Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

• Status: Completed
• Trial end date: 2015-11-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Rochester
• Collaborator: Batten Disease Support and Research Assocation (BDSRA)
• Treatments: Mycophenolate mofetil; Mycophenolic Acid

Criteria

Inclusion Criteria:

• JNCL as determined by a characteristic clinical presentation and confirmatory genetic evidence.

• Able to walk 10 feet without assistance beyond that required due to vision impairment.

• Subjects with local treating clinician (pediatrician or neurologist) willing to conduct the trial according to the protocol, good clinical practice, and applicable regulations.

• Subjects with a parent/legal guardian willing to accompany them to all study visits, oversee study drug compliance, and monitor and report to local treating clinician/investigator and the URBC investigative personnel any signs of adversity.

Exclusion Criteria:

• Inability to tolerate oral administration of medications

• Concomitant medical condition, which, in the opinion of the local treating clinician, the parent(s)/guardian, or the URBC study investigator would place the child at greater than acceptable risk from: 1) travel by plane or car to the URBC on four occasions over the course of 22 weeks, 2) exposure to mycophenolate mofetil at protocol defined dosages for periods up to 8 weeks.

• Anticipated inability of the child (on the part of the investigator, parent/guardian, or URBC study personnel) to comply with the rigors of the protocol..

• Use of disallowed concomitant medications.

• Administration of immunosuppressive medications

• History of any prior exposure to mycophenolate mofetil

• History of hypersensitivity to mycophenolate mofetil, or any other component of the product

• History of frank gastrointestinal hemorrhage, ulceration, or melena

• White blood cell count < 3000/μL, absolute neutrophil count (ANC) < 1500/μL, hemoglobin < 10g/dL, or thrombocytopenia <100,000/μL.

• Abnormal liver function (aspartate aminotransferase (AST) or alanine aminotransferase (ALT) or bilirubin greater than 3 times the upper limit of normal)

• Pregnancy or vulnerability to engage in sexual intercourse based on report of the parent/guardian, judgment of the local treating clinician/investigator or judgment of the URBC study personnel.

• Positive Tuberculosis test

• Immunizations not up to date for age according to Centers for Disease Control guidelines