NCT07875972
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
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Record History
All 1 version: 1 with changes below.
- Study First Submitted
Endpoints
Primary
Serious adverse event (SAE) incidence
Timeframe: Week 0, Week 1, Week 4, Week 8, Week 12, Week 16, Week 24, and Week 28
Adverse event (AE) incidence and severity
Timeframe: Week 0, Week 1, Week 4, Week 8, Week 12, Week 16, Week 24, and Week 28
Dose-limiting toxicity (DLT)
Timeframe: Week 4
Number of subjects who discontinue treatment prematurely
Timeframe: Week 0, Week 1, Week 4, Week 8, Week 12, Week 16, and Week 24
Number of patients who discontinue treatment prematurely due to adverse events
Timeframe: Week 0, Week 1, Week 4, Week 8, Week 12, Week 16, and Week 24
Number of patients with clinically significant abnormal laboratory and ancillary examinations (including routine laboratory tests, cardiac safety, infection markers, bleeding markers, and new-onset tumor examinations)
Timeframe: Week -12, Week -8, Week -4, Week 0, Week 1, Week 4, Week 8, Week 12, Week 16, and Week 24; tumor markers and echocardiography at Week 0, Week 4, Week 12, and Week 24; CT of the chest, abdomen, and pelvis at Week 0 and Week 24
Secondary (15)
Plasma Orelabrutinib concentration
Cerebrospinal fluid (CSF) Orelabrutinib concentration
Peripheral blood PBMC BTK target occupancy (TOR %)
Peripheral blood PBMC p-BTK level
Peripheral blood B-cell count
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ALSFRS-R score and its rate of change slope
Forced vital capacity (FVC)
Medical Research Council (MRC) Muscle Strength Score
Composite survival rate at 6 months of treatment(Composite endpoint of overall survival (defined as time to death from any cause) or respiratory insufficiency (defined as tracheostomy or daily use of non-invasive ventilation ≥22 hours for ≥10 consecutive
Change from baseline to the follow-up time point in ALSAQ-40 quality of life
Slow vital capacity (SVC)
Cerebrospinal fluid (CSF)-to-plasma concentration ratio
Change from baseline to the follow-up time point in EuroQol 5-Dimension (EQ-5D)
Change from baseline to the follow-up time point in Mini-Mental State Examination (MMSE)
Change from baseline to the follow-up time point in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)
Arms and interventions
Cohort 1: Orelabrutinib 50 mg QDExperimental
Participants receive orelabrutinib 50 mg orally once daily (QD) for 24 weeks.
Cohort 2: Orelabrutinib 100 mg QDExperimental
Participants receive orelabrutinib 100 mg orally once daily (QD) for 24 weeks.
Cohort 3 (Optional): Orelabrutinib 150 mg QDExperimental
Optional exploratory cohort. Participants receive orelabrutinib 150 mg orally once daily (QD) for 24 weeks.
Drugs: Orelabrutinib