MedTrace Logo

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

NCT01907087 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2019-03-08

Study results available
· View outcomes & findings →

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.

Conditions

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

Interventions

BIOLOGICAL

BMN 190

30-300 mg ICV infusion administered every other week for at least 48 weeks.

Sponsors & Collaborators

Principal Investigators

  • David Jacoby · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-09-30
Primary Completion
2015-11-30
Completion
2015-11-30

Countries

  • United States
  • Germany
  • Italy
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01907087 on ClinicalTrials.gov