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NCT02485899 · ClinicalTrials.gov registry record · Phase 1
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
A Phase 1 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 1
- Development phase
- 23
- Enrollment target
NCT02485899: Completed Phase 1 study, sponsored by BioMarin Pharmaceutical.
NCT02485899 is a Phase 1 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 23 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (62% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02485899, a Phase 1 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 23 participants
- Enrollment target
Study 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.
Primary Outcome
Motor and Language are each 0-3 point subscales in which 3 represents best function and 0 represents loss of function. Thus, the 0-6 point ML score was used as the primary mode of evaluation of loss of function. In 190-201, the primary endpoint was a responder endpoint: unreversed ML 2-point decline or score of 0 at Week 48 compared to the 50% rate expected in natural history using the 1-sample binomial test. For 190-202, the primary endpoint was revised to assess response over the full duratio
Interventions
- BIOLOGICAL BMN 190
- DEVICE Intracerebroventricular (ICV) access device
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 23 participants |
| Start Date | 2015-02 |
| Est. Completion | 2020-12-10 |
| Phase | Phase 1 |
What the finished NCT02485899 record still lists
NCT02485899 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 23 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (62% lower).
The record links to 0 conditions, and to 2 interventions - of which BMN 190 is the first listed.
NCT02485899 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02485899 about?
NCT02485899 is a clinical study titled "An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease". 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 pro...
What is the current status of trial NCT02485899?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 23 participants. The study started on 2015-02. Estimated completion is 2020-12-10.
What interventions are being tested in trial NCT02485899?
The interventions under investigation include: BMN 190 (BIOLOGICAL), Intracerebroventricular (ICV) access device (DEVICE).
Who is sponsoring clinical trial NCT02485899?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02485899's enrollment target sits among peer trials
23 1596th of 2000 higher than 400 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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