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NCT01907087 · ClinicalTrials.gov registry record · Phase 1
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
A Phase 1 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 1
- Development phase
- 24
- Enrollment target
NCT01907087: Completed Phase 1 study, sponsored by BioMarin Pharmaceutical.
NCT01907087 is a Phase 1 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 24 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (60% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01907087, a Phase 1 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 24 participants
- Enrollment target
Study Summary
The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease.
Primary Outcome
The progression of ceroid lipofuscinosis (CLN2) disease was assessed using adapted motor and language domains of the Hamburg rating scale (ML scale score). Motor and Language are each 0 - 3 point subscales in which 3 represents best function and 0 represents loss of function. The sum of the motor and language scores (ML score, 0-6 points) was used to evaluate the loss of function.
Interventions
- BIOLOGICAL BMN 190
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2013-09 |
| Est. Completion | 2015-11 |
| Phase | Phase 1 |
What the finished NCT01907087 record still lists
NCT01907087 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (60% lower).
The record links to 0 conditions, and to 1 intervention - of which BMN 190 is the first listed.
NCT01907087 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01907087 about?
NCT01907087 is a clinical study titled "A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease". The purpose of this study is to determine whether BMN 190 is safe and effective in the treatment of patients with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease.
What is the current status of trial NCT01907087?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 24 participants. The study started on 2013-09. Estimated completion is 2015-11.
What interventions are being tested in trial NCT01907087?
The interventions under investigation include: BMN 190 (BIOLOGICAL).
Who is sponsoring clinical trial NCT01907087?
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 NCT01907087's enrollment target sits among peer trials
24 1520th of 2000 higher than 405 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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