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NCT02678689 · ClinicalTrials.gov registry record · Phase 2
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
A Phase 2 study of Batten Disease and Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 2
- Development phase
- 14
- Enrollment target
- 4
- Study locations
NCT02678689: Completed Phase 2 study of Batten Disease and Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, sponsored by BioMarin Pharmaceutical.
NCT02678689 is a Phase 2 study of Batten Disease and Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 14 participants, below the 2,828-participant average among 11 other Batten Disease trials with a reported enrollment target (100% lower). The trial reports 4 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02678689, a Phase 2 study of Batten Disease and Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 14 participants
- Enrollment target
- 4
- Study locations
Study 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.
Primary Outcome
Rate of decline in 0 to 6-point ML score, \& primary analysis was based on up to 3-1 matching of Study 190-901 evaluable participants with Study 190-203 ITT participants. Rate of decline =(-1)x(48x7)x(Ending score - Starting score)/(Ending date - Starting date) A positive rate of decline means that subject declined, a negative rate of decline means that subject improved. The combined motor/gait and language (ML) score, as derived from Hamburg CLN2 rating scale, immediately preceding first inf
Conditions Studied
Interventions
- BIOLOGICAL BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)
- DEVICE Intracerebroventricular access device
Study Locations (4)
Other
- Universitaetsklinikum Hamburg-Eppendorf - Hamburg
- Great Ormond Street Childrens Hospital - London
Ohio
- Nationwide Children's Hospital - Columbus
Piazza
- Children's Hospital Bambino Gesù,IRCCS - Rome
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 14 participants |
| Start Date | 2016-01-22 |
| Est. Completion | 2022-04-20 |
| Phase | Phase 2 |
What the finished NCT02678689 record still lists
NCT02678689 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 14 participants, a relatively small participant target, below the 2,828-participant average among 11 other Batten Disease trials with a reported enrollment target (100% lower).
The record links to 5 conditions, with Batten Disease appearing as the primary indexed condition, and to 2 interventions - of which BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1) is the first listed.
NCT02678689 reports a single indexed study location in Other, Ohio, Piazza.
Frequently Asked Questions
What is clinical trial NCT02678689 about?
NCT02678689 is a clinical study titled "A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease". 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 treate...
What is the current status of trial NCT02678689?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 14 participants. The study started on 2016-01-22. Estimated completion is 2022-04-20.
What conditions does trial NCT02678689 study?
This clinical trial studies the following conditions: Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, Jansky-Bielschowsky Disease, CLN2 Disease, CLN2 Disorder.
What interventions are being tested in trial NCT02678689?
The interventions under investigation include: BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1) (BIOLOGICAL), Intracerebroventricular access device (DEVICE).
Who is sponsoring clinical trial NCT02678689?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02678689 being conducted?
This trial has 4 study locations across Ohio, Piazza. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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