| NCT ID | NCT04861064 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Medical University of South Carolina |
| Condition | Venous Malformation |
| Study Type | INTERVENTIONAL |
| Enrollment | 24 participants |
| Start Date | 2022-01-18 |
| Primary Completion | 2027-12 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 24 participants in total. It began in 2022-01-18 with a primary completion date of 2027-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, oral ulcerations, and lab abnormalities. The purpose of this study is to determine if once weekly dosed sirolimus will be effective for the treatment of venous and lymphatic malformations. Additionally, the study will evaluate patient satisfaction and identify adverse effects. Participants will be on the medication for 6 months with an option to continue after this time period.
Eligibility Criteria
Inclusion Criteria: * Patient 2 years of age and older * Venous, lymphatic, or venolymphatic malformations Exclusion Criteria: * Children with contraindication to use of sirolimus * Children with history of transplant * Children with a history of natural immunodeficiency * Children with a history of artificially induced immunodeficiency * Children with a history of a serious or life-threatening infection * Children taking CYP3A4 inhibiting medications * Children taking strong CYP3A4 inducers to avoid subtherapeutic dosing/exposure. * Inability or unwillingness of subject or legal guardian/representative to give informed consent * Women that are or may become pregnant o Sirolimus is a Pregnancy Category C drug. No randomized controlled studies have been done on pregnant women. Women of childbearing potential must be on effective contraception prior to, during, and for 12 weeks following sirolimus therapy.
Contact & Investigator
Chelsea Shope, MSCR
PRINCIPAL INVESTIGATOR
Medical University of South Carolina
Frequently Asked Questions
Who can join the NCT04861064 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, studying Venous Malformation. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04861064 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT04861064 currently recruiting?
Yes, NCT04861064 is actively recruiting participants. Contact the research team at munie@musc.edu for enrollment information.
Where is the NCT04861064 trial being conducted?
This trial is being conducted at Charleston, United States.
Who is sponsoring the NCT04861064 clinical trial?
NCT04861064 is sponsored by Medical University of South Carolina. The principal investigator is Chelsea Shope, MSCR at Medical University of South Carolina. The trial plans to enroll 24 participants.