246. メチルマロン酸血症 Methylmalonic acidemia Clinical trials / Disease details

臨床試験数 : 23 / 薬物数 : 31 - (DrugBank : 8) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23

2 trials found

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT05506254 (ClinicalTrials.gov)	July 20, 2022	16/8/2022	Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy	A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001	Methylmalonic Acidemia	Drug: hLB-001	LogicBio Therapeutics, Inc	Alexion	Active, not recruiting	N/A	N/A	All	8		United States
2	NCT04581785 (ClinicalTrials.gov)	May 29, 2021	2/10/2020	Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia	A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations	Methylmalonic Acidemia	Biological: hLB-001	LogicBio Therapeutics, Inc	Alexion	Active, not recruiting	6 Months	12 Years	All	8	Phase 1/Phase 2	United States;Saudi Arabia