Treatment of Warm Antibody Autoimmune Hemolytic Anemia with HMPL-523

A Randomized, Double-Blind, Placebo-Controlled Phase II/III Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of HMPL-523 in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

This study is testing a new oral medication called HMPL-523 to see if it can help adults with warm antibody autoimmune hemolytic anemia improve their hemoglobin levels and feel better.

Quick facts

• Phase: PHASE2; PHASE3
• Study type: Interventional
• Enrollment: 110 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Hutchmed (industry)
• Drugs / interventions: fostamatinib
• Locations: 5 sites (Lanzhou, Gansu and 4 other locations)
• Trial ID: NCT05535933 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of HMPL-523, an oral medication, in adult patients diagnosed with warm antibody autoimmune hemolytic anemia (wAIHA). The study is divided into two phases: Phase II focuses on assessing preliminary efficacy and safety, while Phase III aims to confirm the drug's effectiveness and safety over a 24-week period. Participants will receive either HMPL-523 or a placebo, and the primary outcome is the proportion of patients achieving a durable hemoglobin response. The trial includes patients with stable underlying conditions related to wAIHA.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 75 with a diagnosis of primary or stable secondary wAIHA.

Not a fit: Patients with other types of autoimmune hemolytic anemia or those with unstable underlying conditions will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from wAIHA, potentially improving their quality of life.

How similar studies have performed: Other studies have shown promise in treating autoimmune conditions with similar approaches, but this specific treatment is being evaluated for the first time in this context.

Eligibility criteria

Inclusion Criteria:

1. Voluntarily signed the informed consent form (ICF);
2. Males or females aged 18 to 75 years;
3. Patients diagnosed with primary wAIHA or secondary wAIHA whose underlying diseases are stable;
4. Organs in good function.

Exclusion Criteria:

1. Patients with other types of AIHA other than wAIHA;
2. Patients with secondary wAIHA with unstable underlying disease;
3. Patients with drug-induced secondary wAIHA;
4. Patients with infections requiring systemic treatment;
5. Patients previously treated with Syk inhibitors (e.g., fostamatinib);
6. Patients with known allergy to the active ingredients or excipients of the study drug;
7. Patients with serious psychological or mental disorder;
8. Alcoholic or drug abuser;
9. Female patients who are pregnant and lactating.

Where this trial is running

Lanzhou, Gansu and 4 other locations

• Guangdong Provincial People's Hospital — Lanzhou, Gansu, China (RECRUITING)
• The First affiliated hospital of nanchang uiversity — Nanchang, Jiangxi, China (RECRUITING)
• Bethune First Hospital Of Jilin University — Changchun, Jilin, China (RECRUITING)
• Peking Union Medical College Hospital — Beijing, China (RECRUITING)
• Hematology Hospital of Chinese Academy of Medical Sciences — Tianjin, China (RECRUITING)

Study contacts

• Principal investigator: Fengkui Zhang, professor — offices director
• Study coordinator: Angela Niu

Conditions: Warm Antibody Autoimmune Hemolytic Anemia