Efficacy and Safety of Interferon-Gamma Monoclonal Antibody Combined With Dexamethasone in the Treatment of Refractory Adult Secondary Hemophagocytic Lymphohistiocytosis

NCT07531069 | Completed DMC

• 1 other identifier: 2024K017
• Study Type: observational
• Target: 22
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this observational study is to learn if interferon-gamma monoclonal antibody combined with dexamethasone works to treat adults with refractory secondary hemophagocytic lymphohistiocytosis (HLH). The main questions it aims to answer are: How well does this treatment help patients recover from refractory secondary HLH? How safe is this treatment for these patients? Participants will receive interferon-gamma monoclonal antibody and dexamethasone as part of their clinical care. Researchers will monitor participants with regular blood tests, physical exams, and safety checks for up to 8 weeks of treatment. They will collect information about how participants respond to treatment and any side effects that occur. This study will include about 22 adult participants at Huadong Hospital Affiliated to Fudan University.

Conditions

Adult Refractory Secondary Hemophagocytic Lymphohistiocytosis; Secondary Hemophagocytic Lymphohistiocytosis; HLH

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate (ORR)

  The proportion of participants who achieve complete response (CR) or partial response (PR) at the end of 8-week treatment, defined by normalized or improved clinical and laboratory parameters including ferritin, triglycerides, sCD25, blood cell counts, and hemophagocytosis.

  Up to 8 weeks after initiation of treatment

Secondary Outcomes (3)

• Progression-Free Survival (PFS)

  Up to 12 months after treatment initiation

• Overall Survival (OS)

  Up to 12 months after treatment initiation

• Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Up to 28 days after the last dose of study treatment

Study Arms (1)

Observation group

Participants receive IFN-γ monoclonal antibody by weight-based intravenous infusion. Dexamethasone is administered orally or intravenously. Prophylaxis for infections and standard supportive care are provided as clinically needed. Treatment continues for up to 8 weeks, with regular safety and efficacy assessments.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This study will enroll adult participants aged 18 years and older with a confirmed diagnosis of refractory secondary hemophagocytic lymphohistiocytosis (HLH) based on the HLH-2004 diagnostic criteria. Participants must have demonstrated inadequate response or disease progression following standard first-line or second-line HLH-directed therapy. All participants must be able to provide written informed consent prior to any study-related procedures.

You may qualify if:

• Age ≥ 18 years
• Confirmed diagnosis of secondary hemophagocytic lymphohistiocytosis (HLH) according to HLH-2004 criteria
• Refractory HLH defined as inadequate response or progression after at least 2 weeks of standard first-line or second-line therapy
• Able to provide written informed consent

You may not qualify if:

• Active infection with mycobacteria, histoplasma, CMV, herpes zoster, or HIV
• Left ventricular ejection fraction \< 50%
• Concurrent use of other anti-neoplastic or investigational agents
• Other uncontrolled infections judged by the investigator
• Psychiatric disorders or poor protocol compliance

Sponsors & Collaborators

• Fudan University: lead

Study Sites (1)

Huadong Hospital, Fudan University

Shanghai, Shanghai Municipality, 200040, China

Location

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 12 Months
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director

Study Record Dates

• First Submitted: April 2, 2026
• First Posted: April 15, 2026
• Study Start: May 21, 2024
• Primary Completion: August 31, 2025
• Study Completion: August 31, 2025
• Last Updated: April 15, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: 1 year after completion of this study

Locations

CN (1)