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Topical Rapamycin/Sirolimus for Complicated Vascular Anomalies and Other Susceptible Lesions

NCT04172922 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2025-12-31

No results posted yet for this study

Summary

Proposed Study: Treatment protocol for the use of the topical Rapamycin/Sirolimus for Complicated Vascular Anomalies and other susceptible lesions

1. Aim The aim of this treatment study is to evaluate the benefit and tolerability of topical sirolimus applied to cutaneous vascular anomalies in pediatric patients. The primary end point will be individually determined based on improvement in lesional clinical characteristics over baseline
2. Rationale for topical sirolimus use in VA The rationale for the use of topical sirolimus is to minimize these potential side effects and risks. Data for the use of topical sirolimus for vascular anomalies at this time are anecdotal and case reports only. As such, this prospective protocol seeks to determine the effectiveness and tolerability of topical sirolimus on patients with vascular anomalies that have a cutaneous component.
3. Experimental design This is an open-labeled efficacy trial with the aim to determine if topical sirolimus can be safe and efficacious in treating the cutaneous component of complicated vascular anomalies. Patients who meet eligibility criteria with a diagnosis of vascular anomaly (VA) with cutaneous component will be offered treatment with the investigational topical sirolimus. Patients will receive topical sirolimus therapy for a total of six months and will be monitored regularly at the research site for clinical response. Response will be based on pre-determined clinical criteria. Patients will be removed from study if there is no response at three months after initiation of therapy.

Clinical response will be defined as improvement in measurable parameters defined at the time of initiation of therapy. These include
1. Size of lesions, measured in two parallel longest diameters
2. Flattening of lesion
3. Number of vesicles
4. Episodes of superinfection or bleeding
5. Improvement in pain
4. Drug Information The topical sirolimus formulation will be made at a concentration of 1% sirolimus ointment. Bulk sirolimus powder will be compounded in a liposomal base in a GMP level pharmaceutical company. This base will enhance drug penetration into the skin. It ensures adequate adhesion to the application area and a low degree of systemic absorption. Due to limited absorption only mild side effects are expected.

Conditions

  • Vascular Anomaly

Interventions

DRUG

Topical Sirolimus

Open label, topical sirolimus (1%) cream will be applied to cutaneous component of complicated vascular anomalies twice daily for 4 weeks and once daily thereafter- for the duration of study.

Sponsors & Collaborators

  • Nemours Children's Clinic

    lead OTHER

Principal Investigators

  • Craig Johnson, DO · Nemours

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
36 Months
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-04-01
Primary Completion
2024-02-15
Completion
2024-02-15
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04172922 on ClinicalTrials.gov