Topical Rapamycin for Fibrofolliculomas
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| ClinicalTrials.gov Identifier: NCT00928798 |
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Recruitment Status :
Completed
First Posted : June 26, 2009
Last Update Posted : March 8, 2012
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Sponsor:
Maastricht University Medical Center
Collaborator:
Myrovlytis Trust
Information provided by (Responsible Party):
Maastricht University Medical Center
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Birt-Hogg-Dubé Syndrome | Drug: Rapamycin Drug: placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 19 participants |
| Allocation: | Randomized |
| Intervention Model: | Single Group Assignment |
| Masking: | Double (Participant, Investigator) |
| Primary Purpose: | Treatment |
| Official Title: | Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome |
| Study Start Date : | January 2010 |
| Actual Primary Completion Date : | February 2011 |
| Actual Study Completion Date : | August 2011 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Birt-Hogg-Dubé syndrome
| Arm | Intervention/treatment |
|---|---|
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Experimental: rapamycin
one facial side rapamycin and one facial side placebo
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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.
Other Names:
Drug: 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 |
Primary Outcome Measures :
- Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area. [ Time Frame: 3 and 6 months ]
Secondary Outcome Measures :
- Side effects [ Time Frame: 3 and 6 months ]
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00928798
Locations
| Netherlands | |
| VU Medical Centre Amsterdam | |
| Amsterdam, Netherlands, 1081 HV | |
| Maastricht University Medical Centre | |
| Maastricht, Netherlands, 6229 HX | |
Sponsors and Collaborators
Maastricht University Medical Center
Myrovlytis Trust
Investigators
| Principal Investigator: | Maurice van Steensel, Dr. | Department of Dermatology |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Maastricht University Medical Center |
| ClinicalTrials.gov Identifier: | NCT00928798 |
| Other Study ID Numbers: |
09-2-058 |
| First Posted: | June 26, 2009 Key Record Dates |
| Last Update Posted: | March 8, 2012 |
| Last Verified: | March 2012 |
Additional relevant MeSH terms:
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Birt-Hogg-Dube Syndrome Syndrome Disease Pathologic Processes Neoplastic Syndromes, Hereditary Neoplasms Genetic Diseases, Inborn Sirolimus |
Anti-Bacterial Agents Anti-Infective Agents Antibiotics, Antineoplastic Antineoplastic Agents Antifungal Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |