Weekly Sirolimus Therapy

Lymphatic Malformation Clinical Trial

Official title:

Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04861064
• Other study ID #: 00106369
• Status: Recruiting
• Phase: Phase 2
• Start date: January 18, 2022
• Est. completion date: June 2025

Study information

• Verified date: March 2024
• Source: Medical University of South Carolina
• Contact: Stephnie Munie, BS
• Phone: 843-566-2453
• Email: munie[@]musc.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 24
• Est. completion date: June 2025
• Est. primary completion date: February 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Congenital Abnormalities
• Lymphangioma
• Lymphatic Abnormalities
• Lymphatic Malformation
• Venous Malformation

Intervention

Drug:
• Sirolimus
Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months.

Locations

Country	Name	City	State
United States	Medical University of South Carolina	Charleston	South Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Medical University of South Carolina

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in size of lesion	Will be measuring the size of the lesions (mm) at each patient visit	Baseline and 6 months
Primary	Change in size of lesion through photograph	Will be evaluating clinical photographs of lesions at each patient visit	Baseline and 6 months
Secondary	Number of side effects experienced	Patient will complete side effect questionnaires at each visit	Month One
Secondary	Number of side effects experienced	Patient will complete side effect questionnaires at each visit	Month Two
Secondary	Number of side effects experienced	Patient will complete side effect questionnaires at each visit	Month Three
Secondary	Number of side effects experienced	Patient will complete side effect questionnaires at each visit	Month Four
Secondary	Number of side effects experienced	Patient will complete side effect questionnaires at each visit	Month Five
Secondary	Number of side effects experienced	Patient will complete side effect questionnaires at each visit	Month Six
Secondary	Change in quality of life as assessed by questionnaire	Patient will complete quality of life questionnaire at each visit	Baseline and 6 months
Secondary	Number of participants with laboratory abnormalities	Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored	From baseline visit to 2 month visit