Clinical Trial: Topical Everolimus in Patients With Tuberous Sclerosis Complex

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Topical Everolimus Versus Placebo for the Treatment of Facial Angiofibromas in Patients With Tuberous Sclerosis Complex. A Phase II/III, Multicentre, Randomized, Double-bl

Brief Summary: Tuberous sclerosis complex (TSC) is a rare genetic multisystem disorder characterized by the development of hamartomas in several organs (e.g. brain, heart, kidney, liver, lung), and skin in more than 90% of cases. Facial angiofibromas (FA), present in about 80% of patients, are a stigmatizing hallmark of the disease. Everolimus could be a candidate for use as a topical formulation to treat FA. This adaptive seamless Phase II/III study primary objective is to determine the dose of topical everolimus for treatment of FA and evaluate the efficacy and safety of topical everolimus versus placebo in patients with angiofibromas.

Detailed Summary:
Sponsor: Hospices Civils de Lyon

Current Primary Outcome: Facial Angiofibroma Severity Index (FASI) [ Time Frame: 6 months ]

The FASI is a composite score summing the scores for erythema (0-3), size (0-3) and extension of FA (2:<50% of the cheek surface; 3:>50% of the cheek surface). The FASI will be centrally-measured on patient's face photographs by an independent and blinded adjudication committee of 2 dermatologists, with third-dermatologist review for disagreements.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • FA size [ Time Frame: 6 months ]
    FA size (in millimetres) of the 3 largest targeted FA papules previously identified by the investigator
  • Dermatologist's global assessment of efficacy [ Time Frame: 6 months ]
    using 7-point Likert scale
  • Patient or parents self-assessment [ Time Frame: 6 months ]
    using 7-point Likert scale
  • Local tolerance of the topically applied formulation using patient self-assessment [ Time Frame: 6 months ]
  • blood levels of topically applied everolimus [ Time Frame: 6 months ]
  • dryness score [ Time Frame: 6 months ]
    assessed by physicians
  • scaling scores [ Time Frame: 6 months ]
    assessed by physicians
  • Dermatological quality of life [ Time Frame: 6 months ]
    using DLQI (Dermatology Life Quality Index) for adults, CDLQI (Children's Dermatology Life Quality Index) for children


Original Secondary Outcome: Same as current

Information By: Hospices Civils de Lyon

Dates:
Date Received: August 4, 2016
Date Started: January 2017
Date Completion: April 2021
Last Updated: August 4, 2016
Last Verified: July 2016