| Status | Study |
| Recruiting |
Study Name: Batten CLN6 Gene Therapy Condition: Batten Disease CLN6 Date: 2016-03-16 Interventions: Drug: scAVV9.CB.CLN6 Six subjects with diagnosis of CLN6 disease will receive scAVV9.CB.CLN6 administere |
| Enrolling by invitation |
Study Name: A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease Condition: Jansky-Bielschowsky Disease Batten Disease Late-Infantile Date: 2016-01-15 Interventions: Drug: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1) |
| Active, not recruiting |
Study Name: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease Condition: Jansky-Bielschowsky Disease Batten Disease Late-Infantile Date: 2015-04-24 Interventions: Biological: BMN 190 300 mg ICV infusion administered every other week for up to 240 weeks |
| Recruiting |
Study Name: Inherited Retinal Degenerative Disease Registry Condition: Eye Diseases Hereditary Retinal Disease Achromatopsia Date: 2015-04-03 |
| Recruiting |
Study Name: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Condition: Adrenoleukodystrophy Batten Disease Mucopolysaccharidosis Date: 2014-09-23 Interventions: Biological: DUOC-01 Intrathecal administration of DUOC-01 |
| Recruiting |
Study Name: Genetic Characterization of Movement Disorders and Dementias Condition: Ataxia Dystonia Parkinson's Disease Date: 2013-12-12 |
| Completed |
Study Name: Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities Condition: Neuronal Ceroid Lipofuscinosis Batten's Disease Date: 2013-09-25 |
| Completed |
Study Name: 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 Condition: Jansky-Bielschowsky Disease Batten Disease Late-Infantile Date: 2013-07-19 Interventions: Biological: BMN 190 30-300 mg ICV infusion administered every other week for at least 48 weeks. |
| Recruiting |
Study Name: Clinical and Neuropsychological Investigations in Batten Disease Condition: Neuronal Ceroid Lipofuscinosis Date: 2013-05-17 |
| Terminated |
Study Name: Collection of Cerebrospinal Fluid in Healthy Children Condition: Late Infantile Neuronal Ceroid Lipofuscinosis Batten Disease Date: 2012-08-15 Interventions: Other: Group 1 Collecting a small amount of cerebrospinal fluid (5 mL) from healthy children, ages 2 yrs |