A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy

Prosensa Holding B.V.0 sites18 target enrollmentStarted: January 7, 2008Last updated: October 2, 2012

Overview

No summary available.

•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).
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1\.2 Adults (18\-64 years) no

•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)

Sponsor

Prosensa Holding B.V.