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History of Changes for Study: NCT01399047
Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JUMP)
Latest version (submitted May 7, 2019) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 July 19, 2011 None (earliest Version on record)
2 August 2, 2011 Recruitment Status, Study Status and Contacts/Locations
3 September 7, 2012 Sponsor/Collaborators, Contacts/Locations and Study Status
4 March 18, 2013 Study Status, Contacts/Locations, Eligibility, Outcome Measures, Arms and Interventions and Study Description
5 October 8, 2013 Study Status
6 December 16, 2013 Contacts/Locations, Study Status and Sponsor/Collaborators
7 March 20, 2014 Study Status
8 May 7, 2014 Study Status, Study Description and Sponsor/Collaborators
9 December 2, 2014 Sponsor/Collaborators, Study Status, Eligibility and Study Description
10 May 29, 2015 Study Status
11 August 14, 2015 Recruitment Status, Study Status and Contacts/Locations
12 December 4, 2015 Recruitment Status, Study Status and Sponsor/Collaborators
13 November 28, 2016 Outcome Measures, Study Status, Study Design and Results
14 April 17, 2019 Outcome Measures, Study Status and Baseline Characteristics
15 May 7, 2019 Outcome Measures, Study Status
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Study NCT01399047
Submitted Date:  July 19, 2011 (v1)

Open or close this module Study Identification
Unique Protocol ID: 3908
Brief Title: Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JUMP)
Official Title: Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolate in Children With Juvenile Neuronal Ceroid Lipofuscinosis
Secondary IDs:
Open or close this module Study Status
Record Verification: July 2011
Overall Status: Not yet recruiting
Study Start: August 2011
Primary Completion: August 2014 [Anticipated]
Study Completion: August 2014 [Anticipated]
First Submitted: July 5, 2011
First Submitted that
Met QC Criteria:
July 19, 2011
First Posted: July 21, 2011 [Estimate]
Last Update Submitted that
Met QC Criteria:
July 19, 2011
Last Update Posted: July 21, 2011 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: University of Rochester
Responsible Party:
Collaborators: Batten Disease Support and Research Association
FDA Office of Orphan Products Development
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: Yes
Open or close this module Study Description
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 UBDRS, including motor features, seizures, behavior, cognitive and functional measures.
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. Clinical experience in these children indicates that the drug is reasonably safe and well tolerated.

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 1, 4, 13, 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.

Open or close this module Conditions
Conditions: Juvenile Neuronal Ceroid Lipofuscinosis
Keywords: Batten Disease
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 2
Interventional Study Model: Crossover Assignment
Number of Arms: 2
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Allocation: Randomized
Enrollment: 30 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Active Comparator: Mycophenolate Mofetil 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 and Week 1, then increase to the full dose (600mg/m2/dose BID) in Week 2, continuing at this dose through Week 8. Additionally, due to the risk of gastrointestinal disturbance (hemorrhage, ulcer), children will also receive prophylactic Zantac (Ranitidine) for the duration of the study, during both the mycophenolate and placebo arms. Sufficient study drug and ranitidine for 8 weeks will be dispensed.
Other Names:
  • Cellcept
Placebo Comparator: Placebo liquid 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 and Week 1, then increase to the full dose (600mg/m2/dose BID) in Week 2, continuing at this dose through Week 8. Additionally, due to the risk of gastrointestinal disturbance (hemorrhage, ulcer), children will also receive prophylactic Zantac (Ranitidine) for the duration of the study, during both the mycophenolate and placebo arms. Sufficient study drug and ranitidine for 8 weeks will be dispensed.
Open or close this module Outcome Measures
Primary Outcome Measures:
1. 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
Secondary Outcome Measures:
1. Preliminary evidence of efficacy
[ Time Frame: 3 years ]

To gather preliminary evidence of the short-term (8 week) impact of mycophenolate mofetil on clinically relevant features of JNCL as measured by the UBDRS, including motor features, seizures, behavior, cognitive and functional measures.
2. Feasibility of clinical trials in rare disorder
[ Time Frame: 3 years ]

To pilot the feasibility of conducting controlled clinical trials of this rare neurological disorder base on collaboration between a national center of excellence in the disease (URBC), and children's local care-providers (pediatricians and/or local neurologists.
Open or close this module Eligibility
Minimum Age: 6 Years
Maximum Age: 25 Years
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
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.
  • Between 6 years and 25 years of age.
  • 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 PPD
  • Immunizations not up to date for age according to Centers for Disease Control guidelines
Open or close this module Contacts/Locations
Central Contact Person: Nicole J Newhouse, BS
Telephone: 585-273-3810
Email: Nicole_Newhouse@urmc.rochester.edu
Central Contact Backup: Amy E Vierhile, MS
Telephone: 585-275-4762
Email: Amy_Vierhile@urmc.rochester.edu
Study Officials: Frederick J Marshall, MD
Principal Investigator
University of Rochester
Locations: United States, New York
University of Rochester
Rochester, New York, United States, 14642
Contact:Contact: Nicole J Newhouse, BS 585-750-4215 Nicole_Newhouse@urmc.rochester.edu
Contact:Contact: Amy E Vierhile, MS 585-275-4762 Amy_Vierhile@urmc.rochester.edu
Contact:Principal Investigator: Frederick J Marshall, MD
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
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Available IPD/Information:

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