NCT01907087

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 3 Years – 15 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease.

Detailed description

The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease. This is an open label Phase 1/2 study conducted in patients with CLN2 disease. Efficacy measures (disease rating scale and MRI) will be compared to a natural history control. The study will be conducted under cGCP and patients will be closely monitored.

Conditions

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

Interventions

Type	Name	Description
BIOLOGICAL	BMN 190	30-300 mg ICV infusion administered every other week for at least 48 weeks.

Timeline

• Start date: 2013-09-01
• Primary completion: 2015-11-01
• Completion: 2015-11-01
• First posted: 2013-07-24
• Last updated: 2019-03-08
• Results posted: 2018-06-11

Locations

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