Know Cancer

forgot password

Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I

Phase 2
18 Years
70 Years
Not Enrolling

Thank you

Trial Information

Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I

Specific aims of this study are:

1. To evaluate efficacy of Pirfenidone in NF1 patients with disfiguring or disabling
plexiform neurofibroma (PN) and spinal neurofibromas (SN)

2. To determine the acute, subacute and chronic toxicity of Pirfenidone in patients with

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)

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

tumor volume

Outcome Time Frame:

24 months

Safety Issue:


Principal Investigator

Dusica Babovic-Vuksanovic, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Mayo Clinic


United States: Institutional Review Board

Study ID:




Start Date:

September 2000

Completion Date:

Related Keywords:

  • Neurofibromatosis
  • NF1
  • plexiform neurofibroma
  • Neurofibromatoses
  • Neurofibromatosis 1
  • Osteitis Fibrosa Cystica