What is the mechanism of action of this treatment?

Common treatments for Autoimmune Hemolytic Anemia (AIHA) include corticosteroids, which broadly suppress the immune system, and immunosuppressive drugs like rituximab that target B cells to reduce antibody production. Plasmapheresis is another option to remove antibodies from the blood. Nipocalimab, an investigational drug, inhibits the neonatal Fc receptor (FcRn), reducing the levels of pathogenic IgG antibodies. This targeted approach is significant for AIHA patients as it directly addresses the mechanism responsible for red blood cell destruction, potentially offering a more specific and effective treatment.

What side effects are associated with this type of intervention?

Common treatments for Autoimmune Hemolytic Anemia (AIHA) include corticosteroids, immunosuppressants, and monoclonal antibodies like rituximab. Corticosteroids can cause side effects such as weight gain, hypertension, diabetes, and increased risk of infections. Immunosuppressants, including azathioprine and cyclophosphamide, may lead to bone marrow suppression, increased infection risk, and liver toxicity. Rituximab, a monoclonal antibody, can cause infusion reactions, infections, and rarely, progressive multifocal leukoencephalopathy (PML). Treatments targeting the neonatal Fc receptor (FcRn) like Nipocalimab aim to reduce pathogenic IgG antibodies and may have side effects including headaches, infections, and infusion-related reactions.

What prior FDA approvals exist?

The FDA has approved several treatments for Autoimmune Hemolytic Anemia (AIHA), including corticosteroids, immunosuppressants like rituximab, and splenectomy. Rituximab, a monoclonal antibody targeting CD20 on B cells, is commonly used for its immunosuppressive effects. While there are no specific FDA-approved drugs that target the neonatal Fc receptor (FcRn) like Nipocalimab, the approach of reducing pathogenic IgG antibodies is a novel and promising strategy in the treatment of AIHA.

What other types of research exist?

A promising novel alternative to Nipocalimab for Autoimmune Hemolytic Anemia patients is M281, which is currently being evaluated for its efficacy and safety in adults with warm autoimmune hemolytic anemia (wAIHA). Patients should discuss this option with their healthcare provider to determine its suitability for their specific condition.

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Monoclonal Antibodies

M281 for Autoimmune Hemolytic Anemia (ENERGY Trial)

Phase 2 & 3

Recruiting

Research Sponsored by Janssen Research & Development, LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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)

Must not have

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline (day 1, week 0) and at week 24

Summary

This trial is testing a medication called nipocalimab to see if it can help people with a blood disorder called warm autoimmune hemolytic anemia. The medication works by stopping the immune system from destroying red blood cells, which can help reduce anemia symptoms.

Who is the study for?

Adults over 18 with warm autoimmune hemolytic anemia (wAIHA) who are currently or previously treated for wAIHA can join. Those with cold antibody AIHA, mixed type AIHA, or paroxysmal cold hemoglobinuria cannot participate. Pregnant or breastfeeding individuals and those with other significant health issues are also excluded.

What is being tested?

The trial is testing the effectiveness and safety of a medication called M281 in adults with wAIHA compared to a placebo. Participants will either receive M281 or a placebo to determine if M281 improves their condition.

What are the potential side effects?

While specific side effects of M281 aren't listed here, common side effects in trials may include reactions at the injection site, headaches, nausea, fatigue, and potential immune system responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am 18 years old or older.

Select...

I have been treated for warm autoimmune hemolytic anemia for over 3 months.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have been diagnosed with a type of anemia caused by cold-reacting antibodies.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline (day 1, week 0) and at week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline (day 1, week 0) and at week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day 1, week 0) and at week 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

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

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

Change From Baseline in Medical Outcomes Study Short Form 36 Item Health Survey Version 2 Acute (SF-36v2) Score

+9 more

Side effects data

From 2020 Phase 2 trial • 68 Patients • NCT03772587

21%

Rash

14%

Urinary Tract Infection

14%

Diarrhoea

14%

Oedema Peripheral

14%

Nasopharyngitis

Glucose Tolerance Impaired

Musculoskeletal Chest Pain

Hypothyroidism

Eyelid Ptosis

Vision Blurred

Vomiting

Malaise

Peripheral Swelling

Asymptomatic Bacteriuria

Herpes Zoster

Lymphocyte Count Decreased

Headache

Rash Erythematous

Skin Swelling

Brachiocephalic Vein Thrombosis

Thrombocytopenia

Abdominal Pain Upper

Nausea

Vessel Puncture Site Pruritus

Vessel Puncture Site Swelling

Cellulitis

Conjunctivitis

Blood Pressure Increased

Neutrophil Percentage Increased

Hypophosphataemia

Back Pain

Muscle Spasms

Muscle Twitching

Cough

Dysphonia

Erythema

Pruritus

100%

80%

60%

40%

20%

Study treatment Arm

Nipocalimab 60 mg/kg (Q2W)

Placebo

Nipocalimab 5 Milligrams/Kilogram (mg/kg)

Nipocalimab 30 mg/kg

Nipocalimab 60 mg/kg

Trial Design

5Treatment groups

Experimental Treatment

Group I: Placebo administered every 2 weeks (double-blind period)Experimental Treatment1 Intervention

Participants will receive M281 matching placebo administered every 2 weeks during the 24 weeks double-blind period.

Group II: M281 administered every 4 weeks (open-label extension period)Experimental Treatment1 Intervention

Participants will receive M281 administered every 4 weeks during the 144 weeks open-label extension period.

Group III: M281 administered every 4 weeks (double-blind period)Experimental Treatment2 Interventions

Participants will receive M281 administered every 4 weeks alternating with placebo every 4 weeks during the 24 weeks double-blind period.

Group IV: M281 administered every 2 weeks (open-label extension period)Experimental Treatment1 Intervention

Participants will receive M281 administered every 2 weeks during the 144 weeks open-label extension period.

Group V: M281 administered every 2 weeks (double-blind period)Experimental Treatment1 Intervention

Participants will receive M281 administered every 2 weeks during the 24 weeks double-blind period.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo

1995

Completed Phase 3

~2670

M281

2016

Completed Phase 2

~140

0 / 3Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Autoimmune Hemolytic Anemia (AIHA) include corticosteroids, which broadly suppress the immune system, and immunosuppressive drugs like rituximab that target B cells to reduce antibody production. Plasmapheresis is another option to remove antibodies from the blood. Nipocalimab, an investigational drug, inhibits the neonatal Fc receptor (FcRn), reducing the levels of pathogenic IgG antibodies. This targeted approach is significant for AIHA patients as it directly addresses the mechanism responsible for red blood cell destruction, potentially offering a more specific and effective treatment.