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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

Tracking Information
First Submitted Date  ICMJE June 25, 2009
First Posted Date  ICMJE June 26, 2009
Last Update Posted Date March 8, 2012
Study Start Date  ICMJE January 2010
Actual Primary Completion Date February 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 25, 2009)
Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area. [ Time Frame: 3 and 6 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT00928798 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 25, 2009)
Side effects [ Time Frame: 3 and 6 months ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Topical Rapamycin for Fibrofolliculomas
Official Title  ICMJE Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Birt-Hogg-Dubé Syndrome
Intervention  ICMJE
  • 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
Study Arms  ICMJE Experimental: rapamycin
one facial side rapamycin and one facial side placebo
Interventions:
  • Drug: Rapamycin
  • Drug: placebo
Publications * Gijezen LM, Vernooij M, Martens H, Oduber CE, Henquet CJ, Starink TM, Prins MH, Menko FH, Nelemans PJ, van Steensel MA. Topical rapamycin as a treatment for fibrofolliculomas in Birt-Hogg-Dubé syndrome: a double-blind placebo-controlled randomized split-face trial. PLoS One. 2014 Jun 9;9(6):e99071. doi: 10.1371/journal.pone.0099071. eCollection 2014.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 7, 2012)
19
Original Estimated Enrollment  ICMJE
 (submitted: June 25, 2009)
20
Actual Study Completion Date  ICMJE August 2011
Actual Primary Completion Date February 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Netherlands
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00928798
Other Study ID Numbers  ICMJE 09-2-058
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Maastricht University Medical Center
Study Sponsor  ICMJE Maastricht University Medical Center
Collaborators  ICMJE Myrovlytis Trust
Investigators  ICMJE
Principal Investigator: Maurice van Steensel, Dr. Department of Dermatology
PRS Account Maastricht University Medical Center
Verification Date March 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP