NCT00928798

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
19

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jan 2010

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 25, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 26, 2009

Completed
6 months until next milestone

Study Start

First participant enrolled

January 1, 2010

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2011

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2011

Completed
Last Updated

March 8, 2012

Status Verified

March 1, 2012

Enrollment Period

1.1 years

First QC Date

June 25, 2009

Last Update Submit

March 7, 2012

Conditions

Birt-Hogg-Dubé Syndrome

Outcome Measures

Primary Outcomes (1)

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

    3 and 6 months

Secondary Outcomes (1)

  • Side effects

    3 and 6 months

Study Arms (1)

rapamycin

EXPERIMENTAL

one facial side rapamycin and one facial side placebo

Drug: RapamycinDrug: placebo

Interventions

RapamycinDRUG

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.

Also known as: Rapamune, Sirolimus
rapamycin
placeboDRUG

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

rapamycin

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

VU Medical Centre Amsterdam

Amsterdam, 1081 HV, Netherlands

Location

Maastricht University Medical Centre

Maastricht, 6229 HX, Netherlands

Location

Related Publications (1)

  • 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.

    PMID: 24910976DERIVED

MeSH Terms

Conditions

Birt-Hogg-Dube Syndrome

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Neoplastic Syndromes, HereditaryNeoplasmsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Officials

  • Maurice van Steensel, Dr.

    Department of Dermatology

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 25, 2009

First Posted

June 26, 2009

Study Start

January 1, 2010

Primary Completion

February 1, 2011

Study Completion

August 1, 2011

Last Updated

March 8, 2012

Record last verified: 2012-03

Locations

NL(2)