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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
Results First Received: November 28, 2016  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Juvenile Neuronal Ceroid Lipofuscinosis
Interventions: Drug: Mycophenolate mofetil
Drug: Liquid Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Group A: Mycophenolate to Placebo Crossover Subjects received one treatment period (8 weeks) of mycophenolate, then, after a 4-week washout, received one treatment period (8 weeks) of placebo, in a blinded manner.
Group B: Placebo to Mycophenolate Crossover Subjects received one treatment period (8 weeks) of placebo, then, after a 4-week washout, received one treatment period (8 weeks) of mycophenolate, in a blinded manner.

Participant Flow:   Overall Study
    Group A: Mycophenolate to Placebo Crossover   Group B: Placebo to Mycophenolate Crossover
STARTED   10   9 
COMPLETED   10   8 
NOT COMPLETED   0   1 
Withdrawal by Subject                0                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Group A: Mycophenolate to Placebo Crossover Subjects received one treatment period (8 weeks) of mycophenolate, then, after a 4-week washout, received one treatment period (8 weeks) of placebo, in a blinded manner.
Group B: Placebo to Mycophenolate Crossover Subjects received one treatment period (8 weeks) of placebo, then, after a 4-week washout, received one treatment period (8 weeks) of mycophenolate, in a blinded manner.
Total Total of all reporting groups

Baseline Measures
   Group A: Mycophenolate to Placebo Crossover   Group B: Placebo to Mycophenolate Crossover   Total 
Overall Participants Analyzed 
[Units: Participants]
 10   9   19 
Age 
[Units: Years]
Mean (Standard Deviation)
 10.50  (5.99)   14.78  (2.44)   12.53  (5.04) 
Gender 
[Units: Participants]
Count of Participants
     
Female      2  20.0%      2  22.2%      4  21.1% 
Male      8  80.0%      7  77.8%      15  78.9% 
Race/Ethnicity, Customized 
[Units: Participants]
Count of Participants
     
Race       
White/Caucasian      9  90.0%      8  88.9%      17  89.5% 
More than one Race      1  10.0%      1  11.1%      2  10.5% 
Region of Enrollment 
[Units: Participants]
     
United States   10   9   19 


  Outcome Measures

1.  Primary:   Tolerability   [ Time Frame: 8 weeks ]

2.  Secondary:   Preliminary Evidence of Efficacy   [ Time Frame: 8 week ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

3.  Secondary:   Feasibility of Clinical Trials in Rare Disorder   [ Time Frame: ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Erika Augustine, MD, MS
Organization: University of Rochester Medical Center
phone: 585-275-2808
e-mail: erika_augustine@urmc.rochester.edu


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Erika Augustine, University of Rochester
ClinicalTrials.gov Identifier: NCT01399047     History of Changes
Other Study ID Numbers: 3908
Study First Received: July 5, 2011
Results First Received: November 28, 2016
Last Updated: November 28, 2016