113. Muscular dystrophy Clinical trials / Disease details


Clinical trials : 622 Drugs : 485 - (DrugBank : 99) / Drug target genes : 59 - Drug target pathways : 168

  
1 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04240314
(ClinicalTrials.gov)
January 15, 202022/1/2020AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.Phase I/IIa Systemic Gene Delivery Clinical Trial of scAAV9.U7.ACCA for Exon 2 Duplication-Associated Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: scAAV9.U7.ACCAMegan WaldropAudentes TherapeuticsEnrolling by invitation6 Months13 YearsMale3Phase 1/Phase 2United States