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Completed

NCT Number: NCT00928798

Topical Rapamycin for Fibrofolliculomas

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.

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Key information

Conditions

Birt-Hogg-Dubé Syndrome Birt-Hogg-Dube Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Neoplasms Neoplastic Syndromes, Hereditary

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

VU Medical Centre Amsterdam, Amsterdam, Netherlands

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Rapamycin

Drug

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

Placebo

Drug

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

Primary outcomes

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

    Time frame: 3 and 6 months

Secondary outcomes

  1. Side effects

    Time frame: 3 and 6 months

Sponsors and collaborators

Lead sponsor

Maastricht University Medical Center

Other

Collaborators

  • Myrovlytis Trust

Registry information

Official study title

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

Important dates

Study start
2010
Primary completion
2011
Study completion
2011
First posted
Jun 26, 2009
Registry last updated
Mar 8, 2012

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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