Topical Rapamycin for Fibrofolliculomas

Maastricht University Medical Centre (MUMC)

Status and phase

Completed

Phase 3

Conditions

Birt-Hogg-Dubé Syndrome

Treatments

Drug: placebo

Drug: Rapamycin

Study type

Interventional

Funder types

Other

Identifiers

NCT00928798

09-2-058

Details and patient eligibility

About

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.

Enrollment

19 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.