Weekly Sirolimus Therapy

Sponsor
Medical University of South Carolina (Other)
Overall Status
Recruiting
CT.gov ID
NCT04861064
Collaborator
(none)
24
1
1
12.5
1.9

Study Details

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.

Condition or Disease Intervention/Treatment Phase
Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
24 participants
Allocation:
Non-Randomized
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
Actual Study Start Date :
Jan 18, 2022
Anticipated Primary Completion Date :
Feb 1, 2023
Anticipated Study Completion Date :
Feb 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: Treatment Group

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

Outcome Measures

Primary Outcome Measures

  1. Change in size of lesion [Baseline and 6 months]

    Will be measuring the size of the lesions (mm) at each patient visit

  2. 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

  1. Number of side effects experienced [Month One]

    Patient will complete side effect questionnaires at each visit

  2. Number of side effects experienced [Month Two]

    Patient will complete side effect questionnaires at each visit

  3. Number of side effects experienced [Month Three]

    Patient will complete side effect questionnaires at each visit

  4. Number of side effects experienced [Month Four]

    Patient will complete side effect questionnaires at each visit

  5. Number of side effects experienced [Month Five]

    Patient will complete side effect questionnaires at each visit

  6. Number of side effects experienced [Month Six]

    Patient will complete side effect questionnaires at each visit

  7. Change in quality of life as assessed by questionnaire [Baseline and 6 months]

    Patient will complete quality of life questionnaire at each visit

  8. Number of participants with laboratory abnormalities [From baseline visit to 2 month visit]

    Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored

Eligibility Criteria

Criteria

Ages Eligible for Study:
2 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Medical University of South Carolina Charleston South Carolina United States 29403

Sponsors and Collaborators

  • Medical University of South Carolina

Investigators

  • Principal Investigator: Alexandra Ritter, BS, Medical University of South Carolina

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Chelsea Shope, Principal Investigator, Medical University of South Carolina
ClinicalTrials.gov Identifier:
NCT04861064
Other Study ID Numbers:
  • 00106369
First Posted:
Apr 27, 2021
Last Update Posted:
Feb 24, 2022
Last Verified:
Feb 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
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
Additional relevant MeSH terms:

Study Results

No Results Posted as of Feb 24, 2022