of (BMD like) dystrophin expression after treatment (in muscle biopsy) - Production of exon skip 44 mRNA (in muscle biopsy and mononuclear blood cells) - Muscle function (timed tests and 6-minutes walk test) - Muscle strength (handheld myometry and spirometry)
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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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
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) | Yes |
E.7.1.1 | First administration to humans | Yes |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
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 | Information not present in EudraCT |
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 |
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E.8.7 | Trial has a data monitoring committee | Yes |
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 | 1 |
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 | 1 |
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 |