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Weekly Sirolimus Therapy

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ClinicalTrials.gov Identifier: NCT04861064
Recruitment Status : Recruiting
First Posted : April 27, 2021
Last Update Posted : February 24, 2022
Sponsor:
Information provided by (Responsible Party):
Chelsea Shope, Medical University of South Carolina

Study Description
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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
Venous Malformation Lymphatic Malformation Drug: Sirolimus Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : January 18, 2022
Estimated Primary Completion Date : February 2023
Estimated Study Completion Date : February 2023

Resource links provided by the National Library of Medicine

Drug Information available for: Sirolimus

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


Outcome Measures
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Primary Outcome Measures :
  1. Change in size of lesion [ Time Frame: 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 [ Time Frame: Baseline and 6 months ]
    Will be evaluating clinical photographs of lesions at each patient visit


Secondary Outcome Measures :
  1. Number of side effects experienced [ Time Frame: Month One ]
    Patient will complete side effect questionnaires at each visit

  2. Number of side effects experienced [ Time Frame: Month Two ]
    Patient will complete side effect questionnaires at each visit

  3. Number of side effects experienced [ Time Frame: Month Three ]
    Patient will complete side effect questionnaires at each visit

  4. Number of side effects experienced [ Time Frame: Month Four ]
    Patient will complete side effect questionnaires at each visit

  5. Number of side effects experienced [ Time Frame: Month Five ]
    Patient will complete side effect questionnaires at each visit

  6. Number of side effects experienced [ Time Frame: Month Six ]
    Patient will complete side effect questionnaires at each visit

  7. Change in quality of life as assessed by questionnaire [ Time Frame: Baseline and 6 months ]
    Patient will complete quality of life questionnaire at each visit

  8. Number of participants with laboratory abnormalities [ Time Frame: From baseline visit to 2 month visit ]
    Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored


Eligibility Criteria
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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04861064


Contacts
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Contact: Alexandra Ritter, BS 843-876-3209 ritteral@musc.edu

Locations
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United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29403
Contact: Alexandra M Ritter    843-792-9784    ritteral@musc.edu   
Sponsors and Collaborators
Medical University of South Carolina
Investigators
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Principal Investigator: Alexandra Ritter, BS Medical University of South Carolina
More Information
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Responsible Party: Chelsea Shope, Principal Investigator, Medical University of South Carolina
ClinicalTrials.gov Identifier: NCT04861064    
Other Study ID Numbers: 00106369
First Posted: April 27, 2021    Key Record Dates
Last Update Posted: February 24, 2022
Last Verified: February 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
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Lymphangioma
Lymphatic Abnormalities
Congenital Abnormalities
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Neoplasms
Lymphatic Diseases
Sirolimus
 Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs