Clinical trial · Interventional
Topical Rapamycin for Fibrofolliculomas
Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome
NCT00928798CI-TRIAL-00006412completedPhase 3ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Birt-Hogg-Dubé Syndrome | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| placebo | Drug | — | UNRESOLVED |
| Rapamycin | Drug | Sirolimus | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- rapamycin
- description
- one facial side rapamycin and one facial side placebo
- interventionNames
- Drug: Rapamycin
- Drug: placebo
Primary outcomes (1)
- measure
- Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area.
- timeFrame
- 3 and 6 months
Secondary outcomes (1)
- measure
- Side effects
- timeFrame
- 3 and 6 months
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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.
References
Publications (1)
- DERIVEDGijezen 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 24910976