Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JUMP)
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ClinicalTrials.gov Identifier: NCT01399047 |
Recruitment Status :
Completed
First Posted : July 21, 2011
Results First Posted : January 23, 2017
Last Update Posted : May 21, 2019
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Tracking Information | ||||
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First Submitted Date ICMJE | July 5, 2011 | |||
First Posted Date ICMJE | July 21, 2011 | |||
Results First Submitted Date ICMJE | November 28, 2016 | |||
Results First Posted Date ICMJE | January 23, 2017 | |||
Last Update Posted Date | May 21, 2019 | |||
Study Start Date ICMJE | July 2011 | |||
Actual Primary Completion Date | November 2015 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
Tolerability - Number of Participants Who Completed Each Arm on Assigned Study Drug Dose [ Time Frame: Baseline to 8 weeks ] The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug.
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Original Primary Outcome Measures ICMJE |
Safety and Tolerability as shown by number of subjects with adverse events [ Time Frame: 3 years ] Establish the safety and tolerability of short-erm (8 weeks) administration of mycophenolate mofetil in ambulatory children with JNCL
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Change History | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | |||
Original Other Pre-specified Outcome Measures | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis | |||
Official Title ICMJE | Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolate in Children With Juvenile Neuronal Ceroid Lipofuscinosis | |||
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 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). |
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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 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. |
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Study Type ICMJE | Interventional | |||
Study Phase ICMJE | Phase 2 | |||
Study Design ICMJE | Allocation: Randomized Intervention Model: Crossover Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Condition ICMJE | Juvenile Neuronal Ceroid Lipofuscinosis | |||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Completed | |||
Actual Enrollment ICMJE |
19 | |||
Original Estimated Enrollment ICMJE |
30 | |||
Actual Study Completion Date ICMJE | November 2015 | |||
Actual Primary Completion Date | November 2015 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 6 Years to 25 Years (Child, Adult) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT01399047 | |||
Other Study ID Numbers ICMJE | 3908 | |||
Has Data Monitoring Committee | Yes | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE | Not Provided | |||
Current Responsible Party | Erika Augustine, University of Rochester | |||
Original Responsible Party | Frederick J Marshall, University of Rochester | |||
Current Study Sponsor ICMJE | University of Rochester | |||
Original Study Sponsor ICMJE | Same as current | |||
Collaborators ICMJE | Batten Disease Support and Research Assocation (BDSRA) | |||
Investigators ICMJE |
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PRS Account | University of Rochester | |||
Verification Date | May 2019 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |