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NCT01399047 · ClinicalTrials.gov registry record · Phase 2
Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
A Phase 2 study, sponsored by University of Rochester.
- Completed
- Registry status
- Phase 2
- Development phase
- 19
- Enrollment target
NCT01399047: Completed Phase 2 study, sponsored by University of Rochester.
NCT01399047 is a Phase 2 study that has completed, run by University of Rochester. The registered enrollment target is 19 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (86% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01399047, a Phase 2 study, has completed, sponsored by University of Rochester.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 19 participants
- Enrollment target
Study Summary
The primary objective of this trial is to establish the safety and tolerability of short-term (8 weeks) administration of mycophenolate mofetil in ambulatory children with JNCL. The secondary objective is to gather preliminary evidence of the short-term (8 week) impact of mycophenolate mofetil on clinically relevant features of JNCL as measured by the Unified Batten Disease Rating Scale (UBDRS), including motor features, seizures, behavior, cognitive and functional measures. Funding source-FDA Office of Orphan Product Development (OOPD).
Primary Outcome
The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug.
Interventions
- DRUG Mycophenolate mofetil
- DRUG Liquid Placebo
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 19 participants |
| Start Date | 2011-07 |
| Est. Completion | 2015-11 |
| Phase | Phase 2 |
What the finished NCT01399047 record still lists
NCT01399047 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 19 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (86% lower).
The record links to 0 conditions, and to 2 interventions - of which Mycophenolate mofetil is the first listed.
NCT01399047 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01399047 about?
NCT01399047 is a clinical study titled "Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis". The primary objective of this trial is to establish the safety and tolerability of short-term (8 weeks) administration of mycophenolate mofetil in ambulatory children with JNCL. The secondary objective is to gather preliminary evidence of the short-term (8 week) impact of mycophenolate mofetil on cl...
What is the current status of trial NCT01399047?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 19 participants. The study started on 2011-07. Estimated completion is 2015-11.
What interventions are being tested in trial NCT01399047?
The interventions under investigation include: Mycophenolate mofetil (DRUG), Liquid Placebo (DRUG).
Who is sponsoring clinical trial NCT01399047?
This trial is sponsored by University of Rochester, which has 636 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01399047's enrollment target sits among peer trials
19 1894th of 2000 higher than 103 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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