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Topical Rapamycin for Fibrofolliculomas

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00928798
Recruitment Status : Completed
First Posted : June 26, 2009
Last Update Posted : March 8, 2012
Sponsor:
Collaborator:
Myrovlytis Trust
Information provided by (Responsible Party):
Maastricht University Medical Center

Study Description
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Brief Summary:
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 or disease Intervention/treatment Phase
Birt-Hogg-Dubé Syndrome Drug: Rapamycin Drug: placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome
Study Start Date : January 2010
Actual Primary Completion Date : February 2011
Actual Study Completion Date : August 2011

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Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area. [ Time Frame: 3 and 6 months ]

Secondary Outcome Measures :
  1. Side effects [ Time Frame: 3 and 6 months ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00928798


Locations
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Netherlands
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
Investigators
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Principal Investigator: Maurice van Steensel, Dr. Department of Dermatology
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Maastricht University Medical Center
ClinicalTrials.gov Identifier: NCT00928798    
Other Study ID Numbers: 09-2-058
First Posted: June 26, 2009    Key Record Dates
Last Update Posted: March 8, 2012
Last Verified: March 2012
Additional relevant MeSH terms:
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Birt-Hogg-Dube Syndrome
Syndrome
Disease
Pathologic Processes
Neoplastic Syndromes, Hereditary
Neoplasms
Genetic Diseases, Inborn
Sirolimus
 Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs