24 Participants Needed

Weekly Sirolimus for Lymphatic and Venous Malformations

AR
CL
SM
Overseen ByStephnie Munie, BS
Age: Any Age
Sex: Any
Trial Phase: Phase 2
Sponsor: Medical University of South Carolina
Must be taking: Sirolimus
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 4 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing if taking sirolimus once a week can help people with venous and lymphatic malformations. These patients have few treatment options. Sirolimus aims to reduce abnormal growths by affecting the immune system. The study will also check for side effects and patient satisfaction over several months. Sirolimus has been used in various studies to treat lymphatic malformations and has shown potential in stabilizing lung function in patients with lymphangioleiomyomatosis.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are taking certain medications that affect how sirolimus works. It's best to discuss your current medications with the trial team.

Is sirolimus safe for treating lymphatic and venous malformations?

Sirolimus has shown a generally favorable safety profile in treating vascular anomalies, though some severe adverse events have been reported during its off-label use. In a study using topical sirolimus gel, no adverse events were observed, suggesting it may be safe for certain types of vascular malformations.12345

How is the drug Sirolimus unique in treating lymphatic and venous malformations?

Sirolimus is unique because it is an oral medication that targets the mTOR pathway, which helps control cell growth and proliferation, making it effective for treating vascular malformations that are difficult to manage with traditional methods like surgery or sclerotherapy. It offers a non-invasive option for patients with extensive or recurrent malformations, where other treatments have limited success.46789

What data supports the effectiveness of the drug Sirolimus for treating lymphatic and venous malformations?

Research shows that Sirolimus has been effective in treating lymphatic malformations, especially in children, by reducing the size and symptoms of these growths. It has been used successfully in cases where other treatments like surgery or sclerotherapy (a procedure to shrink blood vessels) were not effective.3491011

Who Is on the Research Team?

CS

Chelsea Shope, MSCR

Principal Investigator

Medical University of South Carolina

Are You a Good Fit for This Trial?

This trial is for individuals aged 2 years and older with venous, lymphatic, or venolymphatic malformations. It excludes those with contraindications to sirolimus, on certain other medications, pregnant women or those who might become pregnant without effective contraception, and children with a history of transplant or immunodeficiency.

Inclusion Criteria

You have malformations in your veins, lymphatic system, or both.
Patients aged 2 years and older

Exclusion Criteria

Children who have had previous immune system problems.
Children who have had a severe or life-threatening infection in the past.
If you are a child taking medications that inhibit CYP3A4.
See 6 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive once weekly dosed sirolimus for the treatment of venous and lymphatic malformations

6 months
Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 months
2 visits (in-person)

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

What Are the Treatments Tested in This Trial?

Interventions

  • Sirolimus
Trial Overview The study tests if taking the oral medication Sirolimus once a week can treat venous and lymphatic malformations effectively. The treatment lasts for six months with an option to continue thereafter. Patient satisfaction and side effects will also be evaluated.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Treatment GroupExperimental Treatment1 Intervention

Sirolimus is already approved in United States, European Union, Canada, Japan for the following indications:

🇺🇸
Approved in United States as Rapamune for:
  • Prevention of organ rejection in kidney transplant patients
  • Treatment of lymphangioleiomyomatosis (LAM)
🇪🇺
Approved in European Union as Rapamune for:
  • Prevention of organ rejection in kidney transplant patients
  • Treatment of lymphangioleiomyomatosis (LAM)
🇨🇦
Approved in Canada as Rapamune for:
  • Prevention of organ rejection in kidney transplant patients
  • Treatment of lymphangioleiomyomatosis (LAM)
🇯🇵
Approved in Japan as Rapamune for:
  • Prevention of organ rejection in kidney transplant patients

Find a Clinic Near You

Who Is Running the Clinical Trial?

Medical University of South Carolina

Lead Sponsor

Trials
994
Recruited
7,408,000+

Published Research Related to This Trial

Sirolimus shows promise in treating pediatric lymphatic malformations, with 19 studies reviewed involving 97 patients, but the overall efficacy and safety profile is still uncertain due to a lack of high-quality research.
Mild-to-moderate adverse events were reported in 94 cases, highlighting the need for systematic reporting of side effects to help clinicians manage treatment risks, especially in younger children.
Sirolimus treatment for paediatric head and neck lymphatic malformations: a systematic review.Saibene, AM., Rosso, C., Felisati, G., et al.[2023]
A 10-year-old boy with an orbital venolymphatic malformation was initially treated with local sclerotherapy, but the condition relapsed, highlighting the need for effective long-term management strategies.
The relapse was successfully managed with oral sirolimus, suggesting that this medication may be a promising treatment option for lymphatic malformations, although further studies are needed to establish optimal dosing and broader applications.
Treatment of orbital lymphatic malformation with oral sirolimus: a case report.Alba-Linero, C., García-Lorente, M., Rachwani-Anil, R., et al.[2023]
Sirolimus is effective in reducing the volume of pediatric cervicofacial lymphatic malformations (LMs), with 78% of patients showing a reduction of 20% or more on MRI, and 32% achieving a reduction of 50% or more.
The treatment also leads to significant clinical improvements, with 97% of patients reporting some benefit, and particularly helps airway LMs, as 33% of tracheostomy-dependent patients were able to be decannulated after starting sirolimus.
Sirolimus for Pediatric Cervicofacial Lymphatic Malformation: A Systematic Review and Meta-Analysis.Kamhieh, Y., Mitra, R., Burnett, T., et al.[2023]

Citations

Sirolimus treatment for paediatric head and neck lymphatic malformations: a systematic review. [2023]
Treatment of orbital lymphatic malformation with oral sirolimus: a case report. [2023]
Sirolimus for Pediatric Cervicofacial Lymphatic Malformation: A Systematic Review and Meta-Analysis. [2023]
Effectiveness of sirolimus in the treatment of complex lymphatic malformations: Single center report of 56 cases. [2021]
Sirolimus: A Successful Medical Treatment for Head and Neck Lymphatic Malformations. [2020]
A narrative review of the role of sirolimus in the treatment of congenital vascular malformations. [2022]
Severe adverse events during sirolimus "off-label" therapy for vascular anomalies. [2022]
Verification of the efficacy of topical sirolimus gel for systemic rare vascular malformations: a pilot study. [2023]
Intraorbital lymphatic-venous malformation in an adult patient: is sirolimus the key? [2023]
Single-center experience with sirolimus therapy for vascular malformations. [2022]
11.United Statespubmed.ncbi.nlm.nih.gov
Treatment of Lymphatic Malformations with the mTOR Inhibitor Sirolimus: A Systematic Review. [2021]
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