20 completed, 11 recruiting, 8 active not recruiting, 4 withdrawn. 17 Phase 1, 13 Phase 2, 2 Not applicable, 2 Phase 3, 1 Phase 4.
Follow Neuronal Ceroid-Lipofuscinoses: new trials by email Literature Review of Neuronal Ceroid-Lipofuscinoses trials Research Report on Neuronal Ceroid-Lipofuscinoses
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 |
|---|---|---|---|---|---|---|
| Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3 PLX-200, Placebo - Juvenile Neuronal Ceroid Lipofuscinosis | Phase 3 | Withdrawn | Polaryx Therapeutics, Inc. | 2026-11-01 | 2026-08-19 | |
| Gene Therapy Trial for CLN6 Batten Disease scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) - CLN6, Batten Disease | Phase 1 / Phase 2 | Not yet recruiting | The Charlotte and Gwenyth Gray Foundation | 12 | 2026-08-01 | 2026-05-14 |
| Italian NCL Registry: a Registry for NCL as an Integration Tool for Future Therapeutic Strategies Neuronal Ceroid Lipofuscinosis | Recruiting | IRCCS Fondazione Stella Maris | 50 | 2024-07-19 | 2026-03-27 | |
| A Registrational Study of TTX-381 to Treat Vision Loss in CLN2 Disease TTX-381 - Neuronal Ceroid Lipofuscinosis Type 2 | Phase 1 / Phase 2 | Recruiting | Tern Therapeutics, LLC | 26 | 2023-05-17 | 2026-10-14 |
| Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome | Completed | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 73 | 2023-01-23 | 2026-10-13 | |
| NYSCF Scientific Discovery Biobank Biological Sample Collection - ALS, Amyotrophic Lateral Sclerosis | Recruiting | New York Stem Cell Foundation Research Institute | 10000 | 2022-11-10 | 2025-03-03 | |
| Caregiving Networks Across Disease Context and the Life Course Inherited Metabolic Disorders, Undiagnosed Diseases | Recruiting | National Human Genome Research Institute (NHGRI) | 2800 | 2022-09-08 | 2026-09-15 | |
| 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 | |
| An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease Miglustat 100 milligrams (mg) Oral Capsule - Batten Disease | Phase 1 / Phase 2 | Completed | Beyond Batten Disease Foundation | 6 | 2022-03-10 | 2025-09-09 |
| Gene Therapy Study for Children With CLN5 Batten Disease NGN-101 - Neuronal Ceroid Lipofuscinosis CLN5 | Phase 1 / Phase 2 | Active not recruiting | Neurogene Inc. | 6 | 2022-01-31 | 2024-08-12 |
| Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2 Cerliponase Alfa - Neuronal Ceroid Lipofuscinosis Type 2 | Phase 1 / Phase 2 | Active not recruiting | David L Rogers, MD | 5 | 2021-11-01 | 2026-04-30 |
| Study for the Treatment for CLN7 Disease AAV9/CLN7 - CLN7 | Phase 1 | Active not recruiting | Benjamin Greenberg | 4 | 2021-05-04 | 2025-12-31 |
| Natural History Study of Batten Disease Batten Disease, Neuronal Ceroid Lipofuscinosis CLN6 | Terminated | Emily de los Reyes | 10 | 2021-04-27 | 2025-07-30 | |
| An Observational Study in Children With CLN2 Batten Disease Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | Withdrawn | REGENXBIO Inc. | 2021-03-31 | 2021-11-01 | ||
| A Retrospective, Natural History Study in Children With CLN2 Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | Withdrawn | REGENXBIO Inc. | 2021-03-31 | 2021-11-01 | ||
| N-3 Polyunsaturated Fatty Acids Prevent Postoperative Recurrence of Crohn's Disease azathioprine, infliximab, N-3 Polyunsaturated Fatty Acids - Inflammatory Bowel Diseases, Crohn Disease | Not applicable | Unknown | Sixth Affiliated Hospital, Sun Yat-sen University | 236 | 2021-02-13 | 2021-02-21 |
| Cerliponase Alfa Observational Study in the US Cerliponase Alfa, Administration Kit - Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Active not recruiting | BioMarin Pharmaceutical | 35 | 2020-08-19 | 2026-06-30 | |
| Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses Neuronal Ceroid-Lipofuscinoses | Completed | IRCCS Fondazione Stella Maris | 8 | 2020-08-01 | 2022-04-11 | |
| Using NPT to Evaluate Providing PPC as ELNEC-PPC WBT for Nurses end-of-life nursing education consortium-pediatric palliative care as web based-training plus usual care - Cancer, Cardiac Anomaly | Not applicable | Unknown | Altoosi University College | 172 | 2020-07-01 | 2020-07-27 |
| Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database Natural History - Neuronal Ceroid Lipofuscinosis, Batten Disease | Recruiting | Universitätsklinikum Hamburg-Eppendorf | 500 | 2020-04-08 | 2021-10-29 |
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Leukodystrophy, Metachromatic (5) · Adrenoleukodystrophy (4) · Gaucher Disease (4) · Leukodystrophy, Globoid Cell (4) · Niemann-Pick Diseases (3) · Sandhoff Disease (3) · Tay-Sachs Disease (3) · Wolman Disease (3) · Anemia, Diamond-Blackfan (2) · Ataxia with vitamin E deficiency (2) · Charcot-Marie-Tooth Disease (2) · Cystic Fibrosis (2) · Cystinosis (2) · Diabetes Mellitus (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (49) · Genetic Diseases, Inborn (49) · Heredodegenerative Disorders, Nervous System (49) · Lipid Metabolism Disorders (49) · Lipid Metabolism, Inborn Errors (49) · Lipidoses (49) · Nervous System Diseases (49) · Nutritional and Metabolic Diseases (49) · Metabolic Diseases (48) · Metabolism, Inborn Errors (48) · Neurodegenerative Diseases (48) · Central Nervous System Diseases (11) · Brain Diseases (10) · Brain Diseases, Metabolic (8) · Brain Diseases, Metabolic, Inborn (7)
cerliponase alfa (5) · Antilymphocyte Serum (1) · Azathioprine (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Cysteamine (1) · Fatty Acids, Omega-3 (1) · Gemfibrozil (1) · Infliximab (1) · Mycophenolic Acid (1) · Palliative Care (1) · Stem Cell Transplantation (1)
Weill Medical College of Cornell University (6) · BioMarin Pharmaceutical (4) · Emily de los Reyes (4) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (3) · IRCCS Fondazione Stella Maris (2) · REGENXBIO Inc. (2) · StemCells, Inc. (2) · University of Rochester (2) · Altoosi University College (1) · Benjamin Greenberg (1) · Beyond Batten Disease Foundation (1) · Centre Hospitalier Universitaire de Liege (1) · Children's Hospital of Orange County (1) · David L Rogers, MD (1) · Foundation Fighting Blindness (1)
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Ronald G. Crystal, MD (3) · Ronald G Crystal, MD (2) · Amean A Yaser, Ph.D (1) · An N Dang Do, M.D. (1) · An N. Dang Do, MD, PhD (1) · Angela Schulz, MD, PhD (1) · Anil B Mukherjee, M.D. (1) · Benjamin Greenberg, MD (1) · David Jacoby (1) · David Rogers, MD (1) · Effie Albanis, MD (1) · Erika F Augustine, MD (1)
PMID 18473686, PMID 29688815, PMID 33980287, PMID 37171121, PMID 38101904, PMID 38776275, PMID 39745261, PMID 17679671, PMID 21586746, PMID 23581634, PMID 24014509, PMID 24997880, PMID 27105763, PMID 31184505, PMID 33550636, PMID 34913584, PMID 35772852, PMID 36367759, PMID 36461157, PMID 37035740, PMID 37614821, PMID 37942487
2025 (1), R01NS061848 (1), U54NS065768 (1), UL1TR000457-06 (1)