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3

111. 先天性ミオパチー


臨床試験数 : 10 薬物数 : 17 - (DrugBank : 5) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 9
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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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 1/2/3, 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
26Phase 1;Phase 2France;United States;Canada;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
3EUCTR2017-000876-27-GB
(EUCTR)
06/12/201731/07/2017First-in-human study of AT132 in X-Linked Myotubular Myopathy (XLMTM) Patients ASPIRO: 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);Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: rAAV8-Des-hMTM1
Product Code: AT132
Audentes Therapeutics Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
20 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Germany;United Kingdom