Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 8 April 2024
• Main ID: NCT04119050
• Date of registration: 18/09/2019
• Prospective Registration: No
• Primary sponsor: Janssen Research & Development, LLC
• Public title: Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia
• Scientific title: 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
• Date of first enrolment: August 15, 2019
• Target sample size: 111
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04119050
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Investigator, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

Brazil, Canada, China, Czechia, Egypt, France, Germany, Greece, Hungary, Israel, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Poland, Spain, Ukraine, United Kingdom, United States

Contacts

• Name: Janssen Research & Development, LLC Clinical Trial
• Affiliation: Janssen Research & Development, LLC

• Name: Study Contact

Key inclusion & exclusion criteria

Inclusion criteria:

- Participants greater than or equal to (>=)18 years of age

- Have been diagnosed with warm autoimmune hemolytic anemia (wAIHA) for at least 3
months, and are currently receiving treatment for wAIHA or have previously received
treatment for wAIHA (treatment-naive participants are not eligible)

- Participants must be able to understand and voluntarily provide written informed
consent to participate in the study and comply with all study procedures

Exclusion criteria:

- Participants must not be pregnant or breastfeeding

- Participants must not have other clinically relevant abnormalities currently or in
their history that the Investigator would deem them ineligible to participate

- Have been diagnosed with cold antibody autoimmune hemolytic anemia (AIHA), cold
agglutinin syndrome, mixed type (that is, warm and cold) AIHA, or paroxysmal cold
hemoglobinuria



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Warm Autoimmune Hemolytic Anemia

Intervention(s)

Drug: M281

Drug: Placebo

Primary Outcome(s)

Percentage of Participants Achieving Durable Response of Improvement in Hemoglobin (Hgb) [Time Frame: Up to Week 20 of the double-blind period]

Secondary Outcome(s)

Absolute Change from Baseline in Average Daily Dose of Prednisone or Equivalent [Time Frame: Baseline (Day 1, Week 0) and at Week 24]

Change From Baseline in Hemolytic Marker - Indirect Bilirubin [Time Frame: Baseline (Day 1, Week 0) through Week 24]

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 [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Mean Time During Which the Primary Endpoint is Maintained [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Patient-reported Status As Assessed by Patient Global Impression of Change (PGIC) Scale Score [Time Frame: At Week 24]

Change From Baseline in Hemolytic Marker - Lactate Dehydrogenase [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Change From Baseline in Reticulocyte Count [Time Frame: 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 [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Change From Baseline in Hemolytic Marker - Haptoglobin [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Change From Baseline in Hgb Concentration [Time Frame: 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 [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Change From Baseline in Patient Global Impression of Severity (PGIS) [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Hgb Range at Steady State [Time Frame: 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 [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Time to Hgb Response [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Change From Baseline in EuroQol 5-dimension 5-level ( EQ-5D-5L) Scale Score [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Change from Baseline in Average Daily Dose of Prednisone or Equivalent [Time Frame: Baseline (Day 1, Week 0) and at Week 24]

Change From Baseline in the Total Score From the FACIT-Fatigue Scale at the end of the Double-blind Period (Week 24) [Time Frame: Baseline (Day 1, Week 0) through Week 24 of the double-blind period]

Change From Baseline in the Total Score, Item Scores, and Impact and Experience Domains From the FACIT-Fatigue Scale [Time Frame: Baseline (Day 1, Week 0) through Week 24 of the double-blind period]

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 [Time Frame: At 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 [Time Frame: Up to 24 weeks]

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 [Time Frame: Baseline (Day 1, Week 0) through Week 24]

Secondary ID(s)

2019-000720-17

2023-505321-14-00

CR108987

MOM-M281-006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors