4 completed, 2 recruiting, 2 unknown, 1 active not recruiting. 3 Phase 1, 3 Phase 2, 2 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 3 | Enrolling by invitation 1, Recruiting 1, Unknown 1 |
| Phase 2 | 3 | Enrolling by invitation 1, Recruiting 1, Withdrawn 1 |
| Not applicable | 2 | Completed 1, Unknown 1 |
| Phase 3 | 1 | Terminated 1 |
Follow Canavan Disease: new trials by email Literature Review of Canavan Disease trials Research Report on Canavan Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) AAV9 BBP-812 - Canavan Disease | Phase 1 / Phase 2 | Recruiting | Aspa Therapeutics | 26 | 2021-09-08 | 2026-04-17 |
| rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease rAAV-Olig001-ASPA, Levetiracetam, Prednisone - Canavan Disease | Phase 1 / Phase 2 | Enrolling by invitation | Myrtelle Inc. | 24 | 2021-04-01 | 2025-11-14 |
| Natural History Study of Patients With Canavan Disease (CANinform Study) Canavan Disease | Completed | Aspa Therapeutics | 67 | 2019-10-10 | 2026-04-17 | |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies Leukodystrophy, White Matter Disease | Completed | Children's Hospital of Philadelphia | 236 | 2017-01-06 | 2025-11-10 | |
| The Myelin Disorders Biorepository Project Leukodystrophy, White Matter Disease | Recruiting | Children's Hospital of Philadelphia | 12000 | 2016-12-08 | 2025-10-23 | |
| A Natural History Study of Canavan Disease Canavan Disease | Completed | NYU Langone Health | 21 | 2016-05-01 | 2019-11-01 | |
| Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent. Online pre-test genetic education tool - Tay Sachs Disease, Canavan Disease | Not applicable | Completed | McGill University Health Centre/Research Institute of the McGill University Health Centre | 60 | 2014-07-01 | 2017-08-17 |
| Lithium and Acetate for Canavan Disease Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug) - Canavan Disease, Infantile | Phase 2 | Withdrawn | Assistance Publique - Hôpitaux de Paris | 2009-09-01 | 2015-04-21 | |
| ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases ALD-101 - Inherited Metabolic Diseases, Lysosomal Storage Disorders | Phase 3 | Terminated | Aldagen | 40 | 2008-03-01 | 2014-07-08 |
| Oral Glyceryl Triacetate (GTA) in Newborns With Canavan GTA (Glyceryl triacetate), GTA glyceryl triacetate - Canavan Disease | Not applicable | Unknown | Shaare Zedek Medical Center | 1 | 2008-03-01 | 2011-06-21 |
| GTA-Glyceryltriacetate for Canavan Disease GTA: Glyceryltriacetate - Infantile Canavan Disease, Deficiency Disease, Aspartoacylase | Phase 1 | Unknown | Sheba Medical Center | 5 | 2006-01-01 | 2006-08-15 |
| Canavan-Single Patient IND rAAV9-CB6-AspA - Canavan Disease | No longer available | University of Florida | 2023-08-23 |
Leukodystrophy, Globoid Cell (4) · Leukodystrophy, Metachromatic (4) · Leukoencephalopathies (4) · Adrenoleukodystrophy (3) · Mucopolysaccharidoses (3) · Peroxisomal Disorders (3) · Xanthomatosis, Cerebrotendinous (3) · Aicardi-Goutieres syndrome (2) · Alexander Disease (2) · Alexanders leukodystrophy (2) · Allan-Herndon-Dudley syndrome (2) · Attention Deficit Disorder with Hyperactivity (2) · CADASIL (2) · Charcot-Marie-Tooth Disease (2)
Brain Diseases (13) · Brain Diseases, Metabolic (13) · Brain Diseases, Metabolic, Inborn (13) · Central Nervous System Diseases (13) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Demyelinating Diseases (13) · Genetic Diseases, Inborn (13) · Hereditary Central Nervous System Demyelinating Diseases (13) · Heredodegenerative Disorders, Nervous System (13) · Metabolic Diseases (13) · Nervous System Diseases (13) · Neurodegenerative Diseases (13) · Nutritional and Metabolic Diseases (13) · Metabolism, Inborn Errors (12) · Leukoencephalopathies (9)
Triacetin (2) · Levetiracetam (1) · Pharmaceutical Preparations (1) · Prednisone (1) · gluconic acid (1)
Aspa Therapeutics (2) · Children's Hospital of Philadelphia (2) · Aldagen (1) · Assistance Publique - Hôpitaux de Paris (1) · McGill University Health Centre/Research Institute of the McGill University Health Centre (1) · Myrtelle Inc. (1) · NYU Langone Health (1) · RTI International (1) · Shaare Zedek Medical Center (1) · Sheba Medical Center (1) · University of Florida (1)
United States (9) · Germany (2) · Israel (2) · Canada (1)
Adeline Vanderver, MD (2) · Barry J Byrne, MD (1) · Caroline Sevin, MD, PhD (1) · Curt Scharfe, MD, PhD (1) · Florian Eichler, MD (1) · Gheona Alterescu, M.D. (1) · Guillaume Sillon, MSc (1) · James Hinson, MD (1) · Joanne Kurtzberg, MD (1) · Patrick Aubourg, MD, PhD (1) · Robert Lober, MD, PhD (1) · Yair Anikster, MD PI (1)
PMID 12064356, PMID 15784740, PMID 16002461, PMID 16194720, PMID 21474353, PMID 31315600, PMID 32342562, PMID 32749716, PMID 35142618, PMID 40957959
U01NS106845 (1), U54NS115052 (1)