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Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)

Primary Purpose

Angiofibromas, Tuberous Sclerosis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Placebo
Rapamycin
Rapamycin
Sponsored by
The University of Texas Health Science Center, Houston
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Angiofibromas focused on measuring Angiofibroma, Tuberous Sclerosis, Rapamycin, Sirolimus, mTOR

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Alabama at Birmingham
  • UCLA Mattel Children's Hospital
  • Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center
  • Kennedy Krieger Institute
  • Herscot Center for Adults and Children with TSC Massachusetts General Hospital
  • Clinic Without Walls
  • Cincinnati Children's Hospital Medical Center
  • Texas Scottish Rite Hospital
  • The University of Texas Medical School at Houston
  • Sydney Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Placebo Comparator

Active Comparator

Active Comparator

Arm Label

Placebo

0.1 % Rapamycin

1% Rapamycin

Arm Description

Cream only

0.1% Rapamycin cream

1% Rapamycin cream

Outcomes

Primary Outcome Measures

Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score
Subject's lesion/s will be digitally photographed before initial application (visit 1), at each study visit (visits 2 through 6), and immediately prior to study termination (visit 7). Following completion of the study, all photographs will be evaluated by two independent dermatologists blinded to both the treatment arm and the stage of treatment. The dermatologists will assess each photograph's appearance using the facial Angiofibroma Grading Scale (AGS), which assesses the forehead, nose, cheeks, and chin for erythema, average lesion size, lesion density, and percent involvement, as well as presence of any pedunculated angiofibromas. A total score is reported with a range of 0 to 202, with higher scores representing a worse outcome.

Secondary Outcome Measures

Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)
The Dermatology life Quality Index (DLQI) is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. It is designed for people aged 16 years and above. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)
The Children's Dermatology life Quality Index (DLQI) is validated from the age of four years to 16 years. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)
The Family Dermatology life Quality Index (FLQI) is designed for adult (more than 16 years of age) family members or partners of patients (of any age) with any skin disease. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).

Full Information

First Posted
February 1, 2012
Last Updated
September 30, 2020
Sponsor
The University of Texas Health Science Center, Houston
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1. Study Identification

Unique Protocol Identification Number
NCT01526356
Brief Title
Topical Rapamycin to Erase Angiofibromas in TSC
Acronym
Treatment
Official Title
Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
Study Type
Interventional

2. Study Status

Record Verification Date
September 2020
Overall Recruitment Status
Completed
Study Start Date
May 2012 (undefined)
Primary Completion Date
August 2014 (Actual)
Study Completion Date
August 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
The University of Texas Health Science Center, Houston

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Angiofibromas, Tuberous Sclerosis
Keywords
Angiofibroma, Tuberous Sclerosis, Rapamycin, Sirolimus, mTOR

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
179 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Cream only
Arm Title
0.1 % Rapamycin
Arm Type
Active Comparator
Arm Description
0.1% Rapamycin cream
Arm Title
1% Rapamycin
Arm Type
Active Comparator
Arm Description
1% Rapamycin cream
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Rapamycin
Intervention Description
Study cream is applied nightly to the affected areas on the face.
Intervention Type
Drug
Intervention Name(s)
Rapamycin
Intervention Description
Study cream is applied nightly to the affected areas on the face. Low Dose
Intervention Type
Drug
Intervention Name(s)
Rapamycin
Intervention Description
Study cream is applied nightly to the affected areas on the face. High Dose
Primary Outcome Measure Information:
Title
Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score
Description
Subject's lesion/s will be digitally photographed before initial application (visit 1), at each study visit (visits 2 through 6), and immediately prior to study termination (visit 7). Following completion of the study, all photographs will be evaluated by two independent dermatologists blinded to both the treatment arm and the stage of treatment. The dermatologists will assess each photograph's appearance using the facial Angiofibroma Grading Scale (AGS), which assesses the forehead, nose, cheeks, and chin for erythema, average lesion size, lesion density, and percent involvement, as well as presence of any pedunculated angiofibromas. A total score is reported with a range of 0 to 202, with higher scores representing a worse outcome.
Time Frame
baseline, 6 months
Secondary Outcome Measure Information:
Title
Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient
Time Frame
baseline, 6 months
Title
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)
Description
The Dermatology life Quality Index (DLQI) is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. It is designed for people aged 16 years and above. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).
Time Frame
baseline, 6 months
Title
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)
Description
The Children's Dermatology life Quality Index (DLQI) is validated from the age of four years to 16 years. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).
Time Frame
baseline, 6 months
Title
Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)
Description
The Family Dermatology life Quality Index (FLQI) is designed for adult (more than 16 years of age) family members or partners of patients (of any age) with any skin disease. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).
Time Frame
baseline, 6 months
Other Pre-specified Outcome Measures:
Title
Number of Events of Dermatologic Sensitivity at the Site of Application
Description
Dermatologic sensitivity includes pain, pruritis, or erythema at the application site.
Time Frame
6 months
Title
Number of Participants With Systemic Uptake of Topically Applied Rapamycin
Description
Blood levels checked to confirm the lack of systemic rapamycin.
Time Frame
6 months

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mary Kay Koenig, MD
Organizational Affiliation
The University of Texas Medical School at Houston
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Hope Northrup, MD
Organizational Affiliation
The University of Texas Medical School at Houston
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
UCLA Mattel Children's Hospital
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Kennedy Krieger Institute
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Herscot Center for Adults and Children with TSC Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Clinic Without Walls
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55102-2697
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Texas Scottish Rite Hospital
City
Dallas
State/Province
Texas
ZIP/Postal Code
75219
Country
United States
Facility Name
The University of Texas Medical School at Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Sydney Children's Hospital
City
Sydney
State/Province
New South Wales
Country
Australia

12. IPD Sharing Statement

Citations:
PubMed Identifier
29800048
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.
Results Reference
derived

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Topical Rapamycin to Erase Angiofibromas in TSC

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