Trial record 9 of 17 for:    Batten Disease

Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JUMP)

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by University of Rochester
Sponsor:
Collaborator:
Batten Disease Support and Research Association
Information provided by (Responsible Party):
Erika Augustine, University of Rochester
ClinicalTrials.gov Identifier:
NCT01399047
First received: July 5, 2011
Last updated: May 7, 2014
Last verified: May 2014
  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 UBDRS, including motor features, seizures, behavior, cognitive and functional measures.

Funding source-FDA OOPD.


Condition Intervention Phase
Juvenile Neuronal Ceroid Lipofuscinosis
Drug: Mycophenolate mofetil
Drug: Liquid Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolate in Children With Juvenile Neuronal Ceroid Lipofuscinosis

Resource links provided by NLM:


Further study details as provided by University of Rochester:

Primary Outcome Measures:
  • Safety and Tolerability as shown by number of subjects with adverse events [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
    Establish the safety and tolerability of short-term (8 weeks) administration of mycophenolate mofetil in ambulatory children with JNCL


Secondary Outcome Measures:
  • Preliminary evidence of efficacy [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: Yes ]
    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.


Estimated Enrollment: 30
Study Start Date: July 2011
Estimated Study Completion Date: August 2015
Estimated Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)
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-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
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-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.

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.

  Eligibility

Ages Eligible for Study:   6 Years to 25 Years
Genders Eligible for Study:   Both
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.
  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01399047

Contacts
Contact: Sara Defendorf, BS 585-273-3810 sara_defendorf@urmc.rochester.edu
Contact: Amy E Vierhile, MS 585-275-4762 Amy_Vierhile@urmc.rochester.edu

Locations
United States, New York
University of Rochester Recruiting
Rochester, New York, United States, 14642
Contact: Sara Defendorf, BS    585-273-3810    sara_defendorf@urmc.rochester.edu   
Contact: Amy E Vierhile, MS    585-275-4762    Amy_Vierhile@urmc.rochester.edu   
Principal Investigator: Erika Augustine, MD         
Sub-Investigator: Jonathan W. Mink, MD, PhD         
Sub-Investigator: Frederick J. Marshall, MD         
Sponsors and Collaborators
University of Rochester
Batten Disease Support and Research Association
Investigators
Principal Investigator: Erika F Augustine, MD University of Rochester
  More Information

No publications provided by University of Rochester

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Erika Augustine, PI, University of Rochester
ClinicalTrials.gov Identifier: NCT01399047     History of Changes
Other Study ID Numbers: 3908
Study First Received: July 5, 2011
Last Updated: May 7, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Rochester:
Batten Disease

Additional relevant MeSH terms:
Neuronal Ceroid-Lipofuscinoses
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Lipid Metabolism Disorders
Metabolic Diseases
Nervous System Diseases
Neurodegenerative Diseases
Lipid Metabolism, Inborn Errors
Lipidoses
Metabolism, Inborn Errors
Mycophenolate mofetil
Mycophenolic Acid
Antibiotics, Antineoplastic
Antineoplastic Agents
Enzyme Inhibitors
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on October 21, 2014