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van CCT (UK)

van CCT (UK)

Effect, Safety and Pharmacokinetics Study of PRO051 to Treat DMD

- candidate number3100
- NTR NumberNTR1241
- ISRCTNISRCTN wordt niet meer aangevraagd
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR7-mrt-2008
- Secondary IDsOG032 EC Leuven
- Public TitleEffect, Safety and Pharmacokinetics Study of PRO051 to Treat DMD
- Scientific TitleA 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
- hypothesis
- Healt Condition(s) or Problem(s) studiedDuchenne muscular dystrophy
- Inclusion criteria1. 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 criteria1. 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
- mec approval receivedyes
- multicenter trialyes
- randomisedno
- group[default]
- TypeSingle arm
- Studytypeintervention
- planned startdate 1-apr-2008
- planned closingdate1-feb-2009
- Target number of participants18
- InterventionsSubcutaneous injection of PRO051, once a week, for 5 weeks, different dose per group, 4 groups
- Primary outcomeEffect of PRO051 on dystrophin expression in muscle and on muscle strength and function

Safety and tolerability of PRO051
Pharmacokinetics of PRO051
- Secondary outcomeN/A
- Timepoints5 weeks of treatment plus 13 weeks follow up
- Trial web site
- statusopen: patient inclusion
- CONTACT FOR PUBLIC QUERIESClinical Study Manager H. Akker, van den
- CONTACT for SCIENTIFIC QUERIESClinical Study Manager H. Akker, van den
- Sponsor/Initiator Prosensa Therapeutics B.V.
- Funding
(Source(s) of Monetary or Material Support)
Prosensa Therapeutics B.V.
- PublicationsN/A
- Brief summaryThis 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.
- Main changes (audit trail)
- RECORD7-mrt-2008 - 13-feb-2009

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