A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02485899|
Recruitment Status : Active, not recruiting
First Posted : June 30, 2015
Last Update Posted : May 31, 2018
|Condition or disease||Intervention/treatment||Phase|
|Jansky-Bielschowsky Disease Batten Disease Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 CLN2 Disease CLN2 Disorder||Biological: BMN 190 Device: Intraventricular Access Device||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||23 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease|
|Actual Study Start Date :||February 2015|
|Estimated Primary Completion Date :||July 2020|
|Estimated Study Completion Date :||January 2021|
|Experimental: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)||
Biological: BMN 190
300 mg ICV infusion administered every other week for up to 240 weeks
Device: Intraventricular Access Device
Surgical implantation of an MRI compatible ICV access device in the lateral ventricle of the right hemisphere is required for administration of study drug.
- Long Term Safety as assessed by analysis of adverse events. [ Time Frame: up to 240 weeks ]Long term safety of BMN 190 administered to subjects with CLN2 disease via an implanted intracerebroventricular (ICV) reservoir and cannula as assessed by analysis of adverse events.
- Motor and Language Changes [ Time Frame: up to 240 weeks ]Change in motor and language (ML) subscales of the CLN2 disease rating scale in patients with CLN2 following administration of 300 mg every other week of BMN 190.
- Quantitative Assessment of Magnetic Resonance Imaging [ Time Frame: up to 240 weeks ]Changes in quantitative assessment of MRI.
- CLN2 Disease Scale Score [ Time Frame: up to 240 weeks ]Changes in the CLN2 disease scale total score
- Quality of Life Changes [ Time Frame: up to 240 weeks ]Changes in the quality of life with long-term use of BMN 190
- Age-Appropriateness of Developmental Milestone [ Time Frame: up to 240 weeks ]Evaluation of age-appropriateness of developmental Milestones
- Impact of Treatment on BioMarkers [ Time Frame: up to 240 weeks ]Evaluation of impact of treatment on disease-related biomarkers from CSF and blood
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02485899
|United States, Ohio|
|Columbus, Ohio, United States|
|London, United Kingdom|
|Study Director:||Temitayo Ajayi, MD||BioMarin Pharmaceutical|