Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster I
Hematology Disease Topics & Pathways:
Research, clinical trials, autoimmune disorders, Biological therapies, Antibody Therapy, adult, autoimmune hemolytic anemia, elderly, Clinical Research, drug development, Diseases, Immune Disorders, Therapies, immunology, young adult , Monoclonal Antibody Therapy, Biological Processes, Study Population, Human
Aim: Describe the rationale and study design of ENERGY, an ongoing, adaptive, phase 2/3 multicenter, randomized, double-blind, placebo-controlled study that will evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of nipocalimab compared with placebo in patients with wAIHA (NCT04119050).
Methods: Subjects ≥18 years of age who have been diagnosed with primary or secondary wAIHA and are currently receiving treatment for wAIHA or have previously received treatment for wAIHA will be included in the study. Subjects with cold antibody AIHA, cold agglutinin syndrome, mixed type (i.e., warm and cold) AIHA, or paroxysmal cold hemoglobinuria will be excluded. Stable doses of corticosteroids or immunosuppressants will be allowed. Approximately 111 patients will be randomized 1:1:1 to receive nipocalimab at two different dose schedules or placebo. Following completion of 24 weeks of double-blind treatment, patients may enter an open-label extension period to receive nipocalimab for 144 weeks with a follow-up period of 6 weeks after last assessment.
Results: ENERGY will include approximately 160 sites across 19 countries, including Brazil, Canada, China, Czech, Egypt, France, Germany, Greece, Hungary, Japan, Israel, Malaysia, Netherlands, Italy, Poland, South Korea, Spain, the United Kingdom, and the United States. The primary endpoint is percentage of participants achieving durable response of improvement in hemoglobin (Hgb). The secondary endpoints include change from baseline in the total score from the Functional Assessment of Chronic Illness Therapy-Fatigue Scale, corticosteroid dose reduction from baseline, and normalization of hemolytic markers.
Conclusion/Summary: The results of ENERGY have the potential to identify a novel treatment option to address the significant unmet needs of patients with wAIHA. Enrollment is ongoing in this clinical trial.
Disclosures: Murakhovskaya: Incyte: Research Funding; Alexion: Research Funding; Annexon: Research Funding; Kezar: Research Funding; Rigel: Consultancy, Research Funding; Sanofi: Consultancy, Research Funding; Novartis: Consultancy, Research Funding. Fattizzo: Sobi: Speakers Bureau; Amgen: Consultancy; Janssen: Consultancy; Momenta: Consultancy; Alexion: Consultancy, Speakers Bureau; Novartis: Consultancy, Speakers Bureau. Ebrahim: Janssen Pharmaceutical Companies of J&J: Current Employment, Current equity holder in private company, Current holder of stock options in a privately-held company. Sweet: Janssen Research & Development, LLC: Current Employment, Current equity holder in private company, Current holder of stock options in a privately-held company. Shu: Janssen Research & Development, LLC: Current Employment, Current equity holder in private company, Current holder of stock options in a privately-held company.
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