Topical Rapamycin for Fibrofolliculomas

Birt-Hogg-Dubé Syndrome Clinical Trial

Official title:

Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00928798
• Other study ID #: 09-2-058
• Status: Completed
• Phase: Phase 3
• First received: June 25, 2009
• Last updated: March 7, 2012
• Start date: January 2010
• Est. completion date: August 2011

Study information

• Verified date: March 2012
• Source: Maastricht University Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: August 2011
• Est. primary completion date: February 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Birt-Hogg-Dube Syndrome
• Birt-Hogg-Dubé Syndrome

Intervention

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.
• 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

Locations

Country	Name	City	State
Netherlands	VU Medical Centre Amsterdam	Amsterdam
Netherlands	Maastricht University Medical Centre	Maastricht

Sponsors (2)

Lead Sponsor	Collaborator
Maastricht University Medical Center	Myrovlytis Trust

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area.		3 and 6 months	No
Secondary	Side effects		3 and 6 months	Yes