Health TrialsbyHealthPanel

Weekly Sirolimus Therapy

ConditionsVenous MalformationLymphatic Malformation
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age2+
SponsorMedical University of South Carolina

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patient 2 years of age and older

Venous, lymphatic, or venolymphatic malformations

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Sirolimus

    Drug

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

Treatment groups

24 Participants
are divided into 1 treatment group
Group A: Treatment GroupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Change in size of lesion

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

Time frame
Baseline and 6 months
2

Change in size of lesion through photograph

Will be evaluating clinical photographs of lesions at each patient visit

Time frame
Baseline and 6 months

Secondary outcomes

1

Number of side effects experienced

Patient will complete side effect questionnaires at each visit

Time frame
Month One
2

Number of side effects experienced

Patient will complete side effect questionnaires at each visit

Time frame
Month Two
3

Number of side effects experienced

Patient will complete side effect questionnaires at each visit

Time frame
Month Three
4

Number of side effects experienced

Patient will complete side effect questionnaires at each visit

Time frame
Month Four

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Medical University of South Carolina

Lead sponsor

Source ClinicalTrials.gov
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