NCT03987152: Sirolimus

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

Status unknown Phase 3 Last updated 29 January 2021

What this trial tests

Phase 3 trial testing Sirolimus in Vascular Malformations in 75 participants. Status unknown.

Timeline

18 September 2017

Primary endpoint
18 September 2021

1 March 2023

Quick facts

Lead sponsor	Radboud University Medical Center
Phase	Phase 3
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	75
Start date	18 September 2017
Primary completion	18 September 2021
Estimated completion	1 March 2023
Sites	1 location across Netherlands

Drugs / interventions tested

• Sirolimus (sirolimus) — full drug profile →

Conditions studied

• Vascular Malformations — all drugs for Vascular Malformations →

Sponsor

Radboud University Medical Center

Who can join

1 and older, any sex, with Vascular Malformations. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Recent advances and limitations of mTOR inhibitors in the treatment of cancer.
   Ali ES, Mitra K, Akter S, Ramproshad S, et al · · 2022 · cited 115× · PMID 36109789 · DOI 10.1186/s12935-022-02706-8
2. Cell-free DNA as a diagnostic analyte for molecular diagnosis of vascular malformations.
   Zenner K, Jensen DM, Cook TT, Dmyterko V, et al · · 2021 · cited 34× · PMID 32884133 · DOI 10.1038/s41436-020-00943-8
3. Patients with Congenital Low-Flow Vascular Malformation Treated with Low Dose Sirolimus.
   Harbers VEM, Rongen GAPJM, van der Vleuten CJM, Verhoeven BH, et al · · 2021 · cited 27× · PMID 34003452 · DOI 10.1007/s12325-021-01758-y
4. Clinical differences in sirolimus treatment with low target levels between children and adults with vascular malformations - A nationwide trial.
   Harbers VEM, Zwerink LGJM, Rongen GA, Klein WM, et al · · 2023 · cited 18× · PMID 36824030 · DOI 10.1111/cts.13488
5. Magnitude and relevance of change in health-related quality of life in patients with vascular malformations treated with sirolimus.
   Harbers VEM, Bouwman FCM, van Rijnsoever IMP, Verhoeven BH, et al · · 2023 · cited 3× · PMID 37153086 · DOI 10.3389/fmed.2023.1155476
6. Venous Malformations: Unraveling Latest Mechanisms and Bridging Gaps in Targeted Therapy Development.
   Jiang X, Hu L, Lai J, Ge S, et al · · 2025 · PMID 41281754 · DOI 10.7150/ijbs.122569
7. Pilot study in patients with congenital low-flow vascular malformation treated with low dose sirolimus
   Harbers V, Rongen G, Vleuten vdC, Verhoeven B, et al · · 2020 · DOI 10.21203/rs.3.rs-132412/v1