Duchenne Muscular Dystrophy
Conditions
Interventions
Product Name: PRO044
Product Code: PRO044
Pharmaceutical Form: Solution for injection
INN or Proposed INN: h44AON188
Current Sponsor code: PRO044
Concentration unit: mg/ml milligram(s)/millilitre
Conc
Sponsors
Prosensa Therapeutics B.V
Eligibility
Sex/Gender
Male
Inclusion criteria
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 Are the trial subjects under 18? yes Number of subjects for this age range: 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
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety and tolerability of PRO044 at different dose levels in patients with Duchenne muscular dystrophy ;Secondary Objective: 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. ;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 a-1-microglobulin) - Complement split products (C3a, SC5b-9, Bb) - Cytokines (IL-6, TNF-a) 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) | — |
Countries
Belgium, Italy, Netherlands, Sweden
Outcome results
None listed