drugset / Trial / NCT02797821
Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
RandomizedParallel-groupOpen-labelTreatment
Summary
The purpose of this study was to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of asfotase alfa in adult participants with pediatric-onset HPP.
Timeline
- Start
- 2016-06-06
- Primary completion
- 2017-06-21
- Completion
- 2017-06-21
Outcome
Met primary endpoint
paper Differences in least squares mean (LSM) changes in PPi were significant with 2.0 mg/kg (p = 0.0008) and 3.0 mg/kg (p < 0.0001) vs. 0.5 mg/kg. PMID 32987199 ↗
Publications
- Pan WJ, Pradhan R, Pelto R, Seefried L. Pharmacokinetics of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia. J Clin Pharmacol. 2021 Oct;61(10):1334-1343. doi: 10.1002/jcph.1870. Epub 2021 Jun 19.
- Seefried L, Kishnani PS, Moseley S, Denker AE, Watsky E, Whyte MP, Dahir KM. Pharmacodynamics of asfotase alfa in adults with pediatric-onset hypophosphatasia. Bone. 2021 Jan;142:115664. doi: 10.1016/j.bone.2020.115664. Epub 2020 Sep 26.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | asfotase alfa | Protein / enzyme biologic | 0.5 mg/kg | Subcutaneous |
| Subject | asfotase alfa | Protein / enzyme biologic | 2 mg/kg | Subcutaneous |
| Subject | asfotase alfa | Protein / enzyme biologic | 3 mg/kg | Subcutaneous |