NCT07656909RecruitingPhase 2/3
Low- vs High-Dose Sirolimus With Prednisolone for KHE and KMP
Low-dose Versus High-dose Sirolimus Combined With Prednisolone for Kaposiform Hemangioendothelioma With Kasabach-Merritt Phenomenon: a Randomized Noninferiority Trial
Source: ClinicalTrials.gov · Last updated 18 Jun 2026
Brief summary (registry, in English)
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 1:1 to receive the assigned regimen, undergo routine blood and imaging monitoring, and be evaluated for clinical response and adverse events.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2/3
- Study type
- Interventional
- Enrollment
- 76 (estimated)
- First submitted
- 15 Jun 2026
- Start date
- 16 Jun 2026
- Primary completion date
- 31 Dec 2027
- Lead sponsor
- Yi Ji
- NCT number
- NCT07656909
What you commit to
- Study type: Interventional · Phase 2/3
- Planned participants: 76
- From 16 Jun 2026 to the primary completion date, 31 Dec 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of randomization.
- Other concurrent severe and/or uncontrolled medical disease which could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration).
Other illness
- Allergy to sirolimus or other rapamycin analogues.
- Patients must not be known to be Human Immunodeficiency Virus positive or known immunodeficiency. Testing is not required unless a condition is suspected.
- Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of sirolimus.
- Patients who have a history of malignancy.
Other
- Patients with an inability to participate or to follow the study treatment and assessment plan.
Who the study is looking for
- Presenting a KHE with the following characteristics:
- 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.
- Organ function requirements:
- 1 Adequate liver function:
- 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.
- 2 Adequate renal function:
- 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
- 11-15 years of age maximum serum creatinine (mg/dL) of 1.2
- 16-18 years of age maximum serum creatinine (mg/dL) of 1.5
- Adequate bone marrow function: Absolute Neutrophil Count (ANC) greater than or equal to 1 x 10 to the ninth/Liter.
- Consent of parents (or the person having parental authority in families): Signed and dated written informed consent.
Age in the registry: 1 day to 18 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| West China Hospital of Sichuan University Coordinator of this site? Confirm its status | Chengdu | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.