Batten Disease Clinical Trial
Official title:
A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
The Phase 1/2 study (190-201) evaluated the efficacy and safety of a 300 mg dose of BMN 190 administered every other week (qow) to patients with CLN2. The dose and regimen for this study (190-202) are based on the results of the 190-201 study. The rationale for this phase 2 extension study is to provide patients who complete the 190-201 study with the option to continue BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.
BMN 190 is a recombinant form of human tripeptidyl peptidase 1 (TPP1), the enzyme deficient in patients with CLN2 disease (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. The Phase 1/2 study (190-201) evaluated the efficacy and safety of a 300 mg dose of BMN 190 administered every other week (qow) to patients with CLN2. The dose and regimen for this study (190-202) are based on the results of the 190-201 study. The rationale for this phase 2 extension study is to provide patients who complete the 190-201 study with the option to continue BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy. ;
| 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 |
NCT02678689 -
A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
|
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
|