Canavan Disease

• CLINICAL_TRIALS trial_result · 2026-04-17
  Clinical Trial Update: Natural History Study of Patients With Canavan Disease (CANinform Study)

  This clinical trial update notes the completion of CANinform, a natural history study of Canavan disease sponsored by Aspa Therapeutics.

• CLINICAL_TRIALS research · 2026-04-17
  Clinical Trial Update: A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) [Phase 1, Phase 2]

  CANaspire study is evaluating an AAV9-mediated gene therapy for Canavan disease and is currently recruiting in Phase 1/2.

• CLINICAL_TRIALS research · 2025-11-14
  Clinical Trial Update: rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease [Phase 1, Phase 2]

  This clinical trial update outlines Phase 1/2 testing of rAAV-Olig001-ASPA gene therapy for Canavan disease. Enrollment is by invitation.

• CLINICAL_TRIALS research · 2025-10-23
  Clinical Trial Update: The Myelin Disorders Biorepository Project

  This clinical trials update describes the Myelin Disorders Biorepository Project (NCT03047369) from Children’s Hospital of Philadelphia, cur…

• CLINICAL_TRIALS trial_result · 2017-08-17
  Clinical Trial Update: Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent. [NA]

  Completed efficacy study evaluating an online educational module used before carrier genetic screening in people of Ashkenazi Jewish descent…

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• CLINICAL_TRIALS withdrawal · 2015-04-21
  Clinical Trial Update: Lithium and Acetate for Canavan Disease [Phase 2]

  Phase 2 trial investigating lithium acetate for Canavan disease has been withdrawn.

• CLINICAL_TRIALS trial_result · 2011-06-21
  Clinical Trial Update: Oral Glyceryl Triacetate (GTA) in Newborns With Canavan [NA]

  An update on a clinical trial evaluating oral glyceryl triacetate (GTA) in newborns with Canavan disease; the current trial status is unknow…

• CLINICAL_TRIALS research · 2006-08-15
  Clinical Trial Update: GTA-Glyceryltriacetate for Canavan Disease [Phase 1]

  Phase 1 clinical trial update for GTA-Glyceryltriacetate in Canavan disease; the trial status is currently unknown.