Duchenne Muscular Dystrophy
Conditions
Interventions
Subcutaneous injection of PRO051, once a week, for 5 weeks, different dose per group, 4 groups
Sponsors
Prosensa
Eligibility
Inclusion criteria
Inclusion criteria: 1. Boys aged between 5 and 16 years inclusive 2. Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO051 3. Not ventilator dependent 4. Life expectancy of at least 6 months 5. No previous treatment with investigational medicinal treatment within 6 months prior to the study 6. Willing and able to adhere to the study visit schedule and other protocol requirements 7. Written informed consent signed (by parent(s)/legal guardian and/or the patient, according to the local regulations).
Exclusion criteria
Exclusion criteria: 1. Aberrant RNA splicing and/or aberrant response to PRO051, detected by in vitro PRO051 assay during screening 2. Known presence of dystrophin in > 5% of fibres in a pre-study diagnostic muscle biopsy 3. Severe muscle abnormalities defined as increased signal intensity in >50% of the tibialis anterior muscle at MRI 4. FEV1 and/or FVC < 60% of predicted 5. Current or history of liver or renal disease 6. Acute illness within 4 weeks prior to treatment (Day 0) which may interfere with the measurements 7. Severe mental retardation which in the opinion of the investigator prohibits participation in this study 8. Severe cardiac myopathy which in the opinion of the investigator prohibits participation in this study 9. Need for mechanical ventilation 10. Creatinine concentration above 1.5 times the upper limit of normal (age corrected) 11. Serum ASAT and/or ALAT concentration(s) which suggest hepatic impairment 12. Use of anticoagulants, antithrombotics or antiplatelet agents 13. Subject has donated blood less than 90 days before the start of the study 14. Current or history of drug and/or alcohol abuse 15. Participation in another trial with an investigational product
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Effect of PRO051 on dystrophin expression in muscle and on muscle strength and function Safety and tolerability of PRO051 Pharmacokinetics of PRO051 | — |
Contacts
Public ContactH. Akker, van den
Prosensa Wassenaarseweg 72
Outcome results
None listed