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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03826628
Recruitment Status : Completed
First Posted : February 1, 2019
Last Update Posted : July 28, 2022
Sponsor:
Information provided by (Responsible Party):
Dermatology Specialties Limited Partnership

Study Description
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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).

Condition or disease Intervention/treatment Phase
Facial Angiofibroma Tuberous Sclerosis Drug: rapamycin Drug: placebo Phase 2 Phase 3

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 107 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : July 28, 2019
Actual Primary Completion Date : July 12, 2022
Actual Study Completion Date : July 12, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 0.5% Rapamycin cream, topical
Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks
 Drug: rapamycin
Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks
Other Name: sirolimus
Experimental: 1.0% Rapamycin cream, topical
Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks
 Drug: rapamycin
Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks
Other Name: sirolimus
Placebo Comparator: Placebo
Placebo cream topical, applied once daily before bed on affected area for 26 weeks
 Drug: placebo
Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, for 26 weeks


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of participants obtaining successful treatment [ Time Frame: After 26 weeks treatment ]
    Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline


Secondary Outcome Measures :
  1. Time to treatment success [ Time Frame: From first dose to 26 weeks ]
    The time elapsed from the first dose to the time of treatment success, according to the IGA scale

  2. Change from baseline in IGA [ Time Frame: At baseline and after 26 weeks treatment ]
    The change in grading on the IGA scale from baseline

  3. Change from baseline in FASI [ Time Frame: At baseline and after 26 weeks treatment ]
    The change in grading on the Facial Angiofibroma Severity Index (FASI) from baseline

  4. Subjective (participant or parent/caregiver) improvement rating [ Time Frame: After 26 weeks treatment ]
    Percentage improvement in FA since beginning treatment, as assessed by the participant or parent/caregiver

  5. Objective (clinician) improvement rating [ Time Frame: After 26 weeks treatment ]
    Percentage improvement in FA since beginning treatment, as assessed by the clinician

  6. Categorical Change in FA [ Time Frame: After 26 weeks treatment ]
    Change in FA since beginning treatment on a 5-point scale, as assessed by the participant or parent/caregiver


Eligibility Criteria
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Ages Eligible for Study:   6 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03826628


Locations
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Sponsors and Collaborators
Dermatology Specialties Limited Partnership
Investigators
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Study Director: Ioana Stanescu Dermatology Specialties Limited Partnership
More Information
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Responsible Party: Dermatology Specialties Limited Partnership
ClinicalTrials.gov Identifier: NCT03826628    
Other Study ID Numbers: DSLP-01
First Posted: February 1, 2019    Key Record Dates
Last Update Posted: July 28, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Individual Participant Data is not planned to be shared with other researchers

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Tuberous Sclerosis
Angiofibroma
Sclerosis
Facies
Pathologic Processes
Disease Attributes
Hamartoma
Neoplasms
Neoplasms, Multiple Primary
Neoplastic Syndromes, Hereditary
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Neurocutaneous Syndromes
 Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs