Phase II Study: To evaluate the safety and preliminary efficacy of HMPL-523 in adult patients with wAIHA. Phase III Study(Part A): Confirmation of Efficacy safety and of HMPL-523 in adult patients with wAIHA. Phase III Study (Part B): To further evaluate the long-term safety and tolerability of HMPL-523 in adult patients with wAIHA.
Phase II Study: An eight-week randomized, placebo-controlled, double-blind phase followed by at least a 16-week open-label HMPL-523 treatment to evaluate the safety and preliminary efficacy of HMPL-523. The primary endpoint was the proportion of patients with overall Hb response by week 24. Phase III study (Part A):A 24-week randomized, placebo-controlled double-blind phase to evaluate efficacy and safety of HMPL-523. The primary endpoint was the proportion of patients who achieve a durable response during weeks 5 to 24. Phase III Study (Part B): An open-label Phase evaluating long-term safety and efficacy of HMPL-523 treatment. Eligible patients include those with lack of efficacy during the 20-week Phase III Part A treatment, completion of the Phase II study, or completion of 24-week Phase III Part A treatment with investigators assessment of potential benefit from open-label treatment with HMPL-523.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
111
HMPL-523(300mg PO QD)
Placebo(300mg PO QD)
Phase II: Overall Hb response rate
Phase II: Overall Hb response rate: The proportion of patients with overall Hb response by Week 24
Time frame: 24Weeks
Phase III(Part A): Durable Hb response rate
Phase III(Part A):Durable Hb response rate: The proportion of patients who achieve a durable response by Week 24 during Part A
Time frame: 24Weeks
Phase II: Overall Hb response rate
Phase II: Overall Hb response rate: the proportion of patients with overall Hb response by Week 8
Time frame: 8 Weeks
Phase II: Durable Hb response rate
Phase II: Durable Hb response rate: the proportion of patients who achieve a durable response by Week 24.
Time frame: 24 Weeks
Phase II: Median change in Hb
Phase II: Median change from baseline in Hb at Weeks 8 and 24 of treatment.
Time frame: 24 Weeks
Phase II: Effects on reticulocyte count
Phase II: Change from baseline in reticulocyte count at Weeks 8 and 24 of treatment.
Time frame: 24 Weeks
Phase II: Effects on lactate dehydrogenase
Phase II: Change from baseline in lactate dehydrogenase(LDH) at Weeks 8 and 24 of treatment.
Time frame: 24 Weeks
Phase II: Effects on haptoglobin
Phase II: Change from baseline in haptoglobin at Weeks 8 and 24 of treatment.
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Fuyang Hospital Of Anhui Medical University
Fuyang, Anhui, China
Fujian Medical University Union Hospital
Fuzhou, Fujian, China
Lanzhou University Second Hospital
Lanzhou, Gansu, China
Nanfang hospital
Guangzhou, Guangdong, China
Shenzhen Second People's Hospital
Shenzhen, Guangdong, China
Affiliated Hospital of Guangdong Medical University
Zhanjiang, Guangdong, China
Guilin Medical College Affiliated Hospital
Guilin, Guangxi, China
Hainan General Hospital
Haikou, Hainan, China
Affiliated hospital of Hebei university
Baoding, Hebei, China
Affiliated hospital of chengde medical university
Chengde, Hebei, China
...and 29 more locations
Time frame: 24 Weeks
Phase II: Effects on total bilirubin(TBIL)
Phase II: Change from baseline in total bilirubin(TBIL) at Weeks 8 and 24 of treatment.
Time frame: 24 Weeks
Phase II: Proportion of rescue therapy
Phase II: Proportion of patients who received rescue therapy by Weeks 8 and 24 of treatment.
Time frame: 24 Weeks
Phase II: Proportion of patients with dose reduction in baseline anti-wAIHA medications
Phase II: Proportion of patients who had a dose reduction in glucocorticoids or other baseline concomitant anti-wAIHA medications by Weeks 8 and 24 of treatment.
Time frame: 24 Weeks
Phase II: Time to response
Phase II: Time to response
Time frame: 24 Weeks
Phase II: Effect of study treatment on patient fatigue
Phase II: Evaluation of the effect of study treatment on fatigue at Weeks 8 and 24, as assessed by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F)
Time frame: 24 Weeks
Phase II: Effect of study treatment on quality of life
Phase II: Evaluation of the effect of study treatment on quality of life at Weeks 8 and 24, as assessed by the 36-Item Short Form Health Survey (SF-36).
Time frame: 24 Weeks
Phase III(Part A): Overall Hb response rate
Phase III(Part A): Proportion of patients who achieved an overall Hb response during the 20-week and 24-week double-blind treatment periods(defined as at least one Hb value ≥100g/L with an increase of at least 20g/L from baseline, not attributable to rescue therapy).
Time frame: 24 Weeks
Phase III(Part A): Median change in Hb
Phase III(Part A): Median change from baseline in Hb during the 20-week and 24-week double-blind treatment periods.
Time frame: 24 Weeks
Phase III(Part A): Effects on reticulocyte count
Phase III(Part A): Change from baseline in reticulocyte count during the 20-week and 24-week double-blind treatment periods.
Time frame: 24 Weeks
Phase III(Part A): Effects on lactate dehydrogenase
Phase III(Part A): Change from baseline in lactate dehydrogenase(LDH) during the 20-week and 24-week double-blind treatment periods.
Time frame: 24 Weeks
Phase III(Part A): Effects on haptoglobin
Phase III(Part A): Change from baseline in haptoglobin during the 20-week and 24-week double-blind treatment periods.
Time frame: 24 Weeks
Phase III(Part A): Effects on total bilirubin(TBIL)
Phase III(Part A): Change from baseline in total bilirubin(TBIL) during the 20-week and 24-week double-blind treatment periods.
Time frame: 24 Weeks
Phase III(Part A): Proportion of rescue therapy
Phase III(Part A): Proportion of patients who received protocol-defined rescue therapy during the 20-week and 24-week double-blind treatment periods.
Time frame: 24 Weeks
Phase III (Part A): Proportion of patients reducing or discontinuing baseline anti-wAIHA medications
Phase III(Part A):The proportion of patients who reduced or discontinued glucocorticoids or other baseline concomitant anti-wAIHA medications during the 20-week and 24-week double-blind treatment periods.
Time frame: 24 Weeks
Phase III(Part A): Time to first response
Phase III(Part A): Time to first response
Time frame: 24 Weeks
Phase III(Part A): Duration of durable response
Phase III(Part A): Duration of durable response
Time frame: 24 Weeks
Phase III(Part A): Cumulative duration of response
Phase III(Part A): Cumulative duration of response
Time frame: 24 Weeks
Phase III(Part A): Effect of study treatment on patient fatigue
Phase III(Part A): Effect of study treatment on patient fatigue during the 20-week and 24-week treatment periods, as assessed by the FACIT-F score (40 items; range, 0-160), including the FACIT-Fatigue subscale (13 items; range, 0-52).
Time frame: 24 Weeks
Phase III(Part A): Effect of study treatment on patients' quality
Phase III(Part A): Effect of study treatment on patients' quality of life during the 20-week and 24-week treatment periods, as assessed by SF-36.
Time frame: 24 Weeks