Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia: A Multicenter, Randomized, Double-blind, Placebo-controlled Study With a Long-term Open-label Extension

简要总结

详细描述

主要结局

次要结局

• Change From Baseline in the Total Score From the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale at the Time of Durable Response(Baseline (Day 1, Week 0) through Week 24)
• Number of Participants That Simultaneously Attain Normal Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin Levels at a Minimum of 3 Consecutive Visits After Baseline(Baseline (Day 1, Week 0) through Week 24)
• Change From Baseline in the Total Score From the FACIT-Fatigue Scale at the end of the Double-blind Period (Week 24)(Baseline (Day 1, Week 0) through Week 24 of the double-blind period)
• Time to Hgb Response(Baseline (Day 1, Week 0) through Week 24)
• Change from Baseline in Average Daily Dose of Prednisone or Equivalent(Baseline (Day 1, Week 0) and at Week 24)
• Change From Baseline in Reticulocyte Count(Baseline (Day 1, Week 0) through Week 24)
• Change From Baseline in Hemolytic Marker - Haptoglobin(Baseline (Day 1, Week 0) through Week 24)
• Percentage of Participants who Experience at Least a 2 g/dL Increase in Hgb From Baseline and Normalization of Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin at any Time During the Study(Baseline (Day 1, Week 0) through Week 24)
• Percentage of Participants who Experience at Least a 2 g/dL Increase in Hgb From Baseline and Normalization of Lactate Dehydrogenase, Haptoglobin, and Indirect Bilirubin at 3 Consecutive Visits(Baseline (Day 1, Week 0) through Week 24)
• Percentage of Participants who Achieve the Durable Response in Improvement of Hgb During the Double-blind Period and Maintain that Response for Up to 24 Weeks, Without the Need of Rescue Therapy(Up to 24 weeks)
• Change From Baseline in Hemolytic Marker - Lactate Dehydrogenase(Baseline (Day 1, Week 0) through Week 24)
• Mean Time During Which the Primary Endpoint is Maintained(Baseline (Day 1, Week 0) through Week 24)
• Change From Baseline in Hgb Concentration(Baseline (Day 1, Week 0) through Week 24)
• Change From Baseline in Hemolytic Marker - Indirect Bilirubin(Baseline (Day 1, Week 0) through Week 24)
• Change From Baseline in the Total Score, Item Scores, and Impact and Experience Domains From the FACIT-Fatigue Scale(Baseline (Day 1, Week 0) through Week 24 of the double-blind period)
• Absolute Change from Baseline in Average Daily Dose of Prednisone or Equivalent(Baseline (Day 1, Week 0) and at Week 24)
• Change From Baseline in EuroQol 5-dimension 5-level ( EQ-5D-5L) Scale Score(Baseline (Day 1, Week 0) through Week 24)
• Hgb Range at Steady State(Baseline (Day 1, Week 0) through Week 24)
• Change From Baseline in Medical Outcomes Study Short Form 36 Item Health Survey Version 2 Acute (SF-36v2) Score(Baseline (Day 1, Week 0) through Week 24)
• Patient-reported Status As Assessed by Patient Global Impression of Change (PGIC) Scale Score(At Week 24)
• Change From Baseline in Patient Global Impression of Severity (PGIS)(Baseline (Day 1, Week 0) through Week 24)
• Percentage of participants who Achieve Corticosteroid Reduction to less than or equal to (<=) 7.5 milligrams per day (mg/day) of Oral Prednisone (or Equivalent), Among Participants with Prednisone or Equivalent greater than (>) 7.5 mg/day at Baseline(At Week 24)