Clinical trial NCT01526356: Topical Rapamycin to Erase Angiofibromas in TSC
Official title: Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Angiofibromas
Tuberous Sclerosis
Keywords: Angiofibroma
Tuberous Sclerosis
Rapamycin
Sirolimus
mTOR
Lead sponsor: The University of Texas Health Science Center, Houston
Sponsor class: OTHER
First posted: 2012-02-03
Enrollment: 179
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

Primary outcomes:
measure: Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score
timeFrame: baseline, 6 months
Secondary outcomes:
measure: Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient
timeFrame: baseline, 6 months
measure: Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)
timeFrame: baseline, 6 months
measure: Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)
timeFrame: baseline, 6 months
measure: Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)
timeFrame: baseline, 6 months
Other outcomes:
measure: Number of Events of Dermatologic Sensitivity at the Site of Application
timeFrame: 6 months
measure: Number of Participants With Systemic Uptake of Topically Applied Rapamycin
timeFrame: 6 months
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Los Angeles
country: United States
facility: UCLA Mattel Children's Hospital
state: California
city: Oakland
country: United States
facility: Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center
state: California
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Boston
country: United States
facility: Herscot Center for Adults and Children with TSC Massachusetts General Hospital
state: Massachusetts
city: Saint Paul
country: United States
facility: Clinic Without Walls
state: Minnesota
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Dallas
country: United States
facility: Texas Scottish Rite Hospital
state: Texas
city: Houston
country: United States
facility: The University of Texas Medical School at Houston
state: Texas
city: Sydney
country: Australia
facility: Sydney Children's Hospital
state: New South Wales
Study officials:
affiliation: The University of Texas Medical School at Houston
name: Mary Kay Koenig, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: The University of Texas Medical School at Houston
name: Hope Northrup, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Koenig 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.