4 completed, 2 terminated, 2 withdrawn, 1 recruiting. 10 Phase 1, 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 10 | Completed 4, Terminated 2, Withdrawn 2, Recruiting 1, Unknown 1 |
| Phase 2 | 5 | Terminated 2, Completed 1, Unknown 1, Withdrawn 1 |
Follow Adrenoleukodystrophy: new trials by email Literature Review of Adrenoleukodystrophy trials Research Report on Adrenoleukodystrophy
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 to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) SBT101 Treatment - AMN, AMN Gene Mutation | Phase 1 / Phase 2 | Terminated | SwanBio Therapeutics, Inc. | 9 | 2022-11-17 | 2026-08-26 |
| Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy Sobetirome (NV1205) - X-Linked Adrenoleukodystrophy | Phase 1 / Phase 2 | Withdrawn | NeuroVia, Inc. | 2018-08-01 | 2019-03-07 | |
| Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Thiotepa--single daily dose, Thiotepa--escalated dose - Bone Marrow Failure Syndrome, Thalassemia | Phase 1 / Phase 2 | Completed | University of Florida | 6 | 2018-06-15 | 2023-09-15 |
| A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers OP-101 - Healthy | Phase 1 | Completed | Orpheris, Inc. | 8 | 2018-03-30 | 2018-09-17 |
| A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy vitamin D3 - X-linked Adrenoleukodystrophy | Phase 1 | Completed | Stanford University | 21 | 2016-11-21 | 2022-06-09 |
| Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy transduced CD34+ hematopoietic stem cell - Metachromatic Leukodystrophy, Adrenoleukodystrophy | Phase 1 / Phase 2 | Unknown | Shenzhen Second People's Hospital | 50 | 2015-01-01 | 2022-05-31 |
| UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells DUOC-01 - Adrenoleukodystrophy, Batten Disease | Phase 1 | Recruiting | Joanne Kurtzberg, MD | 40 | 2014-09-01 | 2026-09-03 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) Sobetirome - X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy | Phase 1 | Withdrawn | Thomas S. Scanlan | 2013-04-01 | 2014-02-14 | |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
Leukodystrophy, Metachromatic (4) · Leukodystrophy, Globoid Cell (3) · Mucopolysaccharidosis I (3) · Anemia, Diamond-Blackfan (2) · Brain Diseases, Metabolic, Inborn (2) · Demyelinating Diseases (2) · Metabolism, Inborn Errors (2) · Mucopolysaccharidosis II (2) · Neuronal Ceroid-Lipofuscinoses (2) · Niemann-Pick Diseases (2) · Sandhoff Disease (2) · Tay-Sachs Disease (2) · alpha-Mannosidosis (2) · Adrenal Insufficiency (1)
Adrenal Gland Diseases (10) · Brain Diseases (10) · Central Nervous System Diseases (10) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · Endocrine System Diseases (10) · Genetic Diseases, Inborn (10) · Genetic Diseases, X-Linked (10) · Intellectual Disability (10) · Nervous System Diseases (10) · Neurobehavioral Manifestations (10) · Neurologic Manifestations (10) · Nutritional and Metabolic Diseases (10) · X-Linked Intellectual Disability (10) · Adrenal Insufficiency (9) · Brain Diseases, Metabolic (9)
GC 1 compound (2) · Cholecalciferol (1) · Stem Cell Transplantation (1) · Vitamin D (1)
Joanne Kurtzberg, MD (1) · NeuroVia, Inc. (1) · New York Medical College (1) · Orpheris, Inc. (1) · Shenzhen Second People's Hospital (1) · Stanford University (1) · SwanBio Therapeutics, Inc. (1) · Talaris Therapeutics Inc. (1) · Thomas S. Scanlan (1) · University of Florida (1)
United States (8) · Argentina (1) · Australia (1) · Chile (1) · China (1) · Colombia (1) · France (1) · Netherlands (1) · Russia (1) · Ukraine (1) · United Kingdom (1)
Biljana Horn, MD (1) · David Koeller, MD (1) · GuangFu Chen, M.D.,Ph.D (1) · Hua Jiang, M.D,Ph.D (1) · Jiacai Zhuo (1) · Joanne Kurtzberg, MD (1) · John Henderson, MD (1) · Keith Van Haren, MD (1) · Mitchell S Cairo, MD (1) · Qizhou Lian, M.D.,Ph.D. (1) · Stephen Huhn, MD (1) · Suzanne T Ildstad, MD (1) · Xin Du, M.D.,Ph.D. (1)
PMID 11231629, PMID 11399225, PMID 19892975, PMID 23845948, PMID 23966770
K23NS087151 (1)