Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1

• Register: ClinicalTrials.gov
• 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
• URL: http://clinicaltrials.gov/show/NCT00754780
• 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

Contacts

• 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
symptoms

- 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

Neurofibromatosis

Intervention(s)

Drug: Pirfenidone

Primary Outcome(s)

tumor volume [Time Frame: 24 months]

Secondary ID(s)

Mayo Pirfenidone Study

1835-99

MC0077

Source(s) of Monetary Support

Please refer to primary and secondary sponsors