Raav8-des-hmtm1    (DrugBank: -)

1 disease
告示番号疾患名(ページ内リンク)臨床試験数
111先天性ミオパチー2

111. 先天性ミオパチー [臨床試験数:7,薬物数:13(DrugBank:4),標的遺伝子数:1,標的パスウェイ数:8
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_
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agemin
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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