Study of ALXN1850 in Participants With Hypophosphatasia (HPP)

Phase 1 Completed 15 enrolled Alexion Pharmaceuticals, Inc.

NaSequentialOpen-labelBasic science

Summary

This is an open-label, dose-escalating study to assess safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and immunogenicity of ALXN1850 when given intravenous (IV) and subcutaneous (SC) to adults with HPP.

Timeline

Start: 2021-09-28
Primary completion: 2022-08-24
Completion: 2022-08-24

Publications

Dahir KM, Shannon A, Dunn D, Voegtli W, Dong Q, Hasan J, Pradhan R, Pelto R, Pan WJ. Safety, pharmacokinetics, and pharmacodynamics of efzimfotase alfa, a second-generation enzyme replacement therapy: phase 1, dose-escalation study in adults with hypophosphatasia. J Bone Miner Res. 2024 Sep 26;39(10):1412-1423. doi: 10.1093/jbmr/zjae128.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	efzimfotase alfa	Protein / enzyme biologic	15 mg	Intravenous
Subject	efzimfotase alfa	Protein / enzyme biologic	45 mg	Intravenous
Subject	efzimfotase alfa	Protein / enzyme biologic	90 mg	Intravenous

Study of ALXN1850 in Participants With Hypophosphatasia (HPP)

Summary

Timeline

Publications

Drugs

Indications