CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 19 enrolled
Drug / intervention
Mycophenolate mofetil +1 moredrug
Likely dose
Mycophenolate mofetil 300mg/m2from 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/NCT01399047
NCT01399047Phase 2Completed
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Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolate in Children With Juvenile Neuronal Ceroid Lipofuscinosis

University of Rochester·interventional·Posted Jul 21, 2011·Updated May 21, 2019

In Brief

A Phase 2 clinical trial evaluating Mycophenolate mofetil and Liquid Placebo for Juvenile Neuronal Ceroid Lipofuscinosis. Completed, enrolled 19 participants across 1 site.

Detailed 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).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsJuvenile Neuronal Ceroid Lipofuscinosis
CountriesUnited States
CollaboratorsBatten Disease Support and Research Assocation (BDSRA)

Timeline

Phase 2CompletedFinished
201220132014201520162017201820192020202120222023202420252026
First PostedJul 21, 2011
Enrollment StartJul 1, 2011
Primary CompletionNov 1, 2015
TodayJul 2, 2026
Enrollment to primary: 4.3 yearsPosted 14.9 years ago

Interventions

Mycophenolate mofetildrug

The liquid dosage will be individualized, contingent upon the subject's weight. Subjects will receive 50% of the target dose (300mg/m2/dose BID) during Week 0-Week 2, then increase to the full dose (600mg/m2/dose BID) in Week 3, continuing at this dose through Week 8. Additionally, due to the risk of gastrointestinal disturbance (hemorrhage, ulcer), children will also receive prophylactic Prilosec (Omeprazole) for the duration of the study, during both the mycophenolate and placebo arms.

Liquid Placebodrug

The dosage will be individualized, contingent upon the subject's weight. Subjects will receive 50% of the target dose (300mg/m2/dose BID) during Week 0-Week 2, then increase to the full dose (600mg/m2/dose BID) in Week 3, continuing at this dose through Week 8. Additionally, due to the risk of gastrointestinal disturbance (hemorrhage, ulcer), children will also receive prophylactic Prilosec (omeprazole) for the duration of the study, during both the mycophenolate and placebo arms.