7 completed, 1 active not recruiting, 1 not yet recruiting, 1 recruiting. 10 Phase 1, 6 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 10 | Completed 7, Active not recruiting 1, Not yet recruiting 1, Recruiting 1 |
| Phase 2 | 6 | Completed 4, Active not recruiting 1, Not yet recruiting 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 |
| 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 |
| 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 |
| 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 |
| Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis AAV2CUhCLN2 (3x10^12 particle units) - Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis | Phase 1 | Completed | Weill Medical College of Cornell University | 10 | 2004-06-01 | 2020-07-24 |
Neuronal Ceroid-Lipofuscinoses (10) · Adrenoleukodystrophy (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Metachromatic (2) · Anemia, Aplastic (1) · Anemia, Diamond-Blackfan (1) · Brain Diseases, Metabolic, Inborn (1) · Fucosidosis (1) · Gaucher Disease (1) · Leukemia, Myeloid, Acute (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis II (1) · Mucopolysaccharidosis VI (1) · Myelodysplastic Syndromes (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · Genetic Diseases, Inborn (10) · Heredodegenerative Disorders, Nervous System (10) · Lipid Metabolism Disorders (10) · Lipid Metabolism, Inborn Errors (10) · Lipidoses (10) · Metabolic Diseases (10) · Nervous System Diseases (10) · Neurodegenerative Diseases (10) · Nutritional and Metabolic Diseases (10) · Metabolism, Inborn Errors (9) · Adrenal Gland Diseases (2) · Adrenal Insufficiency (2) · Brain Diseases (2) · Brain Diseases, Metabolic (2)
cerliponase alfa (2)
Weill Medical College of Cornell University (3) · BioMarin Pharmaceutical (2) · Beyond Batten Disease Foundation (1) · Joanne Kurtzberg, MD (1) · Neela Therapeutics (1) · New York Medical College (1) · The Charlotte and Gwenyth Gray Foundation (1)
United States (10) · Germany (2) · Italy (2) · United Kingdom (2)
An N. Dang Do, MD, PhD (1) · David Jacoby (1) · Gary D. Clark, MD (1) · Joanne Kurtzberg, MD (1) · Kathrin C Meyer, PhD (1) · Mitchell S Cairo, MD (1) · Ronald Crystal, MD (1) · Ronald G Crystal, MD (1) · Ronald G. Crystal, MD (1)
PMID 29688815, PMID 33980287, PMID 37171121, PMID 38101904, PMID 38776275, PMID 18473686, PMID 37035740, PMID 39745261
UL1TR000457-06 (1)