Topical Rapamycin for Fibrofolliculomas

Phase 3

Completed

• Conditions: Birt-Hogg-Dubé Syndrome
• Interventions: Drug: Rapamycin; Drug: placebo

• Registration Number: NCT00928798
• Lead Sponsor: Maastricht University Medical Center

Brief Summary

The purpose of the study is to determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary we evaluate rapamycin safety, formula acceptance and patient satisfaction.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2009-06-25
• Study First Submitted QC: 2009-06-25
• Study First Posted: 2009-06-26
• Study Start: 2010-01
• Primary Completion: 2011-02
• Study Completion: 2011-08
• Status Verified: 2012-03
• Last Update Submitted: 2012-03-07
• Last Update Posted: 2012-03-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 19

Inclusion Criteria

• Minimum age of 18 years.
• At least 10 facial fibrofolliculomas, histologically confirmed.
• Entered in a screening program and free of malignancy as determined during screening (already had a baseline MRI or CT-scan).
• Being able to understand instructions.
• Mutation status must be known.
• For females: not pregnant and willing to use both oral and barrier contraceptives during the treatment period.

Exclusion Criteria

• Not capable of informed consent.
• Age under 18 years.
• Pregnancy or failure to comply with contraceptive measures.
• Proven or suspected malignancy of skin or other organs.
• No histological confirmation.
• Skin lesions other than fibrofolliculoma that might worsen under sirolimus such as active infections.
• Not able to comprehend instructions.
• No proven mutation.
• Less than 10 fibrofolliculomas.
• Planned facial surgery in the treatment period.
• Concomitant disease requiring systemic immunosuppressive treatment
• Concomitant disease requiring facial topical immunosuppressive treatment or facial topical drugs that interfere with rapamycin during trail period or in the 30 days before start trial.
• Tendency to form keloids or hypertrophic scars.
• Drug or alcohol abuse.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
rapamycin	placebo	one facial side rapamycin and one facial side placebo
rapamycin	Rapamycin	one facial side rapamycin and one facial side placebo

Primary Outcome Measures

Name	Time	Method
Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area.	3 and 6 months

Secondary Outcome Measures

Name	Time	Method
Side effects	3 and 6 months

Trial Locations

Locations (2)

Maastricht University Medical Centre; 🇳🇱 Maastricht, Netherlands

VU Medical Centre Amsterdam; 🇳🇱 Amsterdam, Netherlands