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

NCT02485899 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2022-08-24

Study results available
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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.

Conditions

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

Interventions

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.

Sponsors & Collaborators

Principal Investigators

  • Medical Monitor, MD · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

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

Timeline & Regulatory

Start
2015-02-28
Primary Completion
2020-12-10
Completion
2020-12-10
FDA Drug
Yes
FDA Device
Yes

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 NCT02485899 on ClinicalTrials.gov