Venous MalformationPhase 2NCT04861064
Weekly Sirolimus Therapy
A Phase 2 study testing Sirolimus in people with Venous Malformation and Lymphatic Malformation, run by Medical University of South Carolina at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Venous Malformation, Lymphatic Malformation
- What is being tested?
- Sirolimus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Jan 2022to Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient 2 years of age and older
- Venous, lymphatic, or venolymphatic malformations
Who cannot join
- 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
Show all 9Show fewer
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29403
Stephanie Munie, BS, 843-792-9784, munie@musc.edu
About this study
Official title: Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
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
- Being tested
- Sirolimus
- Medical subject headings
- Lymphatic Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Participants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | Not described Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in size of lesion
Will be measuring the size of the lesions (mm) at each patient visit
Measured: Baseline and 6 months
Change in size of lesion through photograph
Will be evaluating clinical photographs of lesions at each patient visit
Measured: Baseline and 6 months
Secondary outcomes (8)
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Measured: Month One
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Measured: Month Two
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Measured: Month Three
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Measured: Month Four
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Measured: Month Five
Number of side effects experienced
Patient will complete side effect questionnaires at each visit
Measured: Month Six
Change in quality of life as assessed by questionnaire
Patient will complete quality of life questionnaire at each visit
Measured: Baseline and 6 months
Number of participants with laboratory abnormalities
Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored
Measured: From baseline visit to 2 month visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2021
- Study start18 Jan 2022
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Venous MalformationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chelsea Shope, Medical University of South Carolina
- Investigators
- Chelsea Shope, MSCR, Principal investigator, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04861064
- Sponsor's protocol ID
- 00106369
- Status last verified
- Mar 2026
- First submitted
- 23 Apr 2021
- Last updated
- 5 Mar 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations
- ClinicalTrials.gov ID
- NCT04861064
- Acronym / sponsor ID
- 00106369
- Conditions
- Venous Malformation, Lymphatic Malformation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Study start
- 18 Jan 2022 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 27 Apr 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Drug: Sirolimus |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Patient 2 years of age and older
- Venous, lymphatic, or venolymphatic malformations
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SirolimusParticipants will get Sirolimus (1.5-2 2mg/m2) weekly for 6 months. | Drug | Treatment Group |
Primary outcome
Change in size of lesion
Time frame: Baseline and 6 months
Will be measuring the size of the lesions (mm) at each patient visit
Change in size of lesion through photograph
Time frame: Baseline and 6 months
Will be evaluating clinical photographs of lesions at each patient visit
8 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Chelsea Shope, MSCR principal investigator
Medical University of South Carolina
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sirolimus in people with Venous Malformation and Lymphatic Malformation, run by Medical University of South Carolina at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Stephnie Munie, BS (843-566-2453, munie@musc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in size of lesion (time frame: Baseline and 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2026.