CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 14 enrolled
Drug / intervention
BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1) +1 morebiological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT02678689
NCT02678689Phase 2Completed
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A Phase 2, Open-Label, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

BioMarin Pharmaceutical·interventional·Posted Feb 10, 2016·Updated Feb 17, 2025

In Brief

A Phase 2 clinical trial evaluating BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1) and Intracerebroventricular access device for Jansky-Bielschowsky Disease and 4 related conditions. Completed, enrolled 14 participants across 4 sites in 4 countries.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsJansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
CountriesGermany, Italy, United Kingdom, United States
Collaborators--

Timeline

Phase 2CompletedFinished
20162017201820192020202120222023202420252026
First PostedFeb 10, 2016
Enrollment StartJan 22, 2016
Primary CompletionApr 20, 2022
TodayJul 2, 2026
Enrollment to primary: 6.2 yearsPosted 10.4 years ago

Interventions

BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)biological

Intracerebroventricular access devicedevice

Surgical implantation of an MRI compatible ICV access device in the lateral ventricle of the right hemisphere is required for administration of study drug.