Overview

This trial is active, not recruiting.

Conditions jansky-bielschowsky disease, batten disease, late-infantile neuronal ceroid lipofuscinosis type 2, cln2 disease, cln2 disorder
Treatment bmn 190
Phase phase 1/phase 2
Sponsor BioMarin Pharmaceutical
Start date February 2015
End date December 2021
Trial size 23 participants
Trial identifier NCT02485899, 190-202

Summary

The Phase 1/2 study (190-201) evaluated the efficacy and safety of doses up to 300 mg/every other week (qow) BMN 190 in 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 to receive continued BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
bmn 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)
300 mg ICV infusion administered every other week for up to 240 weeks

Primary Outcomes

Measure
Long Term Safety as assessed by analysis of adverse events.
time frame: up to 240 weeks
Motor and Language Changes
time frame: up to 240 weeks

Secondary Outcomes

Measure
Quantitative Assessment of Magnetic Resonance Imaging
time frame: up to 240 weeks
CLN2 Disease Scale Score
time frame: up to 240 weeks
Quality of Life Changes
time frame: up to 240 weeks

Eligibility Criteria

Male or female participants from 3 years up to 16 years old.

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.

Additional Information

Official title A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
Description 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 restore TPP1 enzyme activity. BMN 190 is designed to reduce the progressive, pathologic accumulation of lysosomal storage material, and improve the symptoms of disease. The Phase 1/2 study (190-201) evaluated the efficacy and safety of doses up to 300 mg/every other week (qow) BMN 190 in 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 to receive continued BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by BioMarin Pharmaceutical.