Sirolimus Versus Sirolimus Plus Prednisolone for Kaposiform Hemangioendothelioma (NCT03188068) | Clinical Trial Compass
CompletedPhase 2
Sirolimus Versus Sirolimus Plus Prednisolone for Kaposiform Hemangioendothelioma
China30 participantsStarted 2017-06-01
Plain-language summary
Kaposiform hemangioendothelioma (KHE) is a rare vascular neoplasm that occurs predominantly in infancy or early childhood. KHE has a nearly equal sex ratio. The annual incidence of KHE has been estimated at 0.071 per 100,000 children. KHE presents with intermediate-malignant and locally aggressive characteristics but without distant metastases.
This pilot trial studies sirolimus versus sirolimus plus pednisolone in treating patients diagnosed with kaposiform hemangioendothelioma (KHE) and Kasabach-Merritt phenomemon (KMP) that cannot be removed by surgery. The purpose of this study is to compare the efficacy and safety of orally administered sirolimus versus sirolimus plus pednisolone in the treatment of KHE associated with KMP.
Who can participate
Age range
1 Day – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Clinical features and histological findings consistent with progressive, non-resectable KHE associated with KMP.
. Patients must be 0 - 18 years of age at the time of study entry.
. Without functional impairment requiring treatment of corticosteroid.
. Total bilirubin less than or equal to 1.5 x upper limit of normal (ULN)for age, and
. ALT and AST less than or equal to 2.5 x upper limit normal (ULN) for age.
. 0-5 years of age maximum serum creatinine (mg/dL) of 0.8
. 6-10 years of age maximum serum creatinine (mg/dL) of 1.0
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The changes of platelet counts
Timeframe: 2 months
2
The changes of fibrinogen levels
Timeframe: 2 months
3
The changes in KHE volume
Timeframe: 6 and 12 months
4
The changes in the patient's symptoms and/or complications.