6 completed, 1 active not recruiting, 1 not yet recruiting. 8 Phase 2, 6 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 8 | Completed 6, Active not recruiting 1, Not yet recruiting 1 |
| Phase 1 | 6 | Completed 4, 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 |
| 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 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 |
| 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 |
| 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 |
| 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 |
| Stem Cell Transplant for Inborn Errors of Metabolism Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin - Adrenoleukodystrophy, Metachromatic Leukodystrophy | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 135 | 1995-01-01 | 2017-12-28 |
Neuronal Ceroid-Lipofuscinoses (8) · Adrenoleukodystrophy (1) · Fucosidosis (1) · Gangliosidosis, GM1 (1) · Gaucher Disease (1) · Leukodystrophy, Globoid Cell (1) · Leukodystrophy, Metachromatic (1) · Niemann-Pick Diseases (1) · Sandhoff Disease (1) · Tay-Sachs Disease (1) · Wolman Disease (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Genetic Diseases, Inborn (8) · Heredodegenerative Disorders, Nervous System (8) · Lipid Metabolism Disorders (8) · Lipid Metabolism, Inborn Errors (8) · Lipidoses (8) · Metabolic Diseases (8) · Metabolism, Inborn Errors (8) · Nervous System Diseases (8) · Neurodegenerative Diseases (8) · Nutritional and Metabolic Diseases (8) · Adrenal Gland Diseases (1) · Adrenal Insufficiency (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1)
cerliponase alfa (3) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Stem Cell Transplantation (1)
BioMarin Pharmaceutical (3) · Beyond Batten Disease Foundation (1) · Masonic Cancer Center, University of Minnesota (1) · Neela Therapeutics (1) · The Charlotte and Gwenyth Gray Foundation (1) · Weill Medical College of Cornell University (1)
United States (8) · Germany (3) · Italy (3) · United Kingdom (3)
An N. Dang Do, MD, PhD (1) · David Jacoby (1) · Gary D. Clark, MD (1) · Kathrin C Meyer, PhD (1) · Paul Orchard, MD (1) · Ronald Crystal, MD (1)
PMID 29688815, PMID 33980287, PMID 38101904, PMID 38776275, PMID 21586746, PMID 37035740, PMID 37171121