Clinical trial · Interventional
Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream
NCT01853423CI-TRIAL-00023559completedPhase 1ClinicalTrials.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 objective of this study is to evaluate the safety and efficacy of a 0.1% formulation of rapamune cream in children with Tuberous Sclerosis Complex (TSC), ages 3 years and older, who have facial angiofibromas that would benefit from treatment.
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 |
|---|---|---|---|
| Facial Angiofibroma | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Rapamune | Drug | Sirolimus | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Rapamune
- description
- Small amount of 0.1% rapamune ointment applied topically to affected facial areas twice daily for the first two weeks, then once daily.
- interventionNames
- Drug: Rapamune
Primary outcomes (1)
- measure
- Reduction of facial angiofibroma with use of rapamune facial skin cream
- timeFrame
- 1 year
- description
- 1. Tolerability: Number of participants with adverse events 2. Efficacy: Skin assessment to measure improvement of facial angiofibromas 3. Safety: Rapamune levels (serum)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Years
- Maximum age
- 45 Years
Show eligibility criteria text
Inclusion Criteria:
* Patient or his / her legally authorize representative (LAR) must sign and date the approved informed consent prior to study participation or initiation of study procedures. If appropriate, the patient will give written or verbal consent. Surrogate consent will be obtained utilizing the "legally authorized representative (LAR)". The LAR must be an adult who has exhibited special care and concern for the subject, who is familiar with the subject's personal values, who is reasonably available, and who is willing to serve. No person who is identified as a protective order or other court order that directs that person to avoid contact with the subject shall be eligible to serve as the subject's LAR. Identification of LAR should normally be made using the following order of descending preference: Conservator, guardian, attorney in fact, subject's spouse (unless legally separated), the subject's adult child, the subject's parent, the subject's adult sibling, any other adult relative of the subject, or other adult who is familiar with the patient's personal values, who is reasonably available, and who is willing to serve.
1. Patient must have a diagnosis of Tuberous Sclerosis Complex.
2. Female or male patients over the age of 3 years.
3. Female subjects of child bearing potential must not be pregnant and must undergo a pregnancy test, and must agree to use appropriate contraceptive methods
Exclusion Criteria:
* Patients will be excluded from entry into the study if any of the following are true:
1. Patient has a history of drug allergy to rapamune.
2. Patient is pregnant or lactating.
3. Subject is receiving therapy with Rapamycin.
4. Subject is receiving any form of immunosuppression or has previously experienced immune dysfunction.
5. Subject is currently participating in or has participated within the Last 30 days in a clinical trial involving an investigative drug.
6. Subject has other dermatologic conditions that would preclude or prevent adequate assessment of changes to their facial angiofibromas.
7. Subject has had laser surgery, cryotherapy, or other dermatologic treatment to their facial angiofibromas within the previous 6 months.References
Publications (0)
Data not yet available
No reference posted for this study.