Weekly Sirolimus Therapy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Sirolimus.
- Who it may be relevant to
- Registry conditions: Venous Malformation, Lymphatic Malformation. Basic parameters: from 2 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
Overview
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.
Interventions
- Drug Sirolimus
Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months.
Primary outcome measures
- Change in size of lesion [Time frame: Baseline and 6 months]
- Change in size of lesion through photograph [Time frame: Baseline and 6 months]
Secondary outcome measures (8)
- Number of side effects experienced [Time frame: Month One]
- Number of side effects experienced [Time frame: Month Two]
- Number of side effects experienced [Time frame: Month Three]
- Number of side effects experienced [Time frame: Month Four]
- Number of side effects experienced [Time frame: Month Five]
- Number of side effects experienced [Time frame: Month Six]
- Change in quality of life as assessed by questionnaire [Time frame: Baseline and 6 months]
- Number of participants with laboratory abnormalities [Time frame: From baseline visit to 2 month visit]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Medical University of South Carolina — Charleston
Identifiers
NCT: NCT04861064 · 00106369