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Angiofibroma clinical trials

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NCT ID: NCT05549167 Recruiting - Clinical trials for Juvenile Nasopharyngeal Angiofibroma

Efficacy and Safety of Sirolimus in Children and Adolescents With Juvenile Nasopharyngeal Angiofibroma (JNA)

Start date: May 30, 2022
Phase: Phase 3
Study type: Interventional

Juvenile nasopharyngeal angiofibroma (JNA) is a pathologically benign yet locally aggressive and destructive tumor that develops in the choana and nasopharynx. Historical treatment of JNA has included embolization, surgical resection, and radiation. mTOR signaling way demonstrated to be involved in regulation of growth and angiogenesis of JNA. Sirolimus, as mTOR inhibitor, is a potential target JNA therapy. The main purpose of the study is to evaluate the efficacy and safety of sirolimus in children and adolescents with primary or recurrent JNA. Efficacy will be estimated based on dynamics of the JNA progression. Historical control group will be used for comparison as standard therapy. Due to limited experience of sirolimus in JNA in routine practice, study should be conducted in 2 phases: pilot and extended. Decision regarding extended phase will be based on the results of pilot phase.

NCT ID: NCT03826628 Completed - Tuberous Sclerosis Clinical Trials

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

Start date: July 28, 2019
Phase: Phase 2/Phase 3
Study type: Interventional

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).

NCT ID: NCT03363763 Terminated - Tuberous Sclerosis Clinical Trials

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

Start date: April 12, 2017
Phase: Phase 2
Study type: Interventional

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).

NCT ID: NCT03140449 Completed - Facial Angiofibroma Clinical Trials

Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis

Start date: September 5, 2013
Phase: Phase 3
Study type: Interventional

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.

NCT ID: NCT02860494 Withdrawn - Clinical trials for Facial Angiofibromas

Topical Everolimus in Patients With Tuberous Sclerosis Complex

EVEROST
Start date: December 2020
Phase: Phase 2/Phase 3
Study type: Interventional

Tuberous sclerosis complex (TSC) is a rare genetic multisystem disorder characterized by the development of hamartomas in several organs (e.g. brain, heart, kidney, liver, lung), and skin in more than 90% of cases. Facial angiofibromas (FA), present in about 80% of patients, are a stigmatizing hallmark of the disease. Everolimus could be a candidate for use as a topical formulation to treat FA. This adaptive seamless Phase II/III study primary objective is to determine the dose of topical everolimus for treatment of FA and evaluate the efficacy and safety of topical everolimus versus placebo in patients with angiofibromas.

NCT ID: NCT02654340 Terminated - Clinical trials for Lymphangioleiomyomatosis

Biomarkers for Tuberous Sclerosis Complex (BioTuScCom)

TuScCom
Start date: August 1, 2018
Phase:
Study type: Observational

International, multicenter, observational, longitudinal study to identify biomarker/s for Tuberous Sclerosis Complex and to explore the clinical robustness, specificity, and long´-term variability of these biomarker/s

NCT ID: NCT02635789 Completed - Plaque Clinical Trials

Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)

Start date: December 2015
Phase: Phase 3
Study type: Interventional

The purpose of this trial is to evaluate the efficacy and safety of NPC-12G gel (topical formulation of sirolimus) versus placebo gel to facial angiofibroma and other skin lesions in patients with tuberous sclerosis complex (TSC)

NCT ID: NCT02634931 Completed - Plaque Clinical Trials

Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

Start date: December 2015
Phase: Phase 3
Study type: Interventional

The purpose of this trial is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel (0.2% sirolimus gel) to angiofibroma and other skin lesions in patients with tuberous sclerosis complex in the open-label trial.

NCT ID: NCT01853423 Completed - Facial Angiofibroma Clinical Trials

Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream

Start date: October 2013
Phase: Phase 1
Study type: Interventional

The objective of this study is to evaluate the safety and efficacy of a 0.1% formulation of rapamune cream in children with Tuberous Sclerosis Complex (TSC), ages 3 years and older, who have facial angiofibromas that would benefit from treatment.

NCT ID: NCT01526356 Completed - Tuberous Sclerosis Clinical Trials

Topical Rapamycin to Erase Angiofibromas in TSC

Treatment
Start date: May 2012
Phase: Phase 2
Study type: Interventional

The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous Sclerosis Complex (TSC). Subjects will apply either the topical vehicle containing rapamycin or the topical vehicle alone nightly to their angiofibromas for six months. The primary goal of this study is to evaluate the efficacy of the topical medication for reduction of cutaneous angiofibromas in patients with TSC. The secondary goal of this study is to confirm the safety of the topical medication.