search  
 


Home

Who are we?

Why
register?


Signup for
registration


Online registration

Log in to register
your trial


Search a trial

NRT en CCMO

Contact

NEDERLANDS





MetaRegister
van CCT (UK)


ISRCTN-Register
van CCT (UK)


Exploratory study on the local dystrophin production restoring effect and safety and tolerability of a single intramuscular dose of antisense oligoribonucleotide PRO051 in Duchenne muscular dystrophy patients.


- candidate number2143
- NTR NumberNTR712
- ISRCTNIncomplete info for ISRCTN
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR22-jun-2006
- Secondary IDsN/A 
- Public TitleExploratory study on the local dystrophin production restoring effect and safety and tolerability of a single intramuscular dose of antisense oligoribonucleotide PRO051 in Duchenne muscular dystrophy patients.
- Scientific TitleExploratory study on the local dystrophin production restoring effect and safety and tolerability of a single intramuscular dose of antisense oligoribonucleotide PRO051 in Duchenne muscular dystrophy patients.
- ACRONYMN/A
- hypothesisAntisense oligoribonucleotides inducing exon skipping is a promising approach to restore dystrophin synthesis in patients with Duchenne.
- Healt Condition(s) or Problem(s) studiedWet-wrap therapy
- Inclusion criteria1. Duchenne muscular dystrophy patients;
2. Age between 8 and 16 years;
3. Positive in vitro dystrophin RNA skip test in prior skin or muscle biopsy or in screening skin biopsy.
- Exclusion criteriaN/A
- mec approval receivedyes
- multicenter trialno
- randomisedno
- group[default]
- Type2 or more arms, non-randomized
- Studytypeintervention
- planned startdate 22-jun-2006
- planned closingdate1-jan-2007
- Target number of participants6
- InterventionsTreatment with PRO-051.
- Primary outcomePharmacodynamics (dystrophin restoring effect).
- Secondary outcomeSafety and tolerability.
- TimepointsN/A
- Trial web siteN/A
- statusinclusion stopped: follow-up
- CONTACT FOR PUBLIC QUERIES G. Platenburg
- CONTACT for SCIENTIFIC QUERIESDr. J. Verschuuren
- Sponsor/Initiator Prosensa Therapeutics B.V.
- Funding
(Source(s) of Monetary or Material Support)
Prosensa Therapeutics B.V.
- PublicationsN/A
- Brief summarySingle-dose, open, single-center study in children with Duchenne muscular dystrophy. On Day 0 eligible patients will receive PRO051 on a single occasion by intramuscular injections. On Day 28 a biopsy from the tibialis anterior muscle will be taken.
- Main changes (audit trail)
- RECORD22-jun-2006 - 19-okt-2009


  • Indien u gegevens wilt toevoegen of veranderen, kunt u een mail sturen naar nederlands@trialregister.nl