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Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis

Primary Purpose

Facial Angiofibroma

Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Rapamycin
Calcitriol
Rapamycin-calcitriol combination
Sponsored by
National Taiwan University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Facial Angiofibroma focused on measuring Tuberous sclerosis complex, facial angiofibroma, rapamycin, calcitriol

Eligibility Criteria

7 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Subjects must have been diagnosed or highly suspected as having TSC.
  2. Subjects must be aged 7 to 70 years at Screening, and can be either sex.
  3. Subjects must have symmetric facial angiofibromas.

Exclusion Criteria:

  1. Pregnancy or with a plan to be pregnant.
  2. Subjects who cannot comply the treatment protocol.
  3. Subjects with kidney or liver/ biliary dysfunction.
  4. Subjects with hypercalcaemia and patients known to suffer from abnormal calcium metabolism.
  5. Subjects on systemic treatment of calcium deficiency.
  6. Subjects known to be hypersensitive to rapamycin or calcitriol.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Experimental

    Experimental

    Experimental

    Arm Label

    Rapamycin

    Calcitriol

    Rapamycin-calcitriol combination

    Arm Description

    Rapamycin(0.1%)

    Calcitriol(3mcg/g)

    Rapamycin(0.1%) with Calcitriol(3mcg/g)

    Outcomes

    Primary Outcome Measures

    facial angiofibroma severity index (FASI)
    the reduction of facial angiofibroma severity index (FASI) for the grade of erythema, papule size, elevation and extension of the lesions at week 12

    Secondary Outcome Measures

    Full Information

    First Posted
    April 26, 2017
    Last Updated
    May 2, 2017
    Sponsor
    National Taiwan University Hospital
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03140449
    Brief Title
    Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis
    Official Title
    Study of Combination Therapy With Topical Rapamycin and Calcitriol for Cutaneous Lesions of Tuberous Sclerosis: A Double-blind Randomized Controlled Trial
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    September 2013
    Overall Recruitment Status
    Completed
    Study Start Date
    September 5, 2013 (Actual)
    Primary Completion Date
    November 7, 2016 (Actual)
    Study Completion Date
    April 25, 2017 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    National Taiwan University Hospital

    4. Oversight

    5. Study Description

    Brief Summary
    The aim of the study is to determine the effect and safety of topical rapamycin or calcitriol and their combination for the treatment of TSC-associated facial angiofibroma. Methods: A total of 52 TSC patients including 20 male and 32 female subjects were recruited, and 50 of them completed the period 1 study. In period 1, topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. The primary end point was the reduction of facial angiofibroma severity index (FASI) for the grade of erythema, papule size, elevation and extension of the lesions at week 12. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24). A follow-up FASI analysis for recurrence after drug discontinuance for 12 weeks was also performed (week 36). The secondary end point was the reduction of Visual Analysis Score (VAS) evaluated by the subjects themselves at week 12.
    Detailed Description
    Background: Tuberous sclerosis complex (TSC)-associated facial angiofibroma is psychologically debilitating to both patients and their family members. The pathogenesis of TSC stems from TSC1 or TSC2 mutations, leading to the defect in mechanistic target of rapamycin (mTOR) inhibition. Rapamycin is an mTOR inhibitor and is effective for TSC facial angiofibroma through topical administration. Calcitriol, a vitamin D3 analogue, has been shown to lessen skin fibrosis in scleroderma and may be therapeutically beneficial to angiofibromas. Objectives: The aim of the study is to determine the effect and safety of topical rapamycin or calcitriol and their combination for the treatment of TSC-associated facial angiofibroma. Methods: A total of 52 TSC patients including 20 male and 32 female subjects were recruited, and 50 of them completed the period 1 study. In period 1, topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. The primary end point was the reduction of facial angiofibroma severity index (FASI) for the grade of erythema, papule size, elevation and extension of the lesions at week 12. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24). A follow-up FASI analysis for recurrence after drug discontinuance for 12 weeks was also performed (week 36). The secondary end point was the reduction of Visual Analysis Score (VAS) evaluated by the subjects themselves at week 12.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Facial Angiofibroma
    Keywords
    Tuberous sclerosis complex, facial angiofibroma, rapamycin, calcitriol

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Parallel Assignment
    Masking
    ParticipantCare ProviderInvestigator
    Allocation
    Randomized
    Enrollment
    52 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Rapamycin
    Arm Type
    Experimental
    Arm Description
    Rapamycin(0.1%)
    Arm Title
    Calcitriol
    Arm Type
    Experimental
    Arm Description
    Calcitriol(3mcg/g)
    Arm Title
    Rapamycin-calcitriol combination
    Arm Type
    Experimental
    Arm Description
    Rapamycin(0.1%) with Calcitriol(3mcg/g)
    Intervention Type
    Drug
    Intervention Name(s)
    Rapamycin
    Other Intervention Name(s)
    Sirolimus
    Intervention Description
    topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24).
    Intervention Type
    Drug
    Intervention Name(s)
    Calcitriol
    Other Intervention Name(s)
    Rocaltrol
    Intervention Description
    topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24).
    Intervention Type
    Drug
    Intervention Name(s)
    Rapamycin-calcitriol combination
    Other Intervention Name(s)
    Sirolimus-Rocaltrol combination
    Intervention Description
    topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24).
    Primary Outcome Measure Information:
    Title
    facial angiofibroma severity index (FASI)
    Description
    the reduction of facial angiofibroma severity index (FASI) for the grade of erythema, papule size, elevation and extension of the lesions at week 12
    Time Frame
    at week 12

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    7 Years
    Maximum Age & Unit of Time
    65 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Subjects must have been diagnosed or highly suspected as having TSC. Subjects must be aged 7 to 70 years at Screening, and can be either sex. Subjects must have symmetric facial angiofibromas. Exclusion Criteria: Pregnancy or with a plan to be pregnant. Subjects who cannot comply the treatment protocol. Subjects with kidney or liver/ biliary dysfunction. Subjects with hypercalcaemia and patients known to suffer from abnormal calcium metabolism. Subjects on systemic treatment of calcium deficiency. Subjects known to be hypersensitive to rapamycin or calcitriol.
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    YI-HUA LIAO, A.P.
    Organizational Affiliation
    A.P.
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    Undecided

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    Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis

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