Status:
COMPLETED
Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
Lead Sponsor:
University of Rochester
Collaborating Sponsors:
Batten Disease Support and Research Assocation (BDSRA)
Conditions:
Juvenile Neuronal Ceroid Lipofuscinosis
Eligibility:
All Genders
6-25 years
Phase:
PHASE2
Brief 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 objectiv...
Detailed Description
Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) is a fatal disorder. Currently treatment is symptomatic. Thus, there is a real need to intervene and slow the progression of this disease. Preliminary da...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
July 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2015
Estimated Enrollment :
19 Patients enrolled
Trial Details
Trial ID
NCT01399047
Start Date
July 1 2011
End Date
November 1 2015
Last Update
May 21 2019
Active Locations (1)
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1
University of Rochester
Rochester, New York, United States, 14642