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Raav8-des-hmtm1    (DrugBank: -)

1 disease
IDDisease name (Link within this page)Number of trials
111Congenital myopathy2

111. Congenital myopathy    [ 7 clinical trials,   13 drugs,   (DrugBank: 4 drugs),   1 drug target gene,   8 drug target pathways]
Searched query = "Congenital myopathy", "Nemaline myopathy", "Central core disease", "Minicore myopathy", "Myotubular myopathy", "Centronuclear myopathy", "Congenital fiber-type disproportion myopathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 7 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2017-000876-27-DE
(EUCTR)		22/05/201822/09/2017First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) PatientsASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients - ASPIRO X-linked Myotubular Myopathy (XLMTM)
MedDRA version: 20.0;Level: HLGT;Classification code 10029317;Term: Neuromuscular disorders;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		Product Name: rAAV8-Des-hMTM1
Product Code: AT132		Audentes Therapeutics Inc.NULLNot RecruitingFemale: no
Male: yes		20Phase 1;Phase 2France;United States;Germany;United Kingdom
2EUCTR2017-000876-27-FR
(EUCTR)		23/04/201825/05/2018First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) PatientsASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients - ASPIRO X-linked Myotubular Myopathy (XLMTM);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: rAAV8-Des-hMTM1
Product Code: AT132		Audentes Therapeutics Inc.NULLNot RecruitingFemale: no
Male: yes		12Phase 1;Phase 2United States;France;Germany;United Kingdom