Low- vs High-Dose Sirolimus With Prednisolone for KHE and KMP

Recruiting Phase 2 Phase 3 Interventional Study
Kaposiform Hemangioendothelioma (KHE) Kasabach Merritt Phenomenon
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
1 Day – 18
Sex
Any
Trial phase
Phase 2/3
Study type
Interventional
Purpose
Treatment
Participants needed
76 (estimated)
Sponsor
Yi Ji · Other
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About This Trial
This randomized clinical trial evaluates if low-dose sirolimus (target trough 4-8 ng/mL) combined with prednisolone is noninferior in efficacy but superior in safety compared to standard high-dose sirolimus (target trough 10-15 ng/mL) combined with prednisolone in pediatric patients with kaposiform hemangioendothelioma and Kasabach-Merritt phenomenon (KHE with KMP), with participants randomized 1:…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Presenting a KHE with the following characteristics: 1. Clinical features and histological findings consistent with progressive, non-resectable KHE associated with KMP. 2. Patients must be 0 - 18 years of age at the time of study entry. 3. Without functional impairment …
Contacts

Yi Ji, PhD

02885423453

jijiyuanyuan@163.com

CONTACT

Jiangyuan Zhou, MD

13668491160@163.com

CONTACT