Trial | NCT00928798  Report issue

Title

Topical Rapamycin for Fibrofolliculomas
Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome

  • Phase

    Phase 3

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Intervention/Treatment

    sirolimus ...

  • Study Participants

    19
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 Started
Jan 31
2010
Primary Completion
Feb 28
2011
Study Completion
Aug 31
2011
Last Update
Mar 08
2012
Estimate

Drug Rapamycin

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.

  • Other names: Rapamune, Sirolimus

Drug placebo

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

rapamycin Experimental

one facial side rapamycin and one facial side placebo

Criteria 

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.
No Results Posted