Zanubrutinib for HLH

• Sponsor: Beijing Friendship Hospital (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT05320575
• Collaborator: (none)
• Enrollment: 30
• Location: 1
• Arm: 1
• Anticipated Duration (Months): 26.1
• Patients Per Site Per Month: 1.1

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
HLH	Drug: Zanubrutinib	Phase 3

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 30 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Zanubrutinib Monotherapy in Patients With Hemophagocytic Lymphohistiocytosis
• Actual Study Start Date: Jan 26, 2021
• Anticipated Primary Completion Date: Jan 1, 2023
• Anticipated Study Completion Date: Apr 1, 2023

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures

1. evaluation of treatment response [Change from before and 2,4,6 and 8 weeks after initiating therapy]

   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%.

2. Progression Free Survival [6 months]

   from date of inclusion to date of progression, relapse, or death from any cause

3. Adverse events [6 months]

   Adverse events including myelosuppression, infection, hemorrhage

Eligibility Criteria

Criteria

• Ages Eligible for Study: 14 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Beijing Friendship Hospital, Capital Medical University	Beijing		China

Sponsors and Collaborators

• Beijing Friendship Hospital

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Zhao Wang, Dr, Beijing Friendship Hospital
• ClinicalTrials.gov Identifier: NCT05320575
• Other Study ID Numbers: Zanubrutinib HLH
• First Posted: Apr 11, 2022
• Last Update Posted: Apr 11, 2022
• Last Verified: Apr 1, 2022

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Zanubrutinib

Study Results

No Results Posted as of Apr 11, 2022