Low- vs High-Dose Sirolimus With Prednisolone for KHE/KMP in Children

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

In this randomized Phase 2/3 study, pediatric patients with progressive, non-resectable KHE and KMP are assigned to either low-dose sirolimus (trough 4-8 ng/mL) plus prednisolone or standard high-dose sirolimus (trough 10-15 ng/mL) plus prednisolone, in a 1:1 ratio. Participants undergo routine blood tests and imaging to monitor response and safety, with primary endpoints focused on objective response rate and platelet recovery, as well as treatment-related adverse events. The trial aims to determine if lowering the sirolimus dose maintains tumor and hematologic control while reducing toxicity. Researchers will compare the two regimens to see if the lower-dose approach can achieve noninferior efficacy and potentially better tolerability.

Why it matters

Who can join: the full rules

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 requiring treatment of corticosteroid.
* Organ function requirements:

  1 Adequate liver function:
  1. Total bilirubin less than or equal to 1.5 x upper limit of normal (ULN)for age, and
  2. ALT and AST less than or equal to 2.5 x upper limit normal (ULN) for age.

  2 Adequate renal function:
  1. 0-5 years of age maximum serum creatinine (mg/dL) of 0.8
  2. 6-10 years of age maximum serum creatinine (mg/dL) of 1.0
  3. 11-15 years of age maximum serum creatinine (mg/dL) of 1.2
  4. 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.

Exclusion Criteria:

* Allergy to sirolimus or other rapamycin analogues.
* Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of randomization.
* Patients must not be known to be Human Immunodeficiency Virus positive or known immunodeficiency. Testing is not required unless a condition is suspected.
* 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).
* Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of sirolimus.
* Patients who have a history of malignancy.
* Patients with an inability to participate or to follow the study treatment and assessment plan.

Where it's running

• West China Hospital of Sichuan University
  Chengdu, Sichuan, China Recruiting

Study contacts

• Principal investigator: Jiangyuan Zhou, MD — West China Hospital
• Study coordinator: Yi Ji, PhD
• Email: jijiyuanyuan@163.com
• Phone: 02885423453

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.