256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details


臨床試験数 : 180 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105

  
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1EUCTR2019-003595-38-GB
(EUCTR)
20/11/202003/07/2020FORTIS: AAV8-Delivered Gene Therapy in Late Onset Pompe DiseaseFORTIS: A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Disease - FORTIS Late Onset Pompe Disease
MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: AT845
Product Code: AT845
Audentes Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
8Phase 1;Phase 2Germany;Italy;United Kingdom
2NCT04174105
(ClinicalTrials.gov)
October 27, 202013/11/2019Gene Transfer Study in Patients With Late Onset Pompe DiseaseA Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe DiseasePompe Disease (Late-onset)Genetic: AT845Astellas Gene TherapiesNULLRecruiting18 Years80 YearsAll12Phase 1/Phase 2United States;United Kingdom;Germany