Not applicableActive Not RecruitingNCT04126005What this trial is testingNatural History Study of Patients With Canavan Disease (CANinform Study)Who this might be right forCanavan Disease Aspa Therapeutics 70
Not applicableStudy completedNCT02699190What this trial is testingLeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesWho this might be right forLeukodystrophyWhite Matter Disease4H Syndrome+61 more Children's Hospital of Philadelphia 236
Testing effectiveness (Phase 2)Looking for participantsNCT04998396What this trial is testingAAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)Who this might be right forCanavan Disease Aspa Therapeutics 26
Not applicableStudy completedNCT01999257What this trial is testingEfficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.Who this might be right forTay Sachs DiseaseCanavan DiseaseFamilial Dysautonomia McGill University Health Centre/Research Institute of the McGill University Health Centre 60
Large-scale testing (Phase 3)Ended earlyNCT00654433What this trial is testingALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesWho this might be right forInherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more Aldagen 40
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Testing effectiveness (Phase 2)WithdrawnNCT00657748What this trial is testingLithium and Acetate for Canavan DiseaseWho this might be right forCanavan DiseaseInfantileDeficiency Disease+2 more Assistance Publique - Hôpitaux de Paris
Not applicableUnknownNCT00724802What this trial is testingOral Glyceryl Triacetate (GTA) in Newborns With CanavanWho this might be right forCanavan Disease Shaare Zedek Medical Center 1
Testing effectiveness (Phase 2)Enrolling By InvitationNCT04833907What this trial is testingRAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan DiseaseWho this might be right forCanavan Disease Myrtelle Inc. 24
Not applicableNo Longer AvailableNCT05317780What this trial is testingCanavan-Single Patient INDWho this might be right forCanavan Disease University of Florida
Early research (Phase 1)UnknownNCT00278707What this trial is testingGTA-Glyceryltriacetate for Canavan DiseaseWho this might be right forInfantile Canavan DiseaseDeficiency Disease, Aspartoacylase Sheba Medical Center 5
Not applicableLooking for participantsNCT03047369What this trial is testingThe Myelin Disorders Biorepository ProjectWho this might be right forLeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more Children's Hospital of Philadelphia 12,000
Not applicableStudy completedNCT02851563What this trial is testingA Natural History Study of Canavan DiseaseWho this might be right forCanavan Disease NYU Langone Health 21