Clinical Trial: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Brief 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.

Detailed Summary: 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.
Sponsor: BioMarin Pharmaceutical

Current Primary Outcome:

• 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 subscales of the CLN2 disease rating scale in patients with CLN2 following administration of 300 mg every other week of BMN 190.

Original Primary Outcome:

• 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 NCL-2 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 subscales of the CLN2 disease rating scale in patients with CLN2 following administration of 300 mg every other week of BMN 190.

Current Secondary Outcome:

• 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

Original Secondary Outcome: Same as current

Information By: BioMarin Pharmaceutical

Dates:
Date Received: April 24, 2015
Date Started: February 2015
Date Completion:
Last Updated: October 28, 2016
Last Verified: October 2016