Expired Study
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Rochester, New York 14642


Purpose:

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 summary:

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 data on genetic knock-down of the ability to mount an immune response in cln3-knockout mice is supportive of a strategy for treating JNCL with an immuno-suppressive agent. Many drugs with the ability to suppress the immune system are steroidal and deemed unsuitable for long-term administration to children. Mycophenolate mofetil (CellCept) is used as an immunosuppressive agent in allogenic transplants in pediatric patients and is therefore approved by the Food and Drug Administration (FDA) for pediatric use. The study design is a double-blind, randomized, 22-week cross-over study of mycophenolate mofetil vs. placebo. After a 4-week washout period, subjects will undergo blinded crossover from active study drug to placebo or from placebo to active study drug. Subjects and caregivers will be evaluated in person in the University of Rochester Batten Center (URBC) at screening/baseline, and at weeks 8, 12, and 20. In addition, subjects will be evaluated by their local clinician who is a formalized member of the research team. Such contacts will occur at Weeks 2, 4, 14, 16, and any unscheduled or early termination visits. There will also be regular telephone contact between the URBC and the local clinician. We have selected the dosage currently FDA approved for use in children being treated for prophylaxis of renal transplant rejection.


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


Study is Available At:


Original ID:

3908


NCT ID:

NCT01399047


Secondary ID:


Study Acronym:

JUMP


Brief Title:

Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis


Official Title:

Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolate in Children With Juvenile Neuronal Ceroid Lipofuscinosis


Overall Status:

Completed


Study Phase:

Phase 2


Genders:

N/A


Minimum Age:

6 Years


Maximum Age:

25 Years


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

University of Rochester


Oversight Authority:

United States: Food and Drug Administration


Reasons Why Stopped:


Study Type:

Interventional


Study Design:


Number of Arms:

2


Number of Groups:

0


Total Enrollment:

19


Enrollment Type:

Actual


Overall Contact Information

Official Name:Erika F Augustine, MD
Principal Investigator
University of Rochester

Study Dates

Start Date:July 2011
Completion Date:November 2015
Completion Type:Actual
Primary Completion Date:November 2015
Primary Completion Type:Actual
Verification Date:May 2019
Last Changed Date:May 7, 2019
First Received Date:July 5, 2011
First Results Date:November 28, 2016

Study Outcomes

Outcome Type:Secondary Outcome
Measure:Unified Batten Disease Rating Scale Capability Subscale Change
Time Frame:Baseline to 8 weeks
Safety Issues:False
Description:The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Capability severity was measured by the capability subscale of the UBDRS with a range of 0-14 with higher scores indicating better outcome. The overall treatment e
Outcome Type:Secondary Outcome
Measure:Unified Batten Disease Rating Scale Behavior Subscale Change
Time Frame:Baseline to 8 weeks
Safety Issues:False
Description:The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Mood and behavior severity was measured by the behavior subscale of the UBDRS with a range of 0-55 with zero indicating better outcome. The overall treatment effec
Outcome Type:Secondary Outcome
Measure:Unified Batten Disease Rating Scale Seizure Subscale Change
Time Frame:Baseline to 8 weeks
Safety Issues:False
Description:The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Seizure severity was measured by the seizure subscale of the UBDRS with a range of 0-54 with zero indicating better outcome. The overall treatment effect was deter
Outcome Type:Secondary Outcome
Measure:Unified Batten Disease Rating Scale Physical Subscale Change
Time Frame:Baseline to 8 weeks
Safety Issues:False
Description:The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Motor impairment was measured by the physical subscale of the UBDRS with a range of 0-112 with zero indicating better outcome. The overall treatment effect was det
Outcome Type:Primary Outcome
Measure:Tolerability - Number of Participants Who Completed Each Arm on Assigned Study Drug Dose
Time Frame:Baseline to 8 weeks
Safety Issues:False
Description:The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug.

Study Interventions

Intervention Type:Drug
Name:Mycophenolate mofetil
Description: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.
Arm Name:Mycophenolate Mofetil
Other Name:Cellcept
Intervention Type:Drug
Name:Liquid Placebo
Description: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.
Arm Name:Placebo liquid

Study Arms

Study Arm Type:Placebo Comparator
Arm Name:Placebo liquid
Study Arm Type:Active Comparator
Arm Name:Mycophenolate Mofetil

Study Agencies

Agency Class:Other
Agency Type:Lead Sponsor
Agency Name:University of Rochester
Agency Class:Other
Agency Type:Collaborator
Agency Name:Batten Disease Support and Research Assocation (BDSRA)

Sample and Retention Information

There are no available Sample and Retention Information

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: October 09, 2019

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