Zanubrutinib for HLH

Zanubrutinib Monotherapy in Patients With Hemophagocytic Lymphohistiocytosis

This study is a prospective single-arm clinical study, focusing on Hemophagocytic lymphohistocytosis，to evaluate the clinical efficacy and safety of zanubrutinib.

Study Overview

• Status: Completed
• Conditions: HLH
• Intervention / Treatment: Drug: Zanubrutinib

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Beijing Friendship Hospital, Capital Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Unlimited gender, age ≥ 14 years old;
2. HLH was diagnosed according to HLH-04 diagnostic criteria;
3. Before the study, there was no severe liver function injury; Serum creatinine ≤ 1.5 times the upper limit of normal value;
4. Sign informed consent

Exclusion Criteria:

1. Allergic to zebutinib;
2. Currently active important cardiovascular diseases with clinical significance; The functions of important organs such as heart and lung unrelated to HLH were seriously abnormal;
3. Known human immunodeficiency virus (HIV) or active hepatitis B (HBV) or hepatitis C (HCV) infection;
4. Pregnant or lactating women and patients of childbearing age who refuse to take appropriate contraceptive measures during this trial;
5. Serious mental illness;
6. Active massive hemorrhage of internal organs;
7. Uncontrollable infection;
8. At the same time, participate in other clinical researchers

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: zanubrutinib	Drug: Zanubrutinib; The dosage of zanubrutinib is 160mg twice a day (80mg twice a day for patients over 75 years old) for 2 months. The remission rate is evaluated after treatment. If it is above PR, continue the dose maintenance treatment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
evaluation of treatment response	A complete response was defined as normalization of all of the quantifiable symptoms and laboratory markers of HLH, including levels of sCD25, ferritin, and triglyceride; hemoglobin; neutrophil counts; platelet counts; and alanine aminotransferase (ALT). A partial response was defined as at least a 25% improvement in 2 or more quantifiable symptoms and laboratory markers as follows: sCD25 response was>1.5-fold decreased; ferritin and triglyceride decreased at least 25%; for patients with an initial neutrophil count of<0.5 ×109/L, a response was defined as an increase by at least 100% to>0.5×109/L; for patients with a neutrophil count of 0.5 to 2.0× 109/L, an increase by at least 100% to >2.0 × 109/L was considered a response; and for patients with ALT >400 U/L,response was defined as an ALT decrease of at least 50%.	Change from before and 2,4,6 and 8 weeks after initiating therapy
Progression Free Survival	from date of inclusion to date of progression, relapse, or death from any cause	6 months
Adverse events	Adverse events including myelosuppression, infection, hemorrhage	6 months

Collaborators and Investigators

• Sponsor: Beijing Friendship Hospital

Study record dates

Study Major Dates

• Study Start (Actual): February 1, 2022
• Primary Completion (Actual): August 1, 2023
• Study Completion (Actual): August 1, 2023

Study Registration Dates

• First Submitted: April 2, 2022
• First Submitted That Met QC Criteria: April 2, 2022
• First Posted (Actual): April 11, 2022

Study Record Updates

• Last Update Posted (Actual): April 11, 2024
• Last Update Submitted That Met QC Criteria: April 10, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Protein Kinase Inhibitors; Tyrosine Kinase Inhibitors; Zanubrutinib
• Other Study ID Numbers: Zanubrutinib HLH

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No