Topical rapamycin to treat fibrofolliculomas in Birt-Hogg-Dubé syndrome

Phase 3

Completed

Conditions: fibrofolliculomas
hair follicle tumors
10083624
10040798

Registration Number: NL-OMON35713

Lead Sponsor: niversiteit Maastricht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 20

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 during the trial or within 30 days before start of therapy.
Concomitant disease requiring facial topical immunosuppressive treatment or facial topical drugs that interfere with rapamycin during trial period or within 30 days before start of therapy.
Tendency to form keloids or hypertrophic scars.
Drug or alcohol abuse.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Significant regression of lesions (reduction of fibrofolliculoma size and<br /><br>count) in the treated area. Defined as assessment by at least 2 of 3 observers<br /><br>with at least 2 points.<br /><br>The following 7-point scale is used:<br /><br>-3= strong worsening<br /><br>-2= moderate worsening<br /><br>-1= minimal worsening<br /><br>0= no improvement<br /><br>1= minimal improvement<br /><br>2= moderate improvement<br /><br>3= strong improvement</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary parameters/endpoints:<br /><br>The absence of any effect.<br /><br>Safety evaluation is assessed by tolerance and adverse event profiles.<br /><br>Formula acceptance is assessed with a 5-point rating scale en patient<br /><br>satisfaction with some statements.</p><br>