32 completed, 21 recruiting, 11 terminated, 7 withdrawn. 22 Phase 2, 13 Not applicable, 11 Phase 1, 11 Phase 3, 1 Phase 4.
Follow Peroxisomal Disorders: new trials by email Literature Review of Peroxisomal Disorders trials Research Report on Peroxisomal Disorders
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 |
|---|---|---|---|---|---|---|
| IT and IV Lentiviral Gene Therapy for X-ALD Intrathecal and intravenous LV gene therapy - X-linked Adrenoleukodystrophy | Not applicable | Not yet recruiting | Shenzhen Geno-Immune Medical Institute | 30 | 2027-06-01 | 2026-11-02 |
| Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena) Cushing s Syndrome, Cushing Disease | Recruiting | Azienda Ospedaliero-Universitaria di Modena | 2000 | 2026-07-09 | 2026-11-09 | |
| Ilaris NIS in Korea Ilaris - Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS) | Recruiting | Novartis Pharmaceuticals | 25 | 2025-03-29 | 2026-05-19 | |
| Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy Spinal cord stimulation - Adrenomyeloneuropathy Without Cerebral Involvement | Not applicable | Recruiting | Third Military Medical University | 10 | 2025-02-10 | 2025-01-28 |
| A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel No Intervention - Cerebral Adrenoleukodystrophy (CALD) | Recruiting | Genetix Biotherapeutics Inc. | 120 | 2024-03-27 | 2025-07-02 | |
| Disease Progression in Women With X-linked Adrenoleukodystrophy X-linked Adrenoleukodystrophy | Recruiting | Minoryx Therapeutics, S.L. | 40 | 2024-01-02 | 2024-01-31 | |
| Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder Zellweger Spectrum Disorder | Recruiting | McGill University Health Centre/Research Institute of the McGill University Health Centre | 30 | 2023-12-18 | 2026-01-21 | |
| Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) PXL065 - ALD (Adrenoleukodystrophy) | Phase 2 | Withdrawn | Poxel SA | 2023-09-01 | 2024-12-16 | |
| Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD) PXL770 - Adrenomyeloneuropathy | Phase 2 | Withdrawn | Poxel SA | 2023-09-01 | 2024-12-16 | |
| Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked Adrenoleukodystrophy brainQuant module of brainTale-care platform - X-linked Adrenoleukodystrophy | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 200 | 2023-09-01 | 2023-07-03 | |
| Registry of X-linked Adrenoleukodystrophy X-linked Adrenoleukodystrophy | Recruiting | Beijing Tiantan Hospital | 200 | 2023-07-20 | 2023-07-11 | |
| A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to Assess Safety, Tolerability, and Pharmacokinetics PPI-1011, Placebo - Rhizomelic Chondrodysplasia Punctata | Phase 1 | Completed | MED-LIFE DISCOVERIES LP | 56 | 2023-06-28 | 2026-10-09 |
| Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy Pramipexole, Placebo - Adrenoleukodystrophy, Restless Legs Syndrome | Phase 4 | Active not recruiting | Massachusetts General Hospital | 24 | 2023-04-01 | 2026-07-01 |
| Home Exercise for Individuals With Neurodegenerative Disease Home exercise - Neurodegenerative Diseases, Leukodystrophy | Not applicable | Recruiting | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | 30 | 2023-02-13 | 2026-09-16 |
| 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 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD) SMART-ALD, Waiting list - X-linked Adrenoleukodystrophy | Not applicable | Completed | Leipzig University Medical Center | 68 | 2022-05-20 | 2024-03-13 |
| Geranylgeraniol Supplementation in Patients With Mevalonate Kinase Deficiency geranylgeraniol - Mevalonate Kinase Deficiency | Not applicable | Unknown | University Hospital, Motol | 6 | 2022-01-25 | 2024-07-12 |
| Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children Xlsma, DMD | Withdrawn | Children's Hospital of Fudan University | 2021-09-30 | 2021-08-11 | ||
| Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia Blood sample collection - Adrenoleukodystrophy, Adrenomyeloneuropathy | Recruiting | Assistance Publique - Hôpitaux de Paris | 100 | 2021-08-30 | 2025-09-24 |
Showing 20 of 86. Sign in to see all of them.
Adrenoleukodystrophy (63) · Leukodystrophy, Metachromatic (21) · Leukodystrophy, Globoid Cell (17) · Mucopolysaccharidosis I (12) · Metabolism, Inborn Errors (11) · Zellweger Syndrome (11) · Mucopolysaccharidosis II (8) · Mucopolysaccharidosis VI (8) · Rare Diseases (8) · Sphingolipidoses (8) · Wolman Disease (8) · alpha-Mannosidosis (8) · Fucosidosis (7) · Anemia, Sickle Cell (6)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (86) · Nutritional and Metabolic Diseases (86) · Genetic Diseases, Inborn (83) · Brain Diseases (81) · Metabolic Diseases (81) · Brain Diseases, Metabolic (79) · Nervous System Diseases (79) · Central Nervous System Diseases (78) · Brain Diseases, Metabolic, Inborn (77) · Metabolism, Inborn Errors (75) · Neurologic Manifestations (68) · Heredodegenerative Disorders, Nervous System (67) · Intellectual Disability (66) · Neurobehavioral Manifestations (66)
Alemtuzumab (5) · Stem Cell Transplantation (5) · Busulfan (3) · Cholic Acid (3) · Cyclophosphamide (3) · Lorenzo's oil (3) · Melphalan (3) · canakinumab (3) · trierucate (3) · Bone Marrow Transplantation (2) · Clofarabine (2) · Cyclosporine (2) · GC 1 compound (2) · Hematopoietic Stem Cell Transplantation (2) · Hydroxyurea (2)
Masonic Cancer Center, University of Minnesota (8) · Genetix Biotherapeutics Inc. (5) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (3) · McGill University Health Centre/Research Institute of the McGill University Health Centre (3) · Novartis Pharmaceuticals (3) · Pujol, Aurora, M.D. (3) · Assistance Publique - Hôpitaux de Paris (2) · Children's Hospital of Philadelphia (2) · Joanne Kurtzberg, MD (2) · Leipzig University Medical Center (2) · MED-LIFE DISCOVERIES LP (2) · Minoryx Therapeutics, S.L. (2) · Mirum Pharmaceuticals, Inc. (2) · NeuroVia, Inc. (2) · Paul Szabolcs (2)
United States (49) · France (10) · Germany (9) · Netherlands (9) · Italy (7) · Canada (6) · Spain (6) · United Kingdom (6) · China (5) · Argentina (4) · Australia (3) · Taiwan (2) · Belgium (1) · Brazil (1) · Chile (1)
Paul Orchard, MD (6) · Joanne Kurtzberg, MD (3) · Adeline Vanderver, MD (2) · Gerald V Raymond, M.D. (2) · John Henderson, MD (2) · Nancy E Braverman, MD, MS (2) · Paul Szabolcs, MD (2) · Adrian Thrasher, MD, PhD (1) · Andrea Biondi, MD, PhD (1) · Arnold W. Strauss (1) · Biljana Horn, MD (1)
PMID 21586746, PMID 39383458, PMID 39383459, PMID 11231629, PMID 11399225, PMID 19833552, PMID 19892975, PMID 22911730, PMID 23845948, PMID 23966770, PMID 26121453, PMID 27479926, PMID 28482144, PMID 28644367, PMID 28976817, PMID 31077039, PMID 31214256, PMID 31315600, PMID 32266384, PMID 32342562, PMID 32634238, PMID 32749716, PMID 32750127, PMID 34534157, PMID 34781360, PMID 35106698, PMID 35142618, PMID 35778568, PMID 36681445, PMID 37318239, PMID 37541626, PMID 37782421, PMID 38151399, PMID 41030609, PMID 41667276
2014 (1), 2015 (1), 2018 (1), K23NS087151 (1), R37DK020407 (1), U01NS106845 (1), U54NS115052 (1)