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Trial record 2 of 2 for:    birt hogg dube
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Topical Rapamycin for Fibrofolliculomas

This study has been completed.
Sponsor:
Collaborator:
Myrovlytis Trust
Information provided by (Responsible Party):
Maastricht University Medical Center
ClinicalTrials.gov Identifier:
NCT00928798
First received: June 25, 2009
Last updated: March 7, 2012
Last verified: March 2012
History of Changes
  Purpose

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:

U.S. FDA Resources

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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00928798

Locations
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
Principal Investigator: Maurice van Steensel, Dr. Department of Dermatology
  More Information

No publications provided

Responsible Party: Maastricht University Medical Center
ClinicalTrials.gov 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
Neoplasms
Genetic Diseases, Inborn
Sirolimus
Everolimus
Antibiotics, Antineoplastic
Antineoplastic Agents
 Therapeutic Uses
Pharmacologic Actions
Antifungal Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents

ClinicalTrials.gov processed this record on May 23, 2013