Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
Summary
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study was to test the safety and efficacy of two doses of the study drug called asfotase alfa as compared to a control group to see effects on adolescents and adults with HPP.
Timeline
- Start
- 2010-06
- Primary completion
- 2016-06
- Completion
- 2016-06
Outcome
Mixed primary results
registry MEAN Concurrent Control: 3.13 (Standard Deviation 242.721) ng/mL NCT01163149 ↗
registry MEAN Combined Asfotase Alfa Group: -397.72 (Standard Deviation 455.249) ng/mL NCT01163149 ↗
registry analysis (superiority test); Concurrent Control vs Combined Asfotase Alfa Group; p = 0.0285; Hodges-Lehman-Sen -302.05 (95% CI -626.40 to -59.20) NCT01163149 ↗
registry MEAN Concurrent Control: -1.052 (Standard Deviation 2.9248) uM NCT01163149 ↗
registry MEAN Combined Asfotase Alfa Group: -2.100 (Standard Deviation 1.3335) uM NCT01163149 ↗
registry analysis (superiority test); Concurrent Control vs Combined Asfotase Alfa Group; p = 0.0715; Hodges-Lehman-Sen -1.825 (95% CI -3.210 to 0.230) NCT01163149 ↗
Publications
- Background Millan JL, Narisawa S, Lemire I, Loisel TP, Boileau G, Leonard P, Gramatikova S, Terkeltaub R, Camacho NP, McKee MD, Crine P, Whyte MP. Enzyme replacement therapy for murine hypophosphatasia. J Bone Miner Res. 2008 Jun;23(6):777-87. doi: 10.1359/jbmr.071213.
- Simmons JH, Rush ET, Petryk A, Zhou S, Martos-Moreno GA. Dual X-ray absorptiometry has limited utility in detecting bone pathology in children with hypophosphatasia: A pooled post hoc analysis of asfotase alfa clinical trial data. Bone. 2020 Aug;137:115413. doi: 10.1016/j.bone.2020.115413. Epub 2020 May 14.
- Gospe SM 3rd, Santiago-Turla C, DeArmey SM, Cummings TJ, Kishnani PS, Bhatti MT. Ectopic Ocular Surface Calcification in Patients With Hypophosphatasia Treated With Asfotase Alfa. Cornea. 2019 Jul;38(7):896-900. doi: 10.1097/ICO.0000000000001947.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | asfotase alfa | Protein / enzyme biologic | 0.3 mg/kg | Subcutaneous |
| Subject | asfotase alfa | Protein / enzyme biologic | 0.5 mg/kg | Subcutaneous |