Weekly Sirolimus Therapy

Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations

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.

Study Overview

• Status: Recruiting
• Conditions: Lymphatic Malformation; Venous Malformation
• Intervention / Treatment: Drug: Sirolimus

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Stephnie Munie, BS; Phone Number: 843-566-2453; Email: munie@musc.edu

Study Locations

• United States
  • South Carolina
    • Charleston, South Carolina, United States, 29403
      • Recruiting
      • Medical University of South Carolina
      • Contact: Stephanie Munie, BS; Phone Number: 843-792-9784; Email: munie@musc.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment Group	Drug: Sirolimus; Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Medical University of South Carolina
• Investigators: Principal Investigator: Chelsea Shope, MSCR, Medical University of South Carolina

Study record dates

Study Major Dates

• Study Start (Actual): January 18, 2022
• Primary Completion (Estimated): February 1, 2025
• Study Completion (Estimated): June 1, 2025

Study Registration Dates

• First Submitted: April 23, 2021
• First Submitted That Met QC Criteria: April 23, 2021
• First Posted (Actual): April 27, 2021

Study Record Updates

• Last Update Posted (Actual): March 15, 2024
• Last Update Submitted That Met QC Criteria: March 12, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Lymphatic Diseases; Lymphatic Vessel Tumors; Congenital Abnormalities; Lymphangioma; Lymphatic Abnormalities; Physiological Effects of Drugs; Anti-Infective Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antifungal Agents; Sirolimus
• Other Study ID Numbers: 00106369

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No