Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

PHASE2CompletedINTERVENTIONAL
Enrollment

19

Participants

Timeline

Start Date

July 31, 2011

Primary Completion Date

November 30, 2015

Study Completion Date

November 30, 2015

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.

Trial Locations (1)

14642

University of Rochester, Rochester

All Listed Sponsors
collaborator

Batten Disease Support and Research Assocation (BDSRA)

UNKNOWN

lead

University of Rochester

OTHER

NCT01399047 - Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis | Biotech Hunter | Biotech Hunter