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
Overview
- Phase
- Phase 2
- Status
- Active, not recruiting
- Sponsor
- Hutchison Medipharma Limited
- Enrollment
- 110
- Locations
- 39
- Primary Endpoint
- Phase II: overall Hb response rate
Overview
Brief Summary
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
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
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
Arms & Interventions
HMPL-523
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.
Intervention: HMPL-523(300mg PO QD) (Drug)
Placebo
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.
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Phase II: overall Hb response rate
Time Frame: 24Weeks
Phase II: Overall Hb response rate: the proportion of patients with overall Hb response by Week 24
Secondary Outcomes
- Phase III: Durable Hb response rate(24Weeks)