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
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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 |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |

Arm | Intervention/treatment |
---|---|
Experimental: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1) |
Biological: BMN 190
300 mg ICV infusion administered every other week for up to 240 weeks
Other Names:
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

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Ages Eligible for Study: | 3 Years to 16 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Must have completed 48 weeks in Study 190-201.
- Is willing and able to provide written, signed informed consent. Or, in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorized representative, after the nature of the study has been explained, and prior to performance of research-related procedures.
- Males and females who are of reproductive age should practice true abstinence, defined as no sexual activity, during the study and for 6 months after the study has been completed (or withdrawal from the study). If sexually active and not practicing true abstinence, males and females of reproductive age must use a highly effective method of contraception while participating in the study.
- If female, of childbearing potential, must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests done during the study.
Exclusion Criteria:
- Has had a loss of 3 or more points in the combined motor and language components of the Hamburg CLN2 rating scale between Baseline of Study 190-201 and the Study Completion visit in Study 190-201 and would not benefit from enrolling in the study in the Investigator's discretion.
- Has a score of 0 points on the combined motor and language components of the Hamburg CLN2 rating scale.
- Is pregnant or breastfeeding, at Baseline, or planning to become pregnant (self or partner) at any time during the study.
- Has used any investigational product (other than BMN 190 in 190-201), or investigational medical device, within 30 days prior to Baseline; or is required to use any investigational agent prior to completion of all scheduled study assessments.
- Has a concurrent disease or condition that would interfere with study participation, or pose a safety risk, as determined by the Investigator.
- Has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.

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 | |
Germany | |
Universitaetsklinikum Hamburg-Eppendorf | |
Hamburg, Germany, 20246 | |
Italy | |
Children's Hospital Bambino Gesù,IRCCS | |
Rome, Piazza, Italy, 00165 | |
United Kingdom | |
Great Ormond Street Childrens Hospital | |
London, United Kingdom, WC1N 3JH |
Study Director: | Medical Monitor, MD | BioMarin Pharmaceutical |
Responsible Party: | BioMarin Pharmaceutical |
ClinicalTrials.gov Identifier: | NCT02485899 |
Other Study ID Numbers: |
190-202 |
First Posted: | June 30, 2015 Key Record Dates |
Last Update Posted: | February 11, 2021 |
Last Verified: | February 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | Yes |
Late infantile Neuronal Ceroid Lipofuscinosis Type 2 LINCL NCL2 CLN2 Jansky-Bielschowsky disease |
Neuronal Ceroid-Lipofuscinoses Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Nervous System Diseases Genetic Diseases, Inborn |
Lipidoses Lipid Metabolism, Inborn Errors Metabolism, Inborn Errors Lipid Metabolism Disorders Metabolic Diseases |