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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: 19 February 2015
Main ID:  NCT00754780
Date of registration: 16/09/2008
Prospective Registration: No
Primary sponsor: Mayo Clinic
Public title: Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
Scientific title: Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I
Date of first enrolment: September 2000
Target sample size: 24
Recruitment status: Completed
Study type:  Interventional
Study design:  Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment  
Phase:  Phase 2
Countries of recruitment
Name:     Dusica Babovic-Vuksanovic, M.D.
Affiliation:  Mayo Clinic
Key inclusion & exclusion criteria

Inclusion Criteria:

- Diagnosis of neurofibromatosis type I, based on clinical criteria (NIH Consensus
Development Conference, 1988). Tumors will not be confirmed histologically, since the
biopsy could cause a change in tumor growth and such interfere with effect of
Pirfenidone treatment.

- Male or female patients

- Age 18 years old

- All patients should be mentally capable of signing the consent form or should have a
legal guardian to provide consent

- Patients who are experiencing symptoms from neurofibromatous lesions and who refuse
surgery or are not good surgical candidates, such as those with plexiform
neurofibroma who are experiencing significant discomfort, disfigurement or nerve
compression or

- Presence of multiple spinal neurofibromas in which the surgical removal would carry a
major risk for spinal cord damage.

Exclusion Criteria:

- Tumors for which surgical removal could lead to permanent (or long-term) relief of

- Patients with open skin lesions and patients for whom surgery is being contemplated
or who had surgery less than 4 weeks from starting treatment

- Patients for whom biopsy is warranted for suspected malignancies

- Individuals younger than 18 years

- Pregnant and lactating women

- Inability to have MR imaging (e.g. claustrophobia, pacemaker or allergy to contrast
dye, if administration is needed for neurofibroma imaging)

Age minimum: 18 Years
Age maximum: 70 Years
Gender: Both
Health Condition(s) or Problem(s) studied
Drug: Pirfenidone
Primary Outcome(s)
tumor volume [Time Frame: 24 months]
Secondary Outcome(s)
Secondary ID(s)
Mayo Pirfenidone Study
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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