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

Study Purpose

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).

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 6 Years - 65 Years
Gender All
More Inclusion & Exclusion 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

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.

NCT03826628
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/Phase 3
Lead Sponsor

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

Dermatology Specialties Limited Partnership
Principal Investigator

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

Ioana Stanescu
Principal Investigator Affiliation Dermatology Specialties Limited Partnership
Agency Class

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

Industry
Overall Status Recruiting
Countries Australia, Hungary, New Zealand, Slovakia, Spain, United States
Conditions

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

Facial Angiofibroma, Tuberous Sclerosis
Additional Details

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).

Arms & Interventions

Arms

Experimental: 0.5% Rapamycin cream, topical

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

Experimental: 1.0% Rapamycin cream, topical

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

Placebo Comparator: Placebo

Placebo cream topical, applied once daily before bed on affected area for 26 weeks

Interventions

Drug: - rapamycin

Apply to the affected area once a day, approximately half an hour before retiring for bed in the evening, 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

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.

Phoenix Children's Hospital, Phoenix, Arizona

Status

Recruiting

Address

Phoenix Children's Hospital

Phoenix, Arizona, 85016

Site Contact

Kellie Badger

kbadger@phoenixchildrens.com

+ 64 9 488 0232

University of California San Diego, La Jolla, California

Status

Recruiting

Address

University of California San Diego

La Jolla, California, 92037

Site Contact

Jessica Reit

jreit@ucsd.edu

+ 64 9 488 0232

All Children's Research Institute, Saint Petersburg, Florida

Status

Recruiting

Address

All Children's Research Institute

Saint Petersburg, Florida, 33701

Site Contact

Allison Weiderhold

aweider1@jhmi.edu

+ 64 9 488 0232

Spectrum Health, Grand Rapids, Michigan

Status

Recruiting

Address

Spectrum Health

Grand Rapids, Michigan, 49503

Site Contact

Christopher L Russell

christopher.russell@spectrumhealth.org

+ 64 9 488 0232

Mayo Clinic, Rochester, Minnesota

Status

Recruiting

Address

Mayo Clinic

Rochester, Minnesota, 55905

Site Contact

Katrina Pierce, CCRP

pierce.katrina@mayo.edu

507-266-1078

University of Virginia, Charlottesville, Virginia

Status

Recruiting

Address

University of Virginia

Charlottesville, Virginia, 22903

Site Contact

Stacy Thompson

src2h@hscmail.mcc.virginia.edu

+ 64 9 488 0232

International Sites

Children's Health Queensland, Brisbane, Queensland, Australia

Status

Recruiting

Address

Children's Health Queensland

Brisbane, Queensland, 4101

Site Contact

Melissa Neylan

melissa.neylan@health.qld.gov.au

07 3069 7267

University of Pécs, Pécs, Hungary

Status

Recruiting

Address

University of Pécs

Pécs, , H-7624

Site Contact

Valentina ú Pavel

VPavel@Hungarotrial.com

0314052573

Canterbury District Health Board, Christchurch, Canterbury, New Zealand

Status

Recruiting

Address

Canterbury District Health Board

Christchurch, Canterbury, 8011

Site Contact

Malina Storer

malina.storer@cdhb.health.nz

+64 3 364 1157

Narodný ústav detských chorȏb, Bratislava, Slovakia

Status

Recruiting

Address

Narodný ústav detských chorȏb

Bratislava, , 83340

Site Contact

Valentina Pavel

VPavel@Hungarotrial.com

031 405 2573

Clinica Universidad de Navarra, Pamplona, Navarra, Spain

Status

Recruiting

Address

Clinica Universidad de Navarra

Pamplona, Navarra, 31008

Site Contact

Irene Barrieutos

ibarrientos@unav.es

948255400 #2742

Clínica Universidad de Navarra, Madrid, Spain

Status

Recruiting

Address

Clínica Universidad de Navarra

Madrid, , 28007

Site Contact

Mar Garcia

mgarciai@unav.es

913531920 #7574

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