Weekly treatment with sirolimus for venous and lymphatic malformations
Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
This study is testing if giving sirolimus once a week can help people with venous and lymphatic malformations feel better while causing fewer side effects than taking it every day.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 2 Years and up |
| Sex | All |
| Sponsor | Medical University of South Carolina Academic / other |
| Locations | 1 site (Charleston, South Carolina) |
| Trial ID | NCT04861064 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the effectiveness of administering sirolimus once a week for patients with venous and lymphatic malformations. The study aims to assess not only the therapeutic benefits of this dosing schedule but also patient satisfaction and any adverse effects experienced during the treatment. Participants will receive the medication for a duration of six months, with the possibility of continuing treatment afterward. The trial seeks to provide a safer alternative to daily dosing, which is associated with significant side effects.
Who should consider this trial
Good fit: Ideal candidates for this study are patients aged 2 years and older diagnosed with venous, lymphatic, or venolymphatic malformations.
Not a fit: Patients with contraindications to sirolimus, a history of transplant, or those who are pregnant may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could offer a more manageable and effective option for patients suffering from venous and lymphatic malformations.
How similar studies have performed: While the use of sirolimus has shown promise in other studies, this specific approach of weekly dosing is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Charleston, South Carolina
- Medical University of South Carolina — Charleston, South Carolina, United States (Recruiting)
Study contacts
- Principal investigator: Chelsea Shope, MSCR — Medical University of South Carolina
- Study coordinator: Stephnie Munie, BS
- Email: munie@musc.edu
- Phone: 843-566-2453
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.