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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
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.
Details
| Lead sponsor | BioMarin Pharmaceutical |
|---|---|
| Phase | Phase 1/Phase 2 |
| Status | COMPLETED |
| Enrolment | 24 |
| Start date | 2013-09 |
| Completion | 2015-11 |
Conditions
- Jansky-Bielschowsky Disease
- Batten Disease
- Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
- CLN2 Disease
Interventions
- BMN 190
Primary outcomes
- Motor-Language (ML) Scale Score During 300 mg Dosing Period — Baseline, Week 49/Last Assessment
The progression of ceroid lipofuscinosis (CLN2) disease was assessed using adapted motor and language domains of the Hamburg rating scale (ML scale score). Motor and Language are each 0 - 3 point subscales in which 3 represents best function and 0 represents loss of function. The sum of the motor and language scores (ML score, 0-6 points) was used to evaluate the loss of function.
Countries
United States, Germany, Italy, United Kingdom