Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia
- Registration Number
- NCT04119050
- Lead Sponsor
- Janssen Research & Development, LLC
- Brief Summary
The main purpose of this study is to evaluate the efficacy and safety of M281 in participants with warm autoimmune hemolytic anemia (wAIHA).
- Detailed Description
The study consists of a 24-week double-blind, placebo control period, a 144-week open-label extension period and follow-up period of 8 weeks after last study drug administration. Eligible participants will be randomized to placebo or nipocalimab (2 dose levels) during the double-blind period and nipocalimab (2 dose levels) during the open-label extension period.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 111
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description M281 administered every 4 weeks (double-blind period) M281 Participants will receive M281 administered every 4 weeks alternating with placebo every 4 weeks during the 24 weeks double-blind period. M281 administered every 4 weeks (double-blind period) Placebo Participants will receive M281 administered every 4 weeks alternating with placebo every 4 weeks during the 24 weeks double-blind period. M281 administered every 2 weeks (double-blind period) M281 Participants will receive M281 administered every 2 weeks during the 24 weeks double-blind period. Placebo administered every 2 weeks (double-blind period) Placebo Participants will receive M281 matching placebo administered every 2 weeks during the 24 weeks double-blind period. M281 administered every 4 weeks (open-label extension period) M281 Participants will receive M281 administered every 4 weeks during the 144 weeks open-label extension period. M281 administered every 2 weeks (open-label extension period) M281 Participants will receive M281 administered every 2 weeks during the 144 weeks open-label extension period.
- Primary Outcome Measures
Name Time Method Percentage of Participants Achieving Durable Response of Improvement in Hemoglobin (Hgb) Up to Week 20 of the double-blind period
- Secondary Outcome Measures
Name Time Method 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 The FACIT-Fatigue is a self-administered 13-item questionnaire that assess patient-reported fatigue associated with chronic illness therapy. It assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue. A positive change from baseline score indicates an improvement.
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 The FACIT-Fatigue is a self-administered 13-item questionnaire that assess patient-reported fatigue associated with chronic illness therapy. It assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue. A positive change from baseline score indicates an improvement.
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 average daily dose of prednisone or equivalent at week 24 among participants on prednisone or equivalent at baseline will be reported.
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 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 will be reported.
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 The FACIT-Fatigue scale is a 13-item self-administered questionnaire that assesses both the physical and functional consequences of fatigue. Each question is answered on a 5-point scale, where 0 means "not at all," and 4 means "very much." The FACIT-Fatigue scale score ranges from 0 to 52, with higher scores denoting lower levels of fatigue. A positive change from baseline score indicates an improvement.
Absolute Change from Baseline in Average Daily Dose of Prednisone or Equivalent Baseline (Day 1, Week 0) and at Week 24 Absolute change from baseline in average daily dose of prednisone or equivalent at Week 24 among all participants will be reported.
Change From Baseline in EuroQol 5-dimension 5-level ( EQ-5D-5L) Scale Score Baseline (Day 1, Week 0) through Week 24 The EQ-5D-5L quality of life questionnaire will be used to assess health related quality of life status. The 5 dimensions are mobility, self-care, usual activities, pain/discomfort, and anxiety/depression; each dimension is rated by the patient on a 5 level scale (no problems, slight problems, moderate problems, severe problems, extreme problems).
Hgb Range at Steady State Baseline (Day 1, Week 0) through Week 24 It will be estimated using a model-based longitudinal analysis of Hgb/hemolysis parameters in relationship to IgG level and dose regimen.
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 The SF-36v2 will be used to assess general quality of life. The 36 items on the SF-36 health survey encompass the following 8 domains: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health. The 8 domains can be aggregated into 2 summary scales that reflect physical and mental health: a physical component summary (PCS) and a mental component summary (MCS). Responses to all items are rated on a 3-, 5- or 6-point Likert scale. Higher scores indicate a higher level of functioning. A positive change from baseline score indicates an improvement.
Patient-reported Status As Assessed by Patient Global Impression of Change (PGIC) Scale Score At Week 24 The PGIC will assess if there has been an improvement or decline in patient-reported status since the beginning of the treatment. The PGIC is a 7-point response scale. Participants will be asked to rate their current fatigue as compared to when they started the study, using the following 7-point scale: 1 = Much better, 2 = Moderately better, 3 = A little better, 4 = No change, 5 = A little worse, 6 = Moderately worse, and 7 = Much worse.
Change From Baseline in Patient Global Impression of Severity (PGIS) Baseline (Day 1, Week 0) through Week 24 The PGIS will be used to assess the severity of warm autoimmune hemolytic anemia (wAIHA) fatigue symptoms. The PGIS is a 5-point response scale. Participant will be asked to rate their fatigue over the past 7 days using the following 5-point scale: 1 = None, 2 = Mild, 3 = Moderate, 4 = Severe, and 5 = Very severe.
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 Percentage of participants who achieve corticosteroid reduction to \<= 7.5 mg/day of oral prednisone (or equivalent) at Week 24 of the double-blind period, among participants with prednisone or equivalent \>7.5 mg/day at baseline will be reported.
Trial Locations
- Locations (171)
MemorialCare Medical Group
🇺🇸Fountain Valley, California, United States
University of Southern California
🇺🇸Los Angeles, California, United States
Compassionate Cancer Care
🇺🇸Riverside, California, United States
American Institute of Research
🇺🇸Whittier, California, United States
University of Colorado Anschutz Medical Campus
🇺🇸Aurora, Colorado, United States
GNP Research
🇺🇸Cooper City, Florida, United States
University of Florida College of Medicine
🇺🇸Gainesville, Florida, United States
21st Century Oncology
🇺🇸Jacksonville, Florida, United States
Lakes Research
🇺🇸Miami Lakes, Florida, United States
AdventHealth Cancer Institute
🇺🇸Orlando, Florida, United States
Scroll for more (161 remaining)MemorialCare Medical Group🇺🇸Fountain Valley, California, United States