Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex

• Register: ClinicalTrials.gov
• Last refreshed on: 10 April 2023
• Main ID: NCT03363763
• Date of registration: 01/12/2017
• Prospective Registration: No
• Primary sponsor: Aucta Pharmaceuticals, Inc
• Public title: Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex
• Scientific title: Phase 2 Multi Center Prospective Rand. Double Blind Placebo Cont. Parallel Design Study to Evaluate Safety & Efficacy of Topical Sirolimus for Cutaneous Angiofibromas in Subjects W/ Tuberous Sclerosis Complex Followed by Opt. Open Label
• Date of first enrolment: April 12, 2017
• Target sample size: 24
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03363763
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

China, United States

Contacts

• Name: Shoufeng Li, Ph.D
• Affiliation: Aucta Pharmaceuticals, Inc

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Generally healthy males or non-pregnant females aged 2 to 21 years, inclusive, at the
time of screening.

2. Diagnosis of TSC with visible facial angiofibromas of at least grade 3 up to grade 5,
inclusive, based on the IGA.

3. Subjects with 3 or more isolated, measurable lesions of facial angiofibroma, with
color grading score =2 for each of the 3 lesions.

4. Females of childbearing potential must have a negative urine pregnancy test (or a
negative serum pregnancy test if a urine pregnancy test cannot be obtained) (For
China, different pregnancy test would be followed) and if sexually active or become
sexually active during the study, must agree to use an effective form of birth control
for the duration of the study. Females using oral contraceptives must also use a
barrier method of contraception during the study. Sexually active male subjects and/or
their female partners should also use appropriate contraception.

Effective contraception is defined as follows:

- Oral/implant/injectable/transdermal/estrogenic vaginal ring contraceptives,
intrauterine device, condom with spermicide, diaphragm with spermicide.

- Abstinence or partner's vasectomy are acceptable if the female agrees to
implement one of the other acceptable methods of birth control if her partner
changes.

5. The subject and/or their parent or guardian must be willing and able to provide
written informed consent/assent.

6. Willing and able to comply with all trial requirements.

7. Subject or parent/guardian must be able to complete the subject self-assessment survey
and subject diary in English or another language into which the documents have been
officially translated.

8. Subjects should be in good general health based on the subject's medical history,
physical exam, and impression of the study doctor.

Exclusion Criteria:

9. Has any chronic or acute medical condition, that in the opinion of the investigator,
may pose a risk to the safety of the subject during the trial period, or may interfere
with the assessment of safety or efficacy in this trial.

10. Has received oral therapy or topical therapy of an mTOR inhibitor (sirolimus,
temsirolimus, or everolimus) within 1 month of Baseline or other dermatological
treatment to facial angiofibromas within 1 month of baseline. (Sunscreen is expected
to be used in this patient population and is not considered treatment.)

11. Is currently receiving any form of immunosuppression therapy or has previously
experienced significant immune dysfunction.

12. Has a history of sensitivity to any component of the investigational product.

13. Is pregnant, plans to become pregnant during the course of the study, or is
breastfeeding.

14. Has other dermatologic conditions, pigmentation, scarring, pigmented lesions or
sunburn in the treatment area that would preclude or prevent adequate assessment of
changes to their facial angiofibromas.

15. Has facial hair (e.g., beard, sideburns, mustache) that could interfere with study
assessments.

16. Has had laser surgery or cryotherapy to facial angiofibromas within 6 months preceding
study entry.

17. Requires the use of any concomitant medication that, in the investigator's opinion,
has the potential to cause an adverse effect when given with the investigational
product or will interfere with the interpretation of the study results (see Section
16.1 Appendix 1 for Potential Drug Interactions).

18. Has participated in another clinical trial or received an investigational product
within 3 months prior to screening.



Age minimum: 2 Years
Age maximum: 21 Years
Gender: All

Health Condition(s) or Problem(s) studied

Angiofibroma of Face

Tuberous Sclerosis

Intervention(s)

Drug: Placebo ointment

Drug: Sirolimus 0.2%

Drug: Sirolimus 0.4%

Primary Outcome(s)

The proportion of subjects with a clinical response of treatment success. [Time Frame: Week 12]

Secondary Outcome(s)

The proportion of subjects with at least a 2-grade improvement on the Week 12 Investigator Global Assessment (IGA) of the facial skin lesions assessed by the IRC [Time Frame: Week 12]

The proportion of subjects with at least 2-grade improvement as compared to Baseline in lesion elevation score [Time Frame: Week 12]

The proportion of subjects with at least 30% improvement at Week 12 as compared to Baseline in the Facial Angiofibromas Severity Index (FASI) score. [Time Frame: Week 12]

The proportion of subjects with at least Moderate Improvement on Modified Nobel Scoring System assessed by the investigator at Week 12 [Time Frame: Week 12]

The proportion of subjects with at least Moderate Improvement on Modified Nobel Scoring System assessed by the IRC at Week 12 [Time Frame: Week 12]

The proportion of subjects with at least 2-grade improvement as compared to Baseline in the subject self-assessment survey [Time Frame: Week 12]

Overall response of angiofibroma assessed by the investigator at Week 12 as compared to baseline based on Modified Nobel Scoring System [Time Frame: Week 12]

The proportion of subjects with an investigator assessed IGA score of clear or almost clear with at least a 2-grade improvement on the Week 12 IGA of the facial skin lesions [Time Frame: Week 12]

Overall response of angiofibroma assessed by the IRC at Week 12 compared to baseline based on Modified Nobel Scoring System [Time Frame: Week 12]

The proportion of subjects with at least 2-grade improvement as compared to Baseline in categorical lesion counts [Time Frame: Week 12]

The time to reach at least 30% improvement from Baseline in the Facial Angiofibromas Severity Index (FASI) score [Time Frame: Week 12]

Secondary ID(s)

AUCTA-UAP006-PH2

Source(s) of Monetary Support

Please refer to primary and secondary sponsors