A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (HDFN)

Summary

The purpose of this study is to evaluate the safety in mother and neonate/infant of M281 administered to pregnant women who are at high risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN). The effectiveness of the investigational drug M281 will be measured by looking at the percentage of participants with live birth at or after gestational age (GA) 32 weeks and without a need for an intrauterine transfusion (IUT) throughout their entire pregnancy.

Timeline

Start

2018-04-05

Primary completion

2024-08-05

Completion

2024-08-05

Outcome

Met primary endpoint

paper Live birth at 32 weeks' gestation or later without intrauterine transfusions occurred in 7 of 13 pregnancies (54%; PMID 39115062 ↗

paper 95% confidence interval, 25 to 81 PMID 39115062 ↗

Publications

• de Winter DP, Moise KJ, Ling LE, Oepkes D, Tiblad E, Joanne Verweij EJT, Smoleniec J, Sachs UJ, Bein G, Kilby MD, Miller RS, Devlieger R, Streisand JB, Bredius RGM, Cafone J, Lam E, Leu JH, Mirza A, Nelson RM, Smith V, Schwartz LB, Tjoa ML, Saeed-Khawaja S, Komatsu Y, Lopriore E. Infant Immunity after Maternal Nipocalimab in Severe Hemolytic Disease of the Fetus and Newborn. NEJM Evid. 2026 Feb;5(2):EVIDoa2500097. doi: 10.1056/EVIDoa2500097. Epub 2026 Jan 27.
• Moise KJ Jr, Ling LE, Oepkes D, Tiblad E, Verweij EJTJ, Lopriore E, Smoleniec J, Sachs UJ, Bein G, Kilby MD, Miller RS, Devlieger R, Audibert F, Emery SP, Markham K, Norton ME, Ocon-Hernandez O, Pandya P, Pereira L, Silver RM, Windrim R, Streisand JB, Leu JH, Mirza A, Smith V, Schwartz LB, Tjoa ML, Saeed-Khawaja S, Komatsu Y, Bussel JB; UNITY Study Group. Nipocalimab in Early-Onset Severe Hemolytic Disease of the Fetus and Newborn. N Engl J Med. 2024 Aug 8;391(6):526-537. doi: 10.1056/NEJMoa2314466.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Nipocalimab	Monoclonal antibody	30 mg/kg	Intravenous
Subject	Nipocalimab	Monoclonal antibody	45 mg/kg	Intravenous

Indications