Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia

Phase 1/2 Completed 12 enrolled AM-Pharma

RandomizedParallel-groupOpen-labelTreatment

Summary

The goal of this clinical trial is to compare the effectiveness of two doses of ilofotase alfa, an enzyme replacement treatment, in patients with hypophosphatasia (HPP). The main question it aims to answer is if the harmful accumulating levels of extracellular inorganic pyrophosphate (PPi) and pyridoxal 5'-phosphate (PLP) can be reduced with ilofotase alfa. Researchers will compare the two doses of ilofotase alfa to see if treatment effects differ between the doses.

Timeline

Start: 2023-05-15
Primary completion: 2023-07-07
Completion: 2023-07-12

Outcome

Met primary endpoint

registry p <0.0001; Difference in LS Means -11.17 (95% CI -16.52 to -5.82); Mixed Models Analysis NCT05890794 ↗

registry p <0.0001; Mean Difference (Final Values) 30.89 (95% CI 20.831 to 40.941); t-test, 2 sided NCT05890794 ↗

paper These data demonstrate that single-dose ilofotase alfa enhances AP activity and results in dose-dependent reductions in primary disease-specific biomarkers PMID 41378916 ↗

Publications

Seefried L, Bernholz J, Kraan M, Nitschke Y, Rutsch F, Mallek M, Kleinert S, Genest F. A randomized Phase 1b trial evaluating the pharmacodynamics of ilofotase alfa in adults with hypophosphatasia. J Bone Miner Res. 2026 Mar 2;41(3):231-241. doi: 10.1093/jbmr/zjaf185.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ilofotase Alfa	Protein / enzyme biologic	0.8 mg/kg	Intravenous
Subject	Ilofotase Alfa	Protein / enzyme biologic	3.2 mg/kg	Intravenous