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

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ClinicalTrials.gov Identifier: NCT04119050
Recruitment Status : Recruiting
First Posted : October 8, 2019
Last Update Posted : November 23, 2022
Sponsor:
Information provided by (Responsible Party):
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).

Condition or disease Intervention/treatment Phase
Warm Autoimmune Hemolytic Anemia Drug: M281 Drug: Placebo Phase 2 Phase 3

Expanded Access : An investigational treatment associated with this study is temporarily not available outside the clinical trial.   More info ...

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 111 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : October 24, 2019
Estimated Primary Completion Date : November 3, 2023
Estimated Study Completion Date : October 15, 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arm Intervention/treatment
Experimental: M281 administered every 4 weeks (double-blind period)
Participants will receive M281 administered every 4 weeks alternating with placebo every 4 weeks during the 24 weeks double-blind period.
Drug: M281
M281 injection administered as intravenous infusion
Other Name: Nipocalimab, JNJ-80202135

Drug: Placebo
Placebo administered as intravenous infusion

Experimental: M281 administered every 2 weeks (double-blind period)
Participants will receive M281 administered every 2 weeks during the 24 weeks double-blind period.
Drug: M281
M281 injection administered as intravenous infusion
Other Name: Nipocalimab, JNJ-80202135

Experimental: Placebo administered every 2 weeks (double-blind period)
Participants will receive M281 matching placebo administered every 2 weeks during the 24 weeks double-blind period.
Drug: Placebo
Placebo administered as intravenous infusion

Experimental: M281 administered every 4 weeks (open-label extension period)
Participants will receive M281 administered every 4 weeks during the 144 weeks open-label extension period.
Drug: M281
M281 injection administered as intravenous infusion
Other Name: Nipocalimab, JNJ-80202135

Experimental: M281 administered every 2 weeks (open-label extension period)
Participants will receive M281 administered every 2 weeks during the 144 weeks open-label extension period.
Drug: M281
M281 injection administered as intravenous infusion
Other Name: Nipocalimab, JNJ-80202135




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

Secondary Outcome Measures :
  1. 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 ]
    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.

  2. 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 ]
    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.

  3. 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 average daily dose of prednisone or equivalent at week 24 among participants on prednisone or equivalent at baseline will be reported.

  4. 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 ]
  5. 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 ]
  6. 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 ]
  7. 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 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.

  8. Change From Baseline in Hgb Concentration [ Time Frame: Baseline (Day 1, Week 0) through Week 24 ]
  9. Change From Baseline in Reticulocyte Count [ Time Frame: Baseline (Day 1, Week 0) through Week 24 ]
  10. Change From Baseline in Hemolytic Marker - Lactate Dehydrogenase [ Time Frame: Baseline (Day 1, Week 0) through Week 24 ]
  11. Change From Baseline in Hemolytic Marker - Haptoglobin [ Time Frame: Baseline (Day 1, Week 0) through Week 24 ]
  12. Change From Baseline in Hemolytic Marker - Indirect Bilirubin [ Time Frame: Baseline (Day 1, Week 0) through Week 24 ]
  13. Time to Hgb Response [ Time Frame: Baseline (Day 1, Week 0) through Week 24 ]
  14. Mean Time During Which the Primary Endpoint is Maintained [ Time Frame: Baseline (Day 1, Week 0) through Week 24 ]
  15. 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 ]
    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.

  16. Change From Baseline in EuroQol 5-dimension 5-level ( EQ-5D-5L) Scale Score [ Time Frame: 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).

  17. 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 ]
    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.

  18. Change From Baseline in Patient Global Impression of Severity (PGIS) [ Time Frame: 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.

  19. Patient-reported Status As Assessed by Patient Global Impression of Change (PGIC) Scale Score [ Time Frame: 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.

  20. Hgb Range at Steady State [ Time Frame: 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.

  21. Absolute Change from Baseline in Average Daily Dose of Prednisone or Equivalent [ Time Frame: 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.

  22. 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 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04119050


Contacts
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Contact: Study Contact 844-434-4210 Participate-In-This-Study@its.jnj.com

Locations
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Sponsors and Collaborators
Janssen Research & Development, LLC
Investigators
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Study Director: Janssen Research & Development, LLC Clinical Trial Janssen Research & Development, LLC
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Janssen Research & Development, LLC
ClinicalTrials.gov Identifier: NCT04119050    
Other Study ID Numbers: CR108987
2019-000720-17 ( EudraCT Number )
MOM-M281-006 ( Other Identifier: Janssen Research & Development, LLC )
First Posted: October 8, 2019    Key Record Dates
Last Update Posted: November 23, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Janssen Research & Development, LLC:
Warm Autoimmune Hemolytic Anemia
M281 (Nipocalimab)
wAIHA
JNJ-80202135
Additional relevant MeSH terms:
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Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Autoimmune
Hemolysis
Hematologic Diseases
Pathologic Processes
Autoimmune Diseases
Immune System Diseases