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Topical Rapamycin to Erase Angiofibromas in TSC (Treatment)

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ClinicalTrials.gov Identifier: NCT01526356
Recruitment Status : Completed
First Posted : February 3, 2012
Results First Posted : October 26, 2020
Last Update Posted : October 26, 2020
Sponsor:
Information provided by (Responsible Party):
Mary Kay Koenig, The University of Texas Health Science Center, Houston

Tracking Information
First Submitted Date  ICMJE February 1, 2012
First Posted Date  ICMJE February 3, 2012
Results First Submitted Date  ICMJE September 30, 2020
Results First Posted Date  ICMJE October 26, 2020
Last Update Posted Date October 26, 2020
Study Start Date  ICMJE May 2012
Actual Primary Completion Date August 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 30, 2020)
Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score [ Time Frame: baseline, 6 months ]
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.
Original Primary Outcome Measures  ICMJE
 (submitted: February 2, 2012)
Reduction in lesion size and appearance [ Time Frame: 6 months ]
Photographic, visual, and measurable reduction in the size and severity of the lesions.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 30, 2020)
  • Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient [ Time Frame: baseline, 6 months ]
  • Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI) [ Time Frame: baseline, 6 months ]
    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) [ Time Frame: baseline, 6 months ]
    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) [ Time Frame: baseline, 6 months ]
    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).
Original Secondary Outcome Measures  ICMJE
 (submitted: February 2, 2012)
  • Confirm the lack of systemic uptake of topically applied rapamycin. [ Time Frame: 6 months ]
    Blood levels checked to confirm the lack of systemic rapamycin.
  • Dermatologic sensitivity at the site of application. [ Time Frame: 6 months ]
    Pain, erythema, or pruritis at the application site.
Current Other Pre-specified Outcome Measures
 (submitted: September 30, 2020)
  • Number of Events of Dermatologic Sensitivity at the Site of Application [ Time Frame: 6 months ]
    Dermatologic sensitivity includes pain, pruritis, or erythema at the application site.
  • Number of Participants With Systemic Uptake of Topically Applied Rapamycin [ Time Frame: 6 months ]
    Blood levels checked to confirm the lack of systemic rapamycin.
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Topical Rapamycin to Erase Angiofibromas in TSC
Official Title  ICMJE Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Angiofibromas
  • Tuberous Sclerosis
Intervention  ICMJE
  • Drug: Placebo
    Study cream is applied nightly to the affected areas on the face.
    Other Name: Rapamycin
  • Drug: Rapamycin
    Study cream is applied nightly to the affected areas on the face. Low Dose
  • Drug: Rapamycin
    Study cream is applied nightly to the affected areas on the face. High Dose
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Cream only
    Intervention: Drug: Placebo
  • Active Comparator: 0.1 % Rapamycin
    0.1% Rapamycin cream
    Intervention: Drug: Rapamycin
  • Active Comparator: 1% Rapamycin
    1% Rapamycin cream
    Intervention: Drug: Rapamycin
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 30, 2020)
179
Original Estimated Enrollment  ICMJE
 (submitted: February 2, 2012)
230
Actual Study Completion Date  ICMJE August 2014
Actual Primary Completion Date August 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01526356
Other Study ID Numbers  ICMJE HSC-MS-11-0501
Department of Defense USAMRMC ( Other Grant/Funding Number: W81XWH-11-1-0240 )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Mary Kay Koenig, The University of Texas Health Science Center, Houston
Study Sponsor  ICMJE The University of Texas Health Science Center, Houston
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Mary Kay Koenig, MD The University of Texas Medical School at Houston
Principal Investigator: Hope Northrup, MD The University of Texas Medical School at Houston
PRS Account The University of Texas Health Science Center, Houston
Verification Date September 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP