Topical Rapamycin for Fibrofolliculomas

Maastricht University Medical Center19 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Birt-Hogg-Dubé Syndrome

Interventions

RapamycinDRUG

Application of rapamycin 1 mg/ml oral solution to one predefined skin area on one facial half, twice daily 0,25 ml, during 6 months. The other facial half will be similarly treated with a placebo.

placeboDRUG

Application of placebo to one predefined skin area on one facial half, during 6 months. The other facial half will be similarly treated with rapamycin 1 mg/ml oral solution

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (2)

VU Medical Centre Amsterdam

Amsterdam, Netherlands

Maastricht University Medical Centre

Maastricht, Netherlands

Outcomes

Primary Outcomes

Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area.

Time frame: 3 and 6 months

Secondary Outcomes

Side effects

Time frame: 3 and 6 months

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.