Congenital arterial malformation Trials in Disease

Study on Sirolimus and Prednisolone for Treating Superficial Arteriovenous Malformations in Patients

This trial is investigating whether sirolimus, marketed as Rapamune, can effectively reduce the size of superficial arteriovenous malformations. These are abnormal connections between arteries and veins that bypass the normal capillary system, creating a tangle of blood vessels near the skin surface. Over time, these malformations can grow and cause swelling, skin discoloration, warmth, and sometimes pain due to increased blood flow and pressure.

Who can participate:

• Adults, adolescents, and children over 2 years old with superficial arteriovenous malformations
• Patients must be in stage II+, III, or IV according to the Schöbinger classification, which describes how advanced the condition is
• The condition can be either progressing or stable, with or without bleeding problems
• Participants or their parents must sign a consent form showing they understand the study's risks and benefits
• Women of childbearing age must have a negative pregnancy test before joining
• All participants must use effective birth control during the study and for up to 12 weeks after treatment ends

Who cannot participate:

• Patients with other medical conditions besides superficial arteriovenous malformations
• Individuals outside the specified age range
• People considered part of a vulnerable population who might need special protection or care

What the trial involves:

The main focus of this study is to determine whether sirolimus can reduce the size of arteriovenous malformations by at least 30% within the first year. Participants will take sirolimus in tablet form, along with prednisolone metasulfobenzoate sodium as orodispersible tablets. The study team will determine the exact dosage and frequency for each participant based on their individual needs.

The trial involves regular monitoring at three, six, and nine months to track changes in the condition using specific imaging techniques. Throughout the study, ongoing assessments ensure the safety and effectiveness of the treatment, with adjustments made as needed. The study is expected to conclude by September 12, 2026.

Investigational drugs:

Sirolimus works by inhibiting a protein called mTOR, which plays a role in cell growth and proliferation. By targeting this pathway, the medication aims to slow down the growth of abnormal blood vessels and reduce the size of the malformations. While sirolimus is commonly used as an immunosuppressant to prevent organ transplant rejection, its potential in treating vascular malformations is currently under investigation. Prednisolone is used alongside sirolimus as part of the treatment protocol.

Summary

Currently, only one clinical trial is actively recruiting patients with congenital arterial malformation, specifically those with superficial arteriovenous malformations. This trial is taking place in France and focuses on evaluating sirolimus, a medication that has shown promise in targeting abnormal blood vessel growth. The study represents an important step in exploring new treatment options for this condition, particularly for patients in advanced stages who experience symptoms such as swelling, pain, or bleeding. The trial's approach of combining sirolimus with prednisolone and using regular imaging assessments demonstrates a careful, systematic evaluation of this treatment strategy. Patients interested in participating should discuss the specific eligibility requirements with their healthcare provider to determine if this trial might be suitable for their individual situation.