Batten Disease Clinical Trial
Official title:
A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 144 weeks, in patients with CLN2. The study is designed to assess disease progression in CLN2 patients treated with BMN 190 compared to natural history data from untreated historical controls.
BMN 190 is a recombinant form of human tripeptidyl peptidase 1 (TPP1), the enzyme deficient in patients with CLN2 diseases (also known as classical late-infantile CLN2, cLINCL, or Jansky-Bielschowsky disease), a form of Batten Disease. As an enzyme replacement therapy (ERT), BMN 190 is designed to help restore TPP1 enzyme activity. BMN 190 is designed to reduce the progressive, pathologic accumulation of lysosomal storage material. 190-203 is a Phase 2 open-label, multicenter study that will evaluate the safety, tolerability, and efficacy of BMN 190 in pediatric patients < 18 years of age with CLN2 disease. Study drug dosing will be determined by the patient's age and administered via intracerebroventricular (ICV) infusion every other week (qow), for a duration of 144 weeks. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT02254863 -
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
|
Phase 1 | |
| Completed |
NCT01907087 -
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
|
Phase 1/Phase 2 | |
| Recruiting |
NCT03307304 -
Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
|
||
| Active, not recruiting |
NCT03770572 -
Gene Therapy for Children With CLN3 Batten Disease
|
Phase 1/Phase 2 | |
| Recruiting |
NCT06203106 -
NYSCF Scientific Discovery Biobank
|
||
| Completed |
NCT00151268 -
Genotype - Phenotype Correlations of LINCL
|
N/A | |
| Recruiting |
NCT02435940 -
Inherited Retinal Degenerative Disease Registry
|
||
| Completed |
NCT01035424 -
Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis
|
||
| Completed |
NCT00151216 -
Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
|
Phase 1 | |
| Active, not recruiting |
NCT04273243 -
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
|
||
| Recruiting |
NCT03285425 -
Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae
|
||
| Completed |
NCT00176904 -
Stem Cell Transplant for Inborn Errors of Metabolism
|
Phase 2/Phase 3 | |
| Recruiting |
NCT03333200 -
Longitudinal Study of Neurodegenerative Disorders
|
||
| Completed |
NCT02485899 -
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
|
Phase 1/Phase 2 | |
| Active, not recruiting |
NCT05174039 -
An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of CLN3 Disease
|
Phase 1/Phase 2 | |
| Terminated |
NCT01698229 -
Collection of Cerebrospinal Fluid in Healthy Children
|
N/A | |
| Enrolling by invitation |
NCT03862274 -
Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease
|
||
| Recruiting |
NCT01873924 -
Clinical and Neuropsychological Investigations in Batten Disease
|
||
| Completed |
NCT01161576 -
Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)
|
Phase 1 | |
| Recruiting |
NCT04613089 -
Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
|