Topical Rapamycin for Fibrofolliculomas

This study has been completed.
Myrovlytis Trust
Information provided by (Responsible Party):
Maastricht University Medical Center Identifier:
First received: June 25, 2009
Last updated: March 7, 2012
Last verified: March 2012

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.

Condition Intervention Phase
Birt-Hogg-Dubé Syndrome
Drug: Rapamycin
Drug: placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome

Resource links provided by NLM:

Further study details as provided by Maastricht University Medical Center:

Primary Outcome Measures:
  • Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area. [ Time Frame: 3 and 6 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Side effects [ Time Frame: 3 and 6 months ] [ Designated as safety issue: Yes ]

Enrollment: 19
Study Start Date: January 2010
Study Completion Date: August 2011
Primary Completion Date: February 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: rapamycin
one facial side rapamycin and one facial side placebo
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


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
Please refer to this study by its identifier: NCT00928798

VU Medical Centre Amsterdam
Amsterdam, Netherlands, 1081 HV
Maastricht University Medical Centre
Maastricht, Netherlands, 6229 HX
Sponsors and Collaborators
Maastricht University Medical Center
Myrovlytis Trust
Principal Investigator: Maurice van Steensel, Dr. Department of Dermatology
  More Information

No publications provided

Responsible Party: Maastricht University Medical Center Identifier: NCT00928798     History of Changes
Other Study ID Numbers: 09-2-058
Study First Received: June 25, 2009
Last Updated: March 7, 2012
Health Authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Additional relevant MeSH terms:
Birt-Hogg-Dube Syndrome
Neoplastic Syndromes, Hereditary
Genetic Diseases, Inborn
Antibiotics, Antineoplastic
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antifungal Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents processed this record on April 15, 2014