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Adult Neuronal Ceroid Lipofuscinosis - 21 Studies Found

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
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