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Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JUMP)

This study has been completed.
Sponsor:
Collaborator:
Batten Disease Support and Research Assocation (BDSRA)
Information provided by (Responsible Party):
Erika Augustine, University of Rochester
ClinicalTrials.gov Identifier:
NCT01399047
First received: July 5, 2011
Last updated: November 28, 2016
Last verified: November 2016
July 5, 2011
November 28, 2016
July 2011
November 2015   (Final data collection date for primary outcome measure)
Tolerability [ Time Frame: 8 weeks ]
The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug.
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
Complete list of historical versions of study NCT01399047 on ClinicalTrials.gov Archive Site
  • Preliminary Evidence of Efficacy [ Time Frame: 8 week ]
    To gather preliminary evidence of the short-term (8 week) impact of mycophenolate mofetil on clinically relevant features of Juvenile Neuronal Ceroid Lipofuscinosis as measured by the Unified Batten Disease Rating Scale, including motor features, seizures, behavior, cognitive and functional measures.
  • Feasibility of Clinical Trials in Rare Disorder
    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.
  • 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.
  • 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.
Not Provided
Not Provided
 
Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolate in Children With Juvenile Neuronal Ceroid Lipofuscinosis

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).

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.

Interventional
Phase 2
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Juvenile Neuronal Ceroid Lipofuscinosis
  • 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-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.
    Other Name: Cellcept
  • 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-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.
  • Active Comparator: Mycophenolate Mofetil
    Intervention: Drug: Mycophenolate mofetil
  • Placebo Comparator: Placebo liquid
    Intervention: Drug: Liquid Placebo
Augustine EF, Adams HR, Mink JW. Clinical trials in rare disease: challenges and opportunities. J Child Neurol. 2013 Sep;28(9):1142-50. doi: 10.1177/0883073813495959.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
19
November 2015
November 2015   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
6 Years to 25 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT01399047
3908
Yes
Not Provided
Not Provided
Erika Augustine, University of Rochester
University of Rochester
Batten Disease Support and Research Assocation (BDSRA)
Principal Investigator: Erika F Augustine, MD University of Rochester
University of Rochester
November 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP