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

Phase 3RecruitingNCT04119050

Janssen Research & Development, LLC111 enrolled

Overview

The main purpose of this study is to evaluate the efficacy and safety of M281 in participants with warm autoimmune hemolytic anemia (wAIHA).

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

111

Conditions

Warm Autoimmune Hemolytic Anemia

Interventions

M281DRUG

M281 injection administered as intravenous infusion

PlaceboDRUG

Placebo administered as intravenous infusion

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (171)

Outcomes

Primary Outcomes

Percentage of Participants Achieving Durable Response of Improvement in Hemoglobin (Hgb)

Time frame: Up to Week 20 of the double-blind period

Secondary Outcomes

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

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 frame: 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)

Time frame: Baseline (Day 1, Week 0) through Week 24 of the double-blind period

Change from Baseline in Average Daily Dose of Prednisone or Equivalent

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.

Time frame: Baseline (Day 1, Week 0) and at 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

Central Contacts

Study Contact

CONTACT

844-434-4210 Participate-In-This-Study1@its.jnj.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

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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 any Time During the Study

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

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

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.

Time frame: Up to 24 weeks

Change From Baseline in Hgb Concentration

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

Change From Baseline in Hemolytic Marker - Lactate Dehydrogenase

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 Hemolytic Marker - Indirect Bilirubin

Time frame: Baseline (Day 1, Week 0) through Week 24

Time to Hgb 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

Change From Baseline in the Total Score, Item Scores, and Impact and Experience Domains From the FACIT-Fatigue Scale

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.

Time frame: Baseline (Day 1, Week 0) through Week 24 of the double-blind period

Change From Baseline in EuroQol 5-dimension 5-level ( EQ-5D-5L) Scale Score

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).

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

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.

Time frame: Baseline (Day 1, Week 0) through Week 24

Change From Baseline in Patient Global Impression of Severity (PGIS)

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.

Time frame: Baseline (Day 1, Week 0) through Week 24

Patient-reported Status As Assessed by Patient Global Impression of Change (PGIC) Scale Score

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.

Time frame: At Week 24

Hgb Range at Steady State

It will be estimated using a model-based longitudinal analysis of Hgb/hemolysis parameters in relationship to IgG level and dose regimen.

Time frame: Baseline (Day 1, Week 0) through Week 24

Absolute Change from Baseline in Average Daily Dose of Prednisone or Equivalent

Absolute change from baseline in average daily dose of prednisone or equivalent at Week 24 among all participants will be reported.

Time frame: Baseline (Day 1, Week 0) and at 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

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.

Time frame: At Week 24