307. カナバン病 Canavan disease Clinical trials / Disease details

臨床試験数 : 6 / 薬物数 : 11 - (DrugBank : 5) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2

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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	NCT04998396 (ClinicalTrials.gov)	September 8, 2021	5/8/2021	A Study of AAV9 Gene Therapy in Participants With Canavan Disease	A Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease ...	Canavan Disease	Biological: AAV9 BBP-812	Aspa Therapeutics	NULL	Recruiting	N/A	30 Months	All	18	Phase 1/Phase 2	United States
2	NCT04833907 (ClinicalTrials.gov)	April 1, 2021	24/3/2021	rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease	Phase 1/2, Open Label, Sequential Cohort Study of a Single Intracranial Dose of AVASPA Gene Therapy ...	Canavan Disease	Drug: rAAV-Olig001-ASPA;Drug: Levetiracetam;Drug: Prednisone	Myrtelle Inc.	NULL	Active, not recruiting	3 Months	60 Months	All	24	Phase 1/Phase 2	United States
3	NCT05317780 (ClinicalTrials.gov)	February 10, 2020	24/3/2022	Canavan-Single Patient IND	Expanded Access Trial of Systemic Delivery of Aspartoacylase ASPA (rAAV9-CB6-AspA) Gene Vector in a ...	Canavan Disease	Drug: rAAV9-CB6-AspA	Barry J. Byrne	University of Miami;University of Massachusetts, Worcester	Available	18 Months	24 Months	Male			United States
4	NCT00657748 (ClinicalTrials.gov)	September 2009	9/4/2008	Lithium and Acetate for Canavan Disease	Evaluation of the Tolerance and Efficiency of a Combined Oral Therapy With Lithium and GTA in Patien ...	Canavan Disease;Infantile;Deficiency Disease;Aspartoacylase;Leukodystrophy, Spongiform	Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)	Assistance Publique - Hôpitaux de Paris	European Leukodystrophy Association;European Leukodystrophy Association	Withdrawn	1 Year	15 Years	Both	0	Phase 2	France
5	NCT00724802 (ClinicalTrials.gov)	March 2008	18/6/2008	Oral Glyceryl Triacetate (GTA) in Newborns With Canavan	Oral Glyceryl Triacetate (GTA) in Newborns With Canavan	Canavan Disease	Dietary Supplement: GTA (Glyceryl triacetate);Drug: GTA glyceryl triacetate	Shaare Zedek Medical Center	NULL	Recruiting	N/A	18 Months	Both	1	N/A	Israel
6	NCT00278707 (ClinicalTrials.gov)	January 2006	15/1/2006	GTA-Glyceryltriacetate for Canavan Disease	Phase 1 Treatment With GTA in Two Infant With Canavan Disease	Infantile Canavan Disease;Deficiency Disease, Aspartoacylase	Drug: GTA: Glyceryltriacetate	Sheba Medical Center	NULL	Active, not recruiting	N/A	15 Months	Both	5	Phase 1	Israel

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