Clinical trial · Interventional
Topical Rapamycin to Erase Angiofibromas in TSC
Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
- 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 study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous Sclerosis Complex (TSC). Subjects will apply either the topical vehicle containing rapamycin or the topical vehicle alone nightly to their angiofibromas for six months. The primary goal of this study is to evaluate the efficacy of the topical medication for reduction of cutaneous angiofibromas in patients with TSC. The secondary goal of this study is to confirm the safety of the topical medication.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Angiofibromas | Angiofibroma | ALIAS | 0.90 |
| Tuberous Sclerosis | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Placebo | Drug | — | UNRESOLVED |
| Rapamycin | Drug | Sirolimus | ALIAS |
Design
Arms and outcomes
Arms (3)
- type
- PLACEBO_COMPARATOR
- label
- Placebo
- description
- Cream only
- interventionNames
- Drug: Placebo
- type
- ACTIVE_COMPARATOR
- label
- 0.1 % Rapamycin
- description
- 0.1% Rapamycin cream
- interventionNames
- Drug: Rapamycin
- type
- ACTIVE_COMPARATOR
- label
- 1% Rapamycin
- description
- 1% Rapamycin cream
- interventionNames
Eligibility
Eligibility (as posted)
- Sex
- All
Show eligibility criteria text
Inclusion Criteria: * Subjects must be willing and able to comply with all trial requirements. * Subject has a diagnosis of TSC and has visible facial angiofibromas. * Female subjects of child bearing potential must not be pregnant and must agree to use appropriate contraceptive methods . Exclusion Criteria: * Subject is currently receiving therapy with Rapamycin. * Subject is receiving any form of immunosuppression or has previously experienced immune dysfunction. * Subject is currently participating in or has participated within the last 30 days in a clinical trial involving an investigational drug. * Subject has a known hypersensitivity to either the vehicle or Rapamycin. * Subject is a pregnant or nursing female. * Subject has other dermatologic conditions that would preclude or prevent adequate assessment of changes to their facial angiofibromas. * Subject has had laser surgery, cryotherapy, or other dermatologic treatment to their facial angiofibromas within the previous 6 months.
References
Publications (1)
- DERIVEDKoenig MK, Bell CS, Hebert AA, Roberson J, Samuels JA, Slopis JM, Tate P, Northrup H; TREATMENT Trial Collaborators. Efficacy and Safety of Topical Rapamycin in Patients With Facial Angiofibromas Secondary to Tuberous Sclerosis Complex: The TREATMENT Randomized Clinical Trial. JAMA Dermatol. 2018 Jul 1;154(7):773-780. doi: 10.1001/jamadermatol.2018.0464. PMID 29800048