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

Janssen (J&J Innovative Medicine)

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Warm Autoimmune Hemolytic Anemia

Treatments: Drug: M281; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT04119050

CR108987

2023-505321-14-00 (Registry Identifier)

MOM-M281-006 (Other Identifier)

2019-000720-17 (EudraCT Number)

Details and patient eligibility

About

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

Full 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.

Enrollment: 111 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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111 participants in 5 patient groups

M281 administered every 4 weeks (double-blind period)

Experimental group

Description:

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

Treatment:

Drug: Placebo

Drug: M281

M281 administered every 2 weeks (double-blind period)

Experimental group

Description:

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

Treatment:

Drug: M281

Placebo administered every 2 weeks (double-blind period)

Experimental group

Description:

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

Treatment:

Drug: Placebo

M281 administered every 4 weeks (open-label extension period)

Experimental group

Description:

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

Treatment:

Drug: M281

M281 administered every 2 weeks (open-label extension period)

Experimental group

Description:

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

Treatment:

Drug: M281