NCT02485899

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 23 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 3 Years – 16 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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 help restore TPP1 enzyme activity. The Phase 1/2 study (190-201) evaluated the efficacy and safety of a 300 mg dose of BMN 190 administered every other week (qow) to 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 BMN 190 treatment. The 190-202 study is an open label extension protocol to assess long-term safety and efficacy.

Conditions

• Jansky-Bielschowsky Disease
• Batten Disease
• Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
• CLN2 Disease
• CLN2 Disorder

Interventions

Type	Name	Description
BIOLOGICAL	BMN 190	300 mg Intracerebroventricular (ICV) infusion administered every other week for up to 240 weeks
DEVICE	Intracerebroventricular (ICV) 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.

Timeline

• Start date: 2015-02-01
• Primary completion: 2020-12-10
• Completion: 2020-12-10
• First posted: 2015-06-30
• Last updated: 2022-08-24
• Results posted: 2022-08-24

Locations

4 sites across 4 countries: United States, Germany, Italy, United Kingdom

Regulatory

• FDA-regulated drug study
• FDA-regulated device study