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

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ClinicalTrials.gov Identifier: NCT00076102
Recruitment Status : Completed
First Posted : January 14, 2004
Results First Posted : July 12, 2012
Last Update Posted : April 23, 2018
Sponsor:
Information provided by (Responsible Party):
Brigitte Widemann, M.D., National Cancer Institute (NCI)

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Neurofibromatosis 1
Neurofibroma, Plexiform
Intervention: Drug: Pirfenidone

  Participant Flow

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
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).

Participant Flow:   Overall Study
    Pirfenidone
STARTED   36 
COMPLETED   31 
NOT COMPLETED   5 
Clinical progression                1 
Progression in pre-existing brain tumor                1 
plexiform neurofibroma surgery                1 
refusal of further therapy                2 



  Baseline Characteristics

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 
Overall Participants Analyzed 
[Units: Participants]
 36 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      34  94.4% 
Between 18 and 65 years      2   5.6% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 9.81  (4.52) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      10  27.8% 
Male      26  72.2% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      6  16.7% 
Not Hispanic or Latino      29  80.6% 
Unknown or Not Reported      1   2.8% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      5  13.9% 
White      29  80.6% 
More than one race      0   0.0% 
Unknown or Not Reported      2   5.6% 
Region of Enrollment 
[Units: Participants]
 
United States   36 


  Outcome Measures

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

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

3.  Primary:   Percentage of Participants Who Had an Objective Response Rate   [ Time Frame: ≥4 weeks ]

4.  Secondary:   Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline   [ Time Frame: Baseline ]

5.  Secondary:   Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale   [ Time Frame: prior to cycles 1, 4, 7 and 10. ]

6.  Secondary:   Number of Participants With A Response Evaluation Determined by the Comparison of One-Dimensional (1D) Magnetic Resonance Imaging   [ Time Frame: Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progression ]

7.  Secondary:   Number of Participants With A Response Evaluation Determined by the Comparison of Two-Dimensional (2D) Magnetic Resonance Imaging   [ Time Frame: Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progression ]

8.  Secondary:   Number of Participants With A Response Evaluation Determined by the Comparison of Three-Dimensional (3D) Magnetic Resonance Imaging   [ Time Frame: Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter, approximately 5 years ]

9.  Secondary:   Number of Participants Who Contributed to the Tissue Bank   [ Time Frame: 5 years ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

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
No text entered.


  More Information

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 of Results:
Other Publications:

Responsible Party: Brigitte Widemann, M.D., National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00076102     History of Changes
Obsolete Identifiers: NCT00078936
Other Study ID Numbers: 040080
04-C-0080
FD-R-0002128 ( Other Grant/Funding Number: FDA OOPD )
First Submitted: January 13, 2004
First Posted: January 14, 2004
Results First Submitted: January 4, 2012
Results First Posted: July 12, 2012
Last Update Posted: April 23, 2018