Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Brigitte Widemann, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00076102
First received: January 13, 2004
Last updated: March 11, 2013
Last verified: March 2013
Results First Received: January 4, 2012  
Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Neurofibromatosis 1
Neurofibroma, Plexiform
Intervention: Drug: Pirfenidone

  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
36 participants were enrolled in this study

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
Pirfenidone Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).

Participant Flow:   Overall Study
    Pirfenidone  
STARTED     36  
COMPLETED     36  
NOT COMPLETED     0  



  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
Pirfenidone Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).

Baseline Measures
    Pirfenidone  
Number of Participants  
[units: participants]
  36  
Age  
[units: participants]
 
<=18 years     34  
Between 18 and 65 years     2  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  9.81  ± 4.52  
Gender  
[units: participants]
 
Female     10  
Male     26  
Ethnicity (NIH/OMB)  
[units: Participants]
 
Hispanic or Latino     6  
Not Hispanic or Latino     29  
Unknown or Not Reported     1  
Race (NIH/OMB)  
[units: Participants]
 
American Indian or Alaska Native     0  
Asian     0  
Native Hawaiian or Other Pacific Islander     0  
Black or African American     5  
White     29  
More than one race     0  
Unknown or Not Reported     2  
Region of Enrollment  
[units: participants]
 
United States     36  



  Outcome Measures
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1.  Primary:   Median Time to Disease Progression   [ Time Frame: 5 years ]

2.  Primary:   Number of Participants With Adverse Events   [ Time Frame: 5 years ]


  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:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Brigitte C. Widemann, M.D.
Organization: National Cancer Institute (NCI), National Institutes of Health (NIH)
phone: 301-496-7387
e-mail: widemanb@mail.nih.gov


Publications:

Responsible Party: Brigitte Widemann, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00076102     History of Changes
Obsolete Identifiers: NCT00078936
Other Study ID Numbers: 040080, 04-C-0080, FD-R-0002128
Study First Received: January 13, 2004
Results First Received: January 4, 2012
Last Updated: March 11, 2013
Health Authority: United States: Federal Government