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Trial record 1 of 1 for:    CellCept (mycophenolate) | Batten Disease
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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
Sponsor:
Collaborator:
Batten Disease Support and Research Assocation (BDSRA)
Information provided by (Responsible Party):
Erika Augustine, University of Rochester

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).


Condition or disease Intervention/treatment Phase
Juvenile Neuronal Ceroid Lipofuscinosis Drug: Mycophenolate mofetil Drug: Liquid Placebo Phase 2

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 : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, 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
Study Start Date : July 2011
Actual Primary Completion Date : November 2015
Actual Study Completion Date : November 2015


Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Unified Batten Disease Rating Scale Physical Subscale Change [ Time Frame: Baseline to 8 weeks ]

    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 determined - mean difference in physical subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.


  2. Unified Batten Disease Rating Scale Seizure Subscale Change [ Time Frame: Baseline to 8 weeks ]

    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 determined - mean difference in seizure subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.


  3. Unified Batten Disease Rating Scale Behavior Subscale Change [ Time Frame: Baseline to 8 weeks ]

    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 effect was determined - mean difference in behavior subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.


  4. Unified Batten Disease Rating Scale Capability Subscale Change [ Time Frame: Baseline to 8 weeks ]

    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 effect was determined - mean difference in capability subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects.




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Ages Eligible for Study:   6 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
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 Tuberculosis test
  • Immunizations not up to date for age according to Centers for Disease Control guidelines

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01399047


Locations
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United States, New York
University of Rochester
Rochester, New York, United States, 14642
Sponsors and Collaborators
University of Rochester
Batten Disease Support and Research Assocation (BDSRA)
Investigators
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Principal Investigator: Erika F Augustine, MD University of Rochester

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Erika Augustine, PI, University of Rochester
ClinicalTrials.gov Identifier: NCT01399047     History of Changes
Other Study ID Numbers: 3908
First Posted: July 21, 2011    Key Record Dates
Results First Posted: January 23, 2017
Last Update Posted: May 21, 2019
Last Verified: May 2019

Keywords provided by Erika Augustine, University of Rochester:
Batten Disease

Additional relevant MeSH terms:
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Neuronal Ceroid-Lipofuscinoses
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Metabolic Diseases
Mycophenolic Acid
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Antibiotics, Antineoplastic
Antineoplastic Agents
Antibiotics, Antitubercular
Antitubercular Agents
Anti-Bacterial Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action