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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Last refreshed on: 11 May 2015
Main ID:  NCT00053937
Date of registration: 05/02/2003
Prospective Registration: No
Primary sponsor: National Cancer Institute (NCI)
Public title: Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas
Scientific title: Phase I Trial Of Pirfenidone In Children With Neurofibromatosis Type 1 And Plexiform Neurofibromas
Date of first enrolment: December 2002
Target sample size:
Recruitment status: Completed
Study type:  Interventional
Study design:  Masking: Open Label, Primary Purpose: Prevention  
Phase:  Phase 1
Countries of recruitment
United States
Name:     Brigitte C. Widemann, MD
Affiliation:  National Cancer Institute (NCI)
Key inclusion & exclusion criteria


- Diagnosis of neurofibromatosis type 1 (NF1) AND

- Plexiform neurofibromas

- Neurofibromas that have grown along the length of a nerve and may involve
multiple fascicles and branches (spinal neurofibromas involve 2 or more levels
with connection between the levels or extending laterally along the nerve)

- Potential to cause significant morbidity such as:

- Head and neck lesions that could compromise airway or great vessels

- Brachial or lumbar plexus lesions that could cause nerve compression and
loss of function

- Lesions that could result in major deformity (e.g., orbital lesions) or
significant cosmetic problems

- Lesions of the extremity that cause limb hypertrophy or loss of function

- Painful lesions

- Meets at least 1 other diagnostic criteria for NF1

- 6 or more cafe-au-lait spots (at least 0.5 cm in prepubertal patients or at
least 1.5 cm in postpubertal patients)

- Freckling in the axilla or groin

- Optic glioma

- 2 or more Lisch nodules

- Distinctive bony lesion (dysplasia of the sphenoid bone or dysplasia or thinning
of long bone cortex)

- First-degree relative with NF1

- Measurable plexiform neurofibromas

- At least 3 cm in 1 dimension

- Tumor resection not feasible

- No history of malignant peripheral nerve sheath tumor or other cancer

- No evidence of an active optic glioma requiring chemotherapy or radiotherapy

- No malignant glioma



- 3 to 21

Performance status

- Karnofsky 50-100% (over 10 years of age)

- Lansky 50-100% (10 years and under)

Life expectancy

- Not specified


- Absolute granulocyte count at least 1,500/mm^3

- Hemoglobin at least 9.0 g/dL

- Platelet count at least 150,000/mm^3


- Bilirubin normal

- SGPT no greater than 2 times upper limit of normal

- No clinically significant hepatic dysfunction that would preclude study participation


- Creatinine normal for age OR

- Creatinine clearance at least 70 mL/min


- No clinically significant cardiac dysfunction that would preclude study participation


- No clinically significant pulmonary dysfunction that would preclude study


- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception during and for 2 months after study

- Must be able to take pirfenidone orally

- No serious infections

- No clinically significant unrelated systemic illness or organ dysfunction that would
preclude study participation


Biologic therapy

- At least 30 days since prior immunotherapy

- No concurrent immunotherapy

- No concurrent hematopoietic growth factors


- At least 30 days since prior chemotherapy

- No concurrent chemotherapy directed at the tumor

Endocrine therapy

- At least 30 days since prior hormonal therapy directed at the tumor

- No concurrent hormonal therapy directed at the tumor


- At least 90 days since prior radiotherapy to the site of the plexiform neurofibroma

- No concurrent radiotherapy directed at the tumor


- Not specified


- Recovered from prior therapy

- More than 30 days since prior investigational agents

- No prior pirfenidone

- No other concurrent investigational agents

Age minimum: 3 Years
Age maximum: 21 Years
Gender: Both
Health Condition(s) or Problem(s) studied
Neurofibromatosis Type 1
Precancerous Condition
Drug: pirfenidone
Primary Outcome(s)
Secondary Outcome(s)
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results available:
Date Posted:
Date Completed:
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