11 completed, 7 recruiting, 3 active not recruiting, 2 terminated. 10 Phase 1, 8 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 10 | Completed 7, Active not recruiting 1, Not yet recruiting 1, Recruiting 1 |
| Phase 2 | 8 | Completed 6, Active not recruiting 1, Not yet recruiting 1 |
| Phase 3 | 1 | Completed 1 |
Follow Batten Disease: new trials by email Literature Review of Batten Disease trials Research Report on Batten 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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 | |
| 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 |
| Natural History Study of Batten Disease Batten Disease, Neuronal Ceroid Lipofuscinosis CLN6 | Terminated | Emily de los Reyes | 10 | 2021-04-27 | 2025-07-30 | |
| 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 | |
| Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer AT-GTX-501 - CLN6, Batten Disease | Active not recruiting | Emily de los Reyes | 10 | 2020-01-24 | 2025-10-02 | |
| Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease CLN2 Treatment - Batten Disease, CLN2 | Enrolling by invitation | Jessica Scherr | 30 | 2018-12-01 | 2025-10-24 | |
| Gene Therapy for Children With CLN3 Batten Disease Low dose CLN-301, High dose CLN-301 - CLN3, Batten Disease | Phase 1 / Phase 2 | Active not recruiting | Neela Therapeutics | 7 | 2018-11-13 | 2026-06-10 |
| Investigations of Juvenile Neuronal Ceroid Lipofuscinosis Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), Batten Disease | Recruiting | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 300 | 2017-11-27 | 2026-10-05 | |
| Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae Natural history - Batten Disease, CLN6 | Active not recruiting | Emily de los Reyes | 30 | 2017-01-01 | 2025-07-30 | |
| A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device - Jansky-Bielschowsky Disease, Batten Disease | Phase 2 | Completed | BioMarin Pharmaceutical | 14 | 2016-01-22 | 2025-02-17 |
| An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease BMN 190, Intracerebroventricular (ICV) access device - Jansky-Bielschowsky Disease, Batten Disease | Phase 1 / Phase 2 | Completed | BioMarin Pharmaceutical | 23 | 2015-02-01 | 2022-08-24 |
| 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 |
| Inherited Retinal Degenerative Disease Registry Eye Diseases Hereditary, Retinal Disease | Recruiting | Foundation Fighting Blindness | 20000 | 2014-06-01 | 2026-05-19 | |
| A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease BMN 190 - Jansky-Bielschowsky Disease, Batten Disease | Phase 1 / Phase 2 | Completed | BioMarin Pharmaceutical | 24 | 2013-09-01 | 2019-03-08 |
| 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 |
| Longitudinal Study of Neurodegenerative Disorders Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease | Recruiting | University of Pittsburgh | 1500 | 2012-01-11 | 2026-02-09 | |
| Collection of Cerebrospinal Fluid in Healthy Children Group 1 - Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease | Terminated | Weill Medical College of Cornell University | 32 | 2011-12-01 | 2016-05-04 | |
| Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies, AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies - Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis | Phase 1 | Completed | Weill Medical College of Cornell University | 12 | 2010-08-19 | 2021-02-02 |
| AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis AAVrh.10CUCLN2 - Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease | Phase 1 / Phase 2 | Completed | Weill Medical College of Cornell University | 8 | 2010-04-15 | 2020-08-31 |
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Neuronal Ceroid-Lipofuscinoses (25) · Leukodystrophy, Globoid Cell (4) · Adrenoleukodystrophy (3) · Gaucher Disease (3) · Leukodystrophy, Metachromatic (3) · Niemann-Pick Diseases (3) · Sandhoff Disease (3) · Tay-Sachs Disease (3) · Fucosidosis (2) · Lysosomal Storage Diseases (2) · Pelizaeus-Merzbacher Disease (2) · Wolman Disease (2) · alpha-Mannosidosis (2) · Abetalipoproteinemia (1) · Achromatopsia 3 (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (25) · Genetic Diseases, Inborn (25) · Heredodegenerative Disorders, Nervous System (25) · Lipid Metabolism Disorders (25) · Lipid Metabolism, Inborn Errors (25) · Lipidoses (25) · Nervous System Diseases (25) · Nutritional and Metabolic Diseases (25) · Metabolic Diseases (24) · Metabolism, Inborn Errors (24) · Neurodegenerative Diseases (24) · Brain Diseases (6) · Central Nervous System Diseases (6) · Brain Diseases, Metabolic (5) · Demyelinating Diseases (5)
cerliponase alfa (3) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Palliative Care (1) · Stem Cell Transplantation (1)
Weill Medical College of Cornell University (6) · BioMarin Pharmaceutical (3) · Emily de los Reyes (3) · Beyond Batten Disease Foundation (1) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (1) · Foundation Fighting Blindness (1) · Jessica Scherr (1) · Joanne Kurtzberg, MD (1) · Masonic Cancer Center, University of Minnesota (1) · Neela Therapeutics (1) · New York Medical College (1) · New York Stem Cell Foundation Research Institute (1) · The Charlotte and Gwenyth Gray Foundation (1) · University of Pittsburgh (1) · University of Rochester (1)
United States (23) · Germany (4) · Italy (3) · United Kingdom (3)
Ronald G. Crystal, MD (3) · Ronald G Crystal, MD (2) · An N Dang Do, M.D. (1) · An N. Dang Do, MD, PhD (1) · Angela Schulz, MD, PhD (1) · David Jacoby (1) · Gary D. Clark, MD (1) · Jessica Scherr, PhD (1) · Joanne Kurtzberg, MD (1) · Jonathan W Mink, MD PhD (1) · Kathrin C Meyer, PhD (1) · Laura Andres-Martin, PhD (1)
PMID 18473686, PMID 29688815, PMID 33980287, PMID 37171121, PMID 38101904, PMID 38776275, PMID 39745261, PMID 17679671, PMID 21586746, PMID 33550636, PMID 34913584, PMID 35772852, PMID 36461157, PMID 37035740
R01NS061848 (1), U54NS065768 (1), UL1TR000457-06 (1)