Phase 2
N=179
Topical Rapamycin to Erase Angiofibromas in TSC
Angiofibromas · Tuberous Sclerosis
Bottom Line
View on ClinicalTrials.gov: NCT01526356 ↗Enrolled (actual)
179
Serious AEs
2.2%
Results posted
Oct 2020
Primary outcome: Primary: Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score — -13.89; -12.71; -1.39 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Placebo (Drug); Rapamycin (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- The University of Texas Health Science Center, Houston
- Primary completion
- Aug 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score |
-13.89; -12.71; -1.39 | — |
| SECONDARY Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient |
45; 38; 13; 5; 11; 15 | — |
| SECONDARY Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI) |
-1.5; -3.2; -1.6 | — |
| SECONDARY Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI) |
-0.2; -1.7; -1.8 | — |
| SECONDARY Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI) |
-2.2; -1.8; 0.3 | — |
Summary
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT01526356). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.