Weekly Sirolimus Therapy

NCT04861064 | Recruiting Phase 2 FDA Drug

• 1 other identifier: 00106369
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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.

Conditions

Venous Malformation; Lymphatic Malformation

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (8)

• Number of side effects experienced

  Month One

• Number of side effects experienced

  Month Two

• Number of side effects experienced

  Month Three

• Number of side effects experienced

  Month Four

• Number of side effects experienced

  Month Five

Study Arms (1)

Treatment Group

EXPERIMENTAL

Drug: Sirolimus

Interventions

Sirolimus DRUG

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

Treatment Group

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Medical University of South Carolina: lead

Study Sites (1)

Medical University of South Carolina

Charleston, South Carolina, 29403, United States

RECRUITING

MeSH Terms

Conditions

Lymphatic Abnormalities

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Lymphatic Diseases; Hemic and Lymphatic Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Chelsea Shope, MSCR

  Medical University of South Carolina

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Stephnie Munie, BS

CONTACT

843-566-2453; munie@musc.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: April 23, 2021
• First Posted: April 27, 2021
• Study Start: January 18, 2022
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: March 5, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)