search
Back to results

Topical Rapamycin for Fibrofolliculomas

Primary Purpose

Birt-Hogg-Dubé Syndrome

Status
Completed
Phase
Phase 3
Locations
Netherlands
Study Type
Interventional
Intervention
Rapamycin
placebo
Sponsored by
Maastricht University Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Birt-Hogg-Dubé Syndrome

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • VU Medical Centre Amsterdam
  • Maastricht University Medical Centre

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

rapamycin

Arm Description

one facial side rapamycin and one facial side placebo

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Side effects

Full Information

First Posted
June 25, 2009
Last Updated
March 7, 2012
Sponsor
Maastricht University Medical Center
Collaborators
Myrovlytis Trust
search

1. Study Identification

Unique Protocol Identification Number
NCT00928798
Brief Title
Topical Rapamycin for Fibrofolliculomas
Official Title
Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
March 2012
Overall Recruitment Status
Completed
Study Start Date
January 2010 (undefined)
Primary Completion Date
February 2011 (Actual)
Study Completion Date
August 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Maastricht University Medical Center
Collaborators
Myrovlytis Trust

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Birt-Hogg-Dubé Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
19 (Actual)

8. Arms, Groups, and Interventions

Arm Title
rapamycin
Arm Type
Experimental
Arm Description
one facial side rapamycin and one facial side placebo
Intervention Type
Drug
Intervention Name(s)
Rapamycin
Other Intervention Name(s)
Rapamune, Sirolimus
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
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 Outcome Measure Information:
Title
Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area.
Time Frame
3 and 6 months
Secondary Outcome Measure Information:
Title
Side effects
Time Frame
3 and 6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Maurice van Steensel, Dr.
Organizational Affiliation
Department of Dermatology
Official's Role
Principal Investigator
Facility Information:
Facility Name
VU Medical Centre Amsterdam
City
Amsterdam
ZIP/Postal Code
1081 HV
Country
Netherlands
Facility Name
Maastricht University Medical Centre
City
Maastricht
ZIP/Postal Code
6229 HX
Country
Netherlands

12. IPD Sharing Statement

Citations:
PubMed Identifier
24910976
Citation
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-Dube 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.
Results Reference
derived

Learn more about this trial

Topical Rapamycin for Fibrofolliculomas

We'll reach out to this number within 24 hrs