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


臨床試験数 : 5 薬物数 : 9 - (DrugBank : 3) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2

  
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PhaseCountries
1NCT04998396
(ClinicalTrials.gov)
September 8, 20215/8/2021A Study of AAV9 Gene Therapy in Participants With Canavan DiseaseA Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA GeneCanavan Disease;Demyelinating Diseases;Genetic Diseases, Inborn;Metabolism, Inborn Errors;Metabolic Disease;Neurological Diseases or Conditions;Leukodystrophy;Leukoencephalopathies;Neurodevelopmental Disorders;Neurodegenerative DiseasesBiological: AAV9 BBP-812Aspa TherapeuticsNULLRecruitingN/A30 MonthsAll18Phase 1/Phase 2United States
2NCT04833907
(ClinicalTrials.gov)
April 1, 202124/3/2021rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan DiseasePhase 1/2, Open Label, Sequential Cohort Study of a Single Intracranial Dose of AVASPA Gene Therapy for Treatment of Children With Typical Canavan DiseaseCanavan DiseaseDrug: rAAV-Olig001-ASPA;Drug: Levetiracetam;Drug: PrednisoloneMyrtelle Inc.NULLRecruiting3 Months60 MonthsAll24Phase 1/Phase 2United States
3NCT00657748
(ClinicalTrials.gov)
September 20099/4/2008Lithium and Acetate for Canavan DiseaseEvaluation of the Tolerance and Efficiency of a Combined Oral Therapy With Lithium and GTA in Patients With Canavan DiseaseCanavan Disease;Infantile;Deficiency Disease;Aspartoacylase;Leukodystrophy, SpongiformDrug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)Assistance Publique - Hôpitaux de ParisEuropean Leukodystrophy Association;European Leukodystrophy AssociationWithdrawn1 Year15 YearsBoth0Phase 2France
4NCT00724802
(ClinicalTrials.gov)
March 200818/6/2008Oral Glyceryl Triacetate (GTA) in Newborns With CanavanOral Glyceryl Triacetate (GTA) in Newborns With CanavanCanavan DiseaseDietary Supplement: GTA (Glyceryl triacetate);Drug: GTA glyceryl triacetateShaare Zedek Medical CenterNULLRecruitingN/A18 MonthsBoth1N/AIsrael
5NCT00278707
(ClinicalTrials.gov)
January 200615/1/2006GTA-Glyceryltriacetate for Canavan DiseasePhase 1 Treatment With GTA in Two Infant With Canavan DiseaseInfantile Canavan Disease;Deficiency Disease, AspartoacylaseDrug: GTA: GlyceryltriacetateSheba Medical CenterNULLActive, not recruitingN/A15 MonthsBoth5Phase 1Israel