No registrations found.
ID
Source
Brief title
Health condition
Duchenne Muscular Dystrophy
Sponsors and support
Intervention
Outcome measures
Primary outcome
Effect of PRO051 on dystrophin expression in muscle and on muscle strength and function
Safety and tolerability of PRO051
Pharmacokinetics of PRO051
Secondary outcome
N/A
Background summary
This is an open-label phase I/II study to assess the effect, the safety and tolerability, and the pharmacokinetics of PRO051 at different dose levels after subcutaneous administration in 12-18 patients with Duchenne muscular dystrophy.
Study design
5 weeks of treatment plus 13 weeks follow up
Intervention
Subcutaneous injection of PRO051, once a week, for 5 weeks, different dose per group, 4 groups
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
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
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL1196 |
| NTR-old | NTR1241 |
| Other | EC Leuven : OG032 |
| ISRCTN | ISRCTN wordt niet meer aangevraagd |