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Completed

NCT Number: NCT01526356

Topical Rapamycin to Erase Angiofibromas in TSC

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.

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Key information

Conditions

Angiofibromas Angiofibroma Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hamartoma Heredodegenerative Disorders, Nervous System Malformations of Cortical Development Malformations of Cortical Development, Group I Neoplasms Neoplasms by Histologic Type Neoplasms, Multiple Primary Neoplasms, Vascular Tissue Neoplastic Syndromes, Hereditary Nervous System Diseases Nervous System Malformations Neurocutaneous Syndromes Neurodegenerative Diseases Tuberous Sclerosis

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Sydney Children's Hospital, Sydney, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Placebo

Drug

Study cream is applied nightly to the affected areas on the face.

Other names: Rapamycin

Rapamycin

Drug

Study cream is applied nightly to the affected areas on the face. Low Dose

Primary outcomes

  1. 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.

Secondary outcomes

  1. Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient

    Time frame: baseline, 6 months

  2. 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).

  3. 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).

  4. 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).

Other outcomes

  1. 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.

  2. Number of Participants With Systemic Uptake of Topically Applied Rapamycin

    Time frame: 6 months

    Blood levels checked to confirm the lack of systemic rapamycin.

Sponsors and collaborators

Lead sponsor

The University of Texas Health Science Center, Houston

Other

Registry information

Official study title

Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy

Acronym: Treatment

Important dates

Study start
2012
Primary completion
2014
Study completion
2014
First posted
Feb 3, 2012
Registry last updated
Oct 26, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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