MedTrace Logo

Treatment of Congenital Vascular Malformations Using Sirolimus: Improving Quality of Life

NCT03987152 · Status: UNKNOWN · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 75

Last updated 2021-01-29

No results posted yet for this study

Summary

Congenital vascular anomalies are uncommon and belong to the group of rare diseases.These vascular malformations can cause serious complications including obstruction of vital organs and their function, recurrent infection and significantly reduced quality of life of persons affected.Treatment options range from conservative to surgical extirpation or intralesional embolisation/sclerosis. Unfortunately, this is often not enough. Many patients still have complaints like severe pain and invalidation due to the lymphatic or venous malformation making a normal functional life impossible. Recent case reports mention the positive effects of refractory patients with Sirolimus. Sirolimus, also known as rapamycin, is currently the only FDA-approved mammalian target of rapamycin (mTOR) inhibitor.

Conditions

  • Vascular Malformations

Interventions

DRUG

Sirolimus

Daily intake of Sirolimus during Challenge and Rechallenge phase

Sponsors & Collaborators

  • Radboud University Medical Center

    lead OTHER

Principal Investigators

  • Maroeska Loo, te, Dr. · Radboud University Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
1 Year
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-09-18
Primary Completion
2021-09-18
Completion
2023-03-01

Countries

  • Netherlands

Study Locations

More Related Trials

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 NCT03987152 on ClinicalTrials.gov