Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1
The purpose of this study is to determine if imiquimod cream can reverse the growth of neurofibromas. Imiquimod is a skin cream that works by stimulating the body's immune system to respond to tumors. Imiquimod cream is approved for use in patients with various skin lesions, including actinic keratosis, superficial basal cell carcinoma, and external genital warts. Information from these studies, as well as previous laboratory studies, suggest that imiquimod cream may help shrink neurofibromas or keep them from growing.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Pilot Study of Topical Imiquimod 5% Cream for Treatment of Cutaneous Neurofibromas in Adults With Neurofibromatosis 1|
- To determine the effect of topical imiquimod 5% cream on tumor volume of cutaneous neurofibromas in adult subjects with neurofibromatosis 1 [ Time Frame: 2 years ] [ Designated as safety issue: No ]
- To correlate the inflammatory infiltrate adjacent to treated lesions during treatment with tumor response and to determine the number of circulating Tregs in this patient population [ Time Frame: 2 years ] [ Designated as safety issue: No ]
|Study Start Date:||March 2009|
|Estimated Study Completion Date:||December 2013|
|Primary Completion Date:||April 2010 (Final data collection date for primary outcome measure)|
Drug: Imiquimod 5% Cream
Applied topically to three tumors 5 times per week for a full 6 weeks
- Three of the participant's tumors will be treated with imiquimod cream and one tumor (out of all the remaining tumors) will be followed without treatment (control tumor).
- Participants will be given a tube of imiquimod cream and be asked to apply it to the three tumors 5 times per week, for a full 6 weeks (Monday through Friday).
- Participants will be required to come to the hospital for examinations on Week 1, 2, 4 and 6 (+/- 3 calendar days) and after their last dose of imiquimod cream on Weeks 9, 12 and 18 (+/- 5 calendar days. The following tests and procedures will be performed: skin test; vital signs and measurements and photographs of the tumors. Participants will be asked to participate in an optional skin biopsy on Week 4 and research blood tests wil lbe taken on Day 1.
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|Principal Investigator:||Scott R. Plotkin, MD, PhD||Massachusetts General Hospital|