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CompletedNCT00754780Updated Mar 19, 2012

Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1

A Phase 2 interventional study of Pirfenidone in Neurofibromatosis, sponsored by Mayo Clinic. Completed. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2012-03-19.

Sponsored by Mayo Clinic · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
24
Allocation
Non-randomized
Ages
18 Years to 70 Years
Sex
All
01

Study summary

The study is a phase II, open label trial of oral Pirfenidone in 24 adult patients with neurofibromatosis type 1.

Pirfenidone is a new, broad-spectrum anti-fibrotic drug, with proven in vitro and in vivo negative effects on fibroblast growth and collagen matrix synthesis. Human studies indicate promising therapeutic effects in arresting and reversing fibrosis in a variety of different conditions, where the excessive formation of fibrous tissue is a major pathogenic mechanism. Since the fibrous tissue is a significant component of neurofibroma, reduction of fibrosis could diminish tumor progression and lead to tumor shrinkage. Therefore, Pirfenidone is an excellent candidate for the treatment of plexiform neurofibromas and surgically unresectable tumors in patients with NF1.

Read the detailed description

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 NF1.
02

Conditions studied

  • Neurofibromatosis

Keywords

  • NF1
  • plexiform neurofibroma
03

In context

Neurofibromatoses

186 studies on the registry are indexed under Neurofibromatoses; 24 are open to participants now.

This study's enrollment of 24 is close to the median of 26 across 120 interventional studies indexed under Neurofibromatoses.

Browse Neurofibromatoses studies →

Lead sponsor

Mayo Clinic is the lead sponsor of 3,218 studies on the registry; 670 are open to participants now.

Of its 445 completed or terminated interventional studies of FDA-regulated products, 313 (70%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 70 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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)
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Single group
Masking
None (open label)
Enrollment
24 participants (actual)

Interventions

  • DrugPirfenidone

    Capsule 800 mg TID, oral

06

What researchers measure

Primary outcomes

  1. tumor volume

    Time frame: 24 months

07

Study locations

No study locations are listed for this record.

08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 19, 2012, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT00754780
Lead sponsor
Mayo Clinic
First posted
Sep 18, 2008
Start date
Sep 2000
Primary completion
Aug 2004
Last update
Mar 19, 2012

Study contacts

Dusica Babovic-Vuksanovic, M.D.
principal investigator · Mayo Clinic

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Mar 2012. You cannot join it, but the record below documents what was studied.

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