Topical Rapamycin for Fibrofolliculomas | NCT00928798

Studies
Topical Rapamycin for Fibrofolliculomas

NCT00928798

Sponsor

Maastricht University Medical Center

Status

Completed

Last Update Posted

Mar 8, 2012Estimated

Enrollment

19Actual

Phase

Phase 3

Conditions

Birt-Hogg-Dubé Syndrome

Interventions

Rapamycin
placebo

Countries

Netherlands

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2012

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 2010

Primary Completion Date

Feb 2011Actual

Completion Date

Aug 2011Actual

First Submitted Date

Jun 25, 2009

First Submission Date that Met QC Criteria

Jun 25, 2009

First Posted Date

Jun 26, 2009Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 7, 2012

Last Update Posted Date

Mar 8, 2012Estimated

Name	Class
Myrovlytis Trust	UNKNOWN

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

19Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
rapamycin	Experimental	one facial side rapamycin and one facial side placebo	Drug: Rapamycin Drug: placebo

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Rapamycin	Drug	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.

Measure	Description	Time Frame
Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area.		3 and 6 months

Measure	Description	Time Frame
Side effects		3 and 6 months

Eligibility Module

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Maurice van Steensel, Dr.	Department of Dermatology	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
VU Medical Centre Amsterdam		Amsterdam		1081 HV	Netherlands
Maastricht University Medical Centre

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
24910976	Derived	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.

ID	Term
D009386	Neoplastic Syndromes, Hereditary
D009369	Neoplasms
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

ID	Term
D018942	Macrolides
D007783	Lactones
D009930	Organic Chemicals