HMPL-523 (Sovleplenib) in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia

Study Overview
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Brief Summary
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Phase II Study: To evaluate the safety and preliminary efficacy of HMPL-523 in adult patients with wAIHA Phase III Studies: Confirmation of Efficacy safety and of HMPL-523 in Adult Patients With wAIHA

Detailed Description
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Phase II Study: the proportion of patients with overall Hb response by Week 24 Phase III study: the proportion of patients who achieve a durable response by Week 24

Official Title
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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

Conditions
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Warm Antibody Autoimmune Hemolytic Anemia

Intervention / Treatment
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* Drug: HMPL-523(300mg PO QD)
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Voluntarily signed the informed consent form (ICF); Males or females aged 18 to 75 years; Patients diagnosed with primary wAIHA or secondary wAIHA whose underlying diseases are stable; Organs in good function. Exclusion Criteria: Patients with other types of AIHA other than wAIHA; Patients with secondary wAIHA with unstable underlying disease; Patients with drug-induced secondary wAIHA; Patients with infections requiring systemic treatment; Patients previously treated with Syk inhibitors (e.g., fostamatinib); Patients with known allergy to the active ingredients or excipients of the study drug; Patients with serious psychological or mental disorder; Alcoholic or drug abuser; Female patients who are pregnant and lactating.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: HMPL-523<br>Phase II: Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 8 weeks and at least 16 weeks open-label treatment. Phase III: Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 24 weeks and enter open-label phase in the opinion of the Investigator. | Drug: HMPL-523(300mg PO QD)<br>* No more than two doses will be explored<br>* Other names: Sovleplenib;|
| Placebo Comparator: Placebo<br>Phase II: Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 8 weeks and at least 16 weeks open-label treatment. Phase III: Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 24 weeks and enter open-label phase in the opinion of the Investigator. | Drug: Placebo<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Phase II: overall Hb response rate | Phase II: Overall Hb response rate: the proportion of patients with overall Hb response by Week 24 | 24Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Phase III: Durable Hb response rate | Phase III: Durable Hb response rate: the proportion of patients who achieve a durable response by Week 24 | 24Weeks |