Back to resultsClinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
Primary Purpose
Neurofibromatosis
Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Pirfenidone
Sponsored by
About this trial
This is an interventional treatment trial for Neurofibromatosis focused on measuring NF1, plexiform neurofibroma
Eligibility 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)
Sites / Locations
Outcomes
Primary Outcome Measures
tumor volume
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00754780
Brief Title
Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
Official Title
Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I
Study Type
Interventional
2. Study Status
Record Verification Date
March 2012
Overall Recruitment Status
Completed
Study Start Date
September 2000 (undefined)
Primary Completion Date
August 2004 (Actual)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
Mayo Clinic
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief 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.
Detailed Description
Specific aims of this study are:
To evaluate efficacy of Pirfenidone in NF1 patients with disfiguring or disabling plexiform neurofibroma (PN) and spinal neurofibromas (SN)
To determine the acute, subacute and chronic toxicity of Pirfenidone in patients with NF1.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurofibromatosis
Keywords
NF1, plexiform neurofibroma
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
24 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Pirfenidone
Intervention Description
Capsule 800 mg TID, oral
Primary Outcome Measure Information:
Title
tumor volume
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility 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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dusica Babovic-Vuksanovic, M.D.
Organizational Affiliation
Mayo Clinic
Official's Role
Principal Investigator
12. IPD Sharing Statement
Links:
URL
http://clinicaltrials.mayo.edu
Description
Mayo Clinic Clinical Trials
Learn more about this trial
Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
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