Weekly Sirolimus Therapy

Phase 2

Recruiting

• Conditions: Venous Malformation; Lymphatic Malformation
• Interventions: Drug: Sirolimus

• Registration Number: NCT04861064
• Lead Sponsor: Medical University of South Carolina

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-04-23
• Study First Submitted QC: 2021-04-23
• Study First Posted: 2021-04-27
• Study Start: 2022-01-18
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-12
• Last Update Posted: 2024-03-15
• Primary Completion: 2025-02
• Study Completion: 2025-06

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

• Patient 2 years of age and older
• Venous, lymphatic, or venolymphatic malformations

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment Group	Sirolimus	-

Primary Outcome Measures

Name	Time	Method
Change in size of lesion	Baseline and 6 months	Will be measuring the size of the lesions (mm) at each patient visit
Change in size of lesion through photograph	Baseline and 6 months	Will be evaluating clinical photographs of lesions at each patient visit

Secondary Outcome Measures

Name	Time	Method
Number of participants with laboratory abnormalities	From baseline visit to 2 month visit	Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored
Change in quality of life as assessed by questionnaire	Baseline and 6 months	Patient will complete quality of life questionnaire at each visit
Number of side effects experienced	Month Six	Patient will complete side effect questionnaires at each visit

Trial Locations

Locations (1)

Medical University of South Carolina; 🇺🇸 Charleston, South Carolina, United States