Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

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Brief Title

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

Official Title

A Phase 2/3, Multi-Center, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Response Comparison of the Efficacy and Safety of a Topical Rapamycin Cream for the Treatment of Facial Angiofibromas (FA) Associated With Tuberous Sclerosis Complex (TSC) in Patients 6 Years of Age and Over

Brief Summary

      The study aims to compare the safety and efficacy of two different strengths of Rapamycin
      cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA)
      associated with Tuberous Sclerosis Complex (TSC).
    

Detailed Description

      Topical rapamycin has previously been used to treat FA associated with TSC, reducing
      erythema, papule size, while flattening lesions and improving skin texture. Topical rapamycin
      has been reported to be well tolerated.

      The efficacy and safety of a topical rapamycin cream at two strengths (0.5% and 1.0%) will be
      assessed during a 26 week double-blind treatment phase with assessments made at clinical
      visits at baseline, 2, 8, 14, 20 and 26 weeks, and at follow-up (4 weeks after the last dose
      of study drug).
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

Percentage of participants obtaining successful treatment

Secondary Outcome

 Time to treatment success

Condition

Facial Angiofibroma

Intervention

rapamycin

Study Arms / Comparison Groups

 0.5% Rapamycin cream, topical
Description:  Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

120

Start Date

July 28, 2019

Completion Date

November 2021

Primary Completion Date

November 2021

Eligibility Criteria

        Inclusion Criteria:

          1. Male and female patients aged ≥ 6 years and ≤ 65 years on the day informed consent is
             obtained

          2. Patients diagnosed with TSC based on the clinical diagnostic criteria of International
             Tuberous Sclerosis Complex Consensus Conference 2012 and presenting visible facial
             angiofibroma

          3. An FA severity score of 2 or 3 on the IGA scale

          4. Patients or their legal representatives capable of understanding the explanation of
             the clinical trial and who give written informed consent for participation

          5. Patients or their legal representatives able to maintain patient diaries following the
             instructions of the investigator or sub-investigator

        Exclusion Criteria:

          1. Patients who cannot carry out the treatment plan or follow-up assessment

          2. Patients with serious skin lesions such as erosions or ulcers

          3. Patients with known hypersensitivity to any component of the study product

          4. Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3
             months of enrolment

          5. Patients who received laser therapy or surgical therapy within 6 months prior to trial
             enrolment

          6. Patients who participated in any other clinical trial within 3 months prior to the day
             of enrolment

          7. Patients judged unsuitable for this clinical trial by the investigator or
             sub-investigator

          8. Pregnant or lactating females

          9. Sexually active females of childbearing potential not using adequate contraception and
             sexually active males not using adequate contraception

         10. Patients with immune dysfunction or receiving any form of immunosuppression

         11. Patients with severe FA, with a score of 4 on the IGA scale

         12. Patients with an FA severity score of less than 2 on the IGA scale
      

Gender

All

Ages

6 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Ioana Stanescu, + 64 9 488 0232, [email protected]

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT03826628

Organization ID

DSLP-01


Responsible Party

Sponsor

Study Sponsor

Dermatology Specialties Limited Partnership


Study Sponsor

Ioana Stanescu, Study Director, Dermatology Specialties Limited Partnership


Verification Date

April 2021