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Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

NCT01399047 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 19

Last updated 2019-05-21

Study results available
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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).

Conditions

  • Juvenile Neuronal Ceroid Lipofuscinosis

Interventions

DRUG

Mycophenolate mofetil

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.

DRUG

Liquid Placebo

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.

Sponsors & Collaborators

  • Batten Disease Support and Research Assocation (BDSRA)

    collaborator UNKNOWN

  • University of Rochester

    lead OTHER

Principal Investigators

  • Erika F Augustine, MD · University of Rochester

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
6 Years
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-07-31
Primary Completion
2015-11-30
Completion
2015-11-30

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01399047 on ClinicalTrials.gov