A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
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|ClinicalTrials.gov Identifier: NCT02485899|
Recruitment Status : Completed
First Posted : June 30, 2015
Last Update Posted : February 11, 2021
|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|
|Actual Primary Completion Date :||December 10, 2020|
|Actual Study Completion Date :||December 10, 2020|
|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
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|
|Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|
|Hamburg, Germany, 20246|
|Children's Hospital Bambino Gesù,IRCCS|
|Rome, Piazza, Italy, 00165|
|Great Ormond Street Childrens Hospital|
|London, United Kingdom, WC1N 3JH|
|Study Director:||Medical Monitor, MD||BioMarin Pharmaceutical|