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Weekly Sirolimus Therapy

Primary Purpose

Venous Malformation, Lymphatic Malformation

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Sirolimus
Sponsored by
Medical University of South Carolina
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Venous Malformation

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Medical University of South CarolinaRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment Group

Arm Description

Outcomes

Primary Outcome Measures

Change in size of lesion
Will be measuring the size of the lesions (mm) at each patient visit
Change in size of lesion through photograph
Will be evaluating clinical photographs of lesions at each patient visit

Secondary Outcome Measures

Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Change in quality of life as assessed by questionnaire
Patient will complete quality of life questionnaire at each visit
Number of participants with laboratory abnormalities
Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored

Full Information

First Posted
April 23, 2021
Last Updated
March 28, 2023
Sponsor
Medical University of South Carolina
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1. Study Identification

Unique Protocol Identification Number
NCT04861064
Brief Title
Weekly Sirolimus Therapy
Official Title
Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 18, 2022 (Actual)
Primary Completion Date
February 2024 (Anticipated)
Study Completion Date
February 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Medical University of South Carolina

4. Oversight

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

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Venous Malformation, Lymphatic Malformation

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
24 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Treatment Group
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Sirolimus
Intervention Description
Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months.
Primary Outcome Measure Information:
Title
Change in size of lesion
Description
Will be measuring the size of the lesions (mm) at each patient visit
Time Frame
Baseline and 6 months
Title
Change in size of lesion through photograph
Description
Will be evaluating clinical photographs of lesions at each patient visit
Time Frame
Baseline and 6 months
Secondary Outcome Measure Information:
Title
Number of side effects experienced
Description
Patient will complete side effect questionnaires at each visit
Time Frame
Month One
Title
Number of side effects experienced
Description
Patient will complete side effect questionnaires at each visit
Time Frame
Month Two
Title
Number of side effects experienced
Description
Patient will complete side effect questionnaires at each visit
Time Frame
Month Three
Title
Number of side effects experienced
Description
Patient will complete side effect questionnaires at each visit
Time Frame
Month Four
Title
Number of side effects experienced
Description
Patient will complete side effect questionnaires at each visit
Time Frame
Month Five
Title
Number of side effects experienced
Description
Patient will complete side effect questionnaires at each visit
Time Frame
Month Six
Title
Change in quality of life as assessed by questionnaire
Description
Patient will complete quality of life questionnaire at each visit
Time Frame
Baseline and 6 months
Title
Number of participants with laboratory abnormalities
Description
Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored
Time Frame
From baseline visit to 2 month visit

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Courtney Linkous, BA
Phone
843-566-2453
Email
linkousc@musc.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Chelsea Shope, MSCR
Organizational Affiliation
Medical University of South Carolina
Official's Role
Principal Investigator
Facility Information:
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29403
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Courtney Linkous, BA
Phone
843-792-9784
Email
linkousc@musc.edu

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Weekly Sirolimus Therapy

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