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A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

NCT02678689 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2025-02-17

Study results available
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Summary

This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 144 weeks, in patients with CLN2. The study is designed to assess disease progression in CLN2 patients treated with BMN 190 compared to natural history data from untreated historical controls.

Conditions

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

Interventions

BIOLOGICAL

BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)

DEVICE

Intracerebroventricular 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 Director, MD · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-22
Primary Completion
2022-04-20
Completion
2022-04-20
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 NCT02678689 on ClinicalTrials.gov