Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)

Phase 2 Completed 24 enrolled Apellis Pharmaceuticals, Inc.

RandomizedParallel-groupOpen-labelTreatment

Summary

This study is to assess the safety, tolerability, preliminary efficacy, and pharmacokinetics of APL-2 in subjects with warm Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD).

Timeline

Start: 2017-08-31
Primary completion: 2022-09-12
Completion: 2022-09-12

Publications

Roman E, Fattizzo B, Shum M, Hanna W, Lentz SR, Araujo SSS, Al-Adhami M, Grossi FV, Gertz MA. Safety and efficacy of pegcetacoplan treatment for cold agglutinin disease and warm antibody autoimmune hemolytic anemia. Blood. 2025 Jan 23;145(4):397-408. doi: 10.1182/blood.2023022549.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Pegcetacoplan	Peptide	270 mg	Subcutaneous
Subject	Pegcetacoplan	Peptide	360 mg	Subcutaneous

Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)

Summary

Timeline

Publications

Drugs

Indications