Clinical Trial of Pirfenidone in Adult Patients With Neurofibromatosis 1
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00754780|
Recruitment Status : Completed
First Posted : September 18, 2008
Last Update Posted : March 19, 2012
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.
|Condition or disease||Intervention/treatment||Phase|
|Neurofibromatosis||Drug: Pirfenidone||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Clinical Trial of Pirfenidone for the Treatment of Patients With Neurofibromatosis Type I|
|Study Start Date :||September 2000|
|Actual Primary Completion Date :||August 2004|
- tumor volume [ Time Frame: 24 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00754780
|Principal Investigator:||Dusica Babovic-Vuksanovic, M.D.||Mayo Clinic|