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

Study Purpose

The objective of this study is to evaluate the safety and efficacy of sirolimus (0.2% and 0.4% formulations) and its vehicle when applied topically once daily for 12 weeks for the treatment of cutaneous angiofibromas in pediatric subjects with tuberous sclerosis complex (TSC).

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 2 Years - 21 Years
Gender All
More 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.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03363763
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Aucta Pharmaceuticals, Inc
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Shoufeng Li, Ph.D
Principal Investigator Affiliation Aucta Pharmaceuticals, Inc
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries China, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Angiofibroma of Face, Tuberous Sclerosis
Additional Details

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study designed to assess the safety and efficacy of topically-applied sirolimus for the treatment of cutaneous angiofibromas in pediatric subjects with TSC. Approximately 45 subjects will be enrolled at investigational sites in the United States (US) and China, though other countries may be added in the future. Approximately 45 subjects who meet the study entry criteria will randomly be assigned in a 1:1:1 ratio to receive 1 of 3 treatments: sirolimus 0.2% ointment, sirolimus 0.4% ointment, or placebo ointment. The randomization is stratified by site. Subjects, or a parent/guardian, will apply the study medication topically to the cutaneous angiofibromas on the face once daily at night before going to bed for 12 weeks. Subjects who complete the double-blind phase of the study, with an overall compliance rate >80% as determined by the dosing diary, will be offered entry into an open-label period for an additional 12 weeks. The maximum study duration for each subject will be approximately 30 weeks and includes a screening period of up to 4 weeks, a blinded treatment period of 12 weeks, optional open-label period of 12 weeks, and a follow-up period of 2 weeks. An interim analysis will be performed when all subjects have completed the double-blind phase (Visit 5

  • - Week 12).
The data will be unblinded to assess for efficacy and results reported.

Arms & Interventions

Arms

Active Comparator: Arm 1

Sirolimus 0.2% ointment applied topically hs x 12 weeks

Active Comparator: Arm 2

Sirolimus 0.4% ointment applied topically hs x 12 weeks

Placebo Comparator: Arm 3

Placebo ointment applied topically hs x 12 weeks

Interventions

Drug: - Sirolimus 0.2%

Ointment for topical administration hs x 12 weeks

Drug: - Sirolimus 0.4%

Ointment for topical administration hs x 12 weeks

Drug: - Placebo ointment

Placebo ointment comparator for topical administration hs x 12 weeks

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Translational Genomics Research, Phoenix, Arizona

Status

Completed

Address

Translational Genomics Research

Phoenix, Arizona, 85004

Los Angeles, California

Status

Recruiting

Address

Children's Hospital of Los Angeles, Division of Neurology

Los Angeles, California, 90027

Site Contact

Martha Arellano-Garcia

margarcia@chla.usc.edu

323-361-5812

Aurora, Colorado

Status

Completed

Address

Children's Clinical Research Organization, Children's Hospital Colorado

Aurora, Colorado, 80045

Children's Healthcare of Atlanta, Atlanta, Georgia

Status

Completed

Address

Children's Healthcare of Atlanta

Atlanta, Georgia, 30329

Boston Children's Hospital, Boston, Massachusetts

Status

Recruiting

Address

Boston Children's Hospital

Boston, Massachusetts, 02115

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania

Status

Recruiting

Address

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

Site Contact

Donnette Paris

PARIS@email.chop.edu

267-426-7167

LeBonheur Children's Hospital, Memphis, Tennessee

Status

Completed

Address

LeBonheur Children's Hospital

Memphis, Tennessee, 38103

International Sites

Children's Hospital of Fudan University, Shanghai, China

Status

Recruiting

Address

Children's Hospital of Fudan University

Shanghai, , 201102

Site Contact

Ji Wang

xiaojizi12@sina.com

732-652-9225

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