Primary study objective: To confirm the efficacy of HBM9161 subcutaneous injection for the treatment of gMG in Chinese patients
This is the phase 3 part of a multi-center, randomized, double-blind, placebo-controlled, group sequential designed, seamless phase 2/3 study to confirm the efficacy and safety of HBM9161 (HL161) subcutaneous injection for the treatment of gMG in patients with generalized myasthenia gravis. Study drug will be administrated by cycle. Each cycle consists of 5-week treatment period, followed by a 4-week observation period (9 weeks in total). During the 5-week treatment period, subjects will receive 6 doses of study drug (HBM9161 680 mg or matching placebo) by subcutaneous injection, once a week (QW). After the 5-week treatment period, subjects will be followed up weekly to assess efficacy and safety. After the completion of first treatment cycle, subjects may start the second cycle of treatment if re-treatment criteria are met. Otherwise, subjects will be followed up weekly until they are eligible for the second cycle or the end of the study, whichever is earlier.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
132
HBM9161 Injection
Placebo Injection
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality, China
In the first treatment cycle, percentage of patients with sustained improvement based on Myasthenia Gravis Activities of Daily Living Scale (MG-ADL), among serum AChR-Ab- or MuSK-Ab-positive subjects
Definition of sustained improvement based on MG-ADL: during the treatment cycle (5-week treatment period plus 4-week observation period), a reduction of ≥ 3 points from the baseline of MG-ADL score for 4 consecutive weekly MG-ADL assessments
Time frame: baseline to Day 64
In the first treatment cycle, percentage of patients with sustained improvement based on MG-ADL, among all study subjects, regardless of , regardless of AChR-Ab and MuSK-Ab serologic status
Time frame: Baseline to Day 64
In the first treatment cycle, percentage of subjects with sustained improvement based on Quantitative Myasthenia Gravis Scale (QMG), among serum AChR-Ab- or MuSK-Ab-positive subjects [ Time Frame: baseline to Day 64 ]
Definition of sustained improvement based on QMG: a reduction of ≥ 3 points from the baseline of QMG score for 4 consecutive weekly QMG assessments during the treatment cycle (5-week treatment period plus 4-week observation period)
Time frame: Baseline to Day 64
During the 24-study period, the proportion of weekly MG-ADL assessments that are improved ≥ 3 points from baseline among serum AChR-Ab- or MuSK-Ab-positive subjects
Time frame: baseline to Day 64
In the first treatment cycle, percentage of serum AChR-Ab- or MuSK-Ab-positive subjects who meet the minimal symptom manifestation (MSE, defined as MG-ADL being 0 or 1) criterion at any visit
Time frame: baseline to Day 64
Within the first 2 weeks of the first treatment cycle, percentage of AChR-Ab- or MuSK-Ab-positive subjects whose MG-ADL improved by ≥3 points from baseline
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Time frame: baseline to Day 64
Within the second treatment cycle, percentage of patients with sustained improvement based on MG-ADL, among serum AChR-Ab- or MuSK-Ab-positive subjects during the second 9-week treatment cycle
Time frame: baseline to Day 64