NCT04189315
Withdrawn
Phase 4
A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia
Overview
- Phase
- Phase 4
- Intervention
- asfotase alfa
- Conditions
- Hypophosphatasia
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary Endpoint
- Change From Baseline To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1
- Status
- Withdrawn
- Last Updated
- 5 years ago
Overview
Brief Summary
The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).
Investigators
Eligibility Criteria
Inclusion Criteria
- •Male or female aged ≥ 18 years at the time of signing the informed consent form
- •Clinical diagnosis of pediatric-onset HPP based on signs and symptoms consistent with HPP
- •Past medical history that includes at least one nonvertebral fracture (or pseudofracture) incurred without evidence of significant trauma.
- •The presence of a current fracture is not necessary, but for participants with current unhealed fracture(s) or pseudofracture(s) of the lower extremity(ies) (that is, femoral, tibial, fibular, metatarsal) documentation must be provided of the presence of these fractures for at least 3 months prior to screening (with or without surgical intervention)
Exclusion Criteria
- •Medical condition, serious concurrent or recurrent illness and/or injury, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance or the assessment of study endpoints, including all protocol required evaluations and follow up activities, or may put the patient at risk
- •Primary or secondary hyperparathyroidism or hypoparathyroidism
- •History of hypersensitivity to any ingredient contained in asfotase alfa
- •Oral bisphosphonate use within 6 to 12 months (depending on the half-life of the drug as assessed by the investigator) and intravenous (IV) bisphosphonate use within 12 months prior to screening
- •Denosumab use within 18 months prior to screening
- •Asfotase alfa use within 6 months prior to screening
- •Teriparatide/parathyroid hormone analog use within 2 months prior to screening
- •Treatment with strontium or sclerostin inhibitors within 6 months prior to the first dose of study drug
- •Vitamin B6 use for at least 2 weeks prior to screening
- •Serum 25-hydroxy (25-OH) vitamin D below 20 nanogram (ng)/milliliter (mL), with repletion and recheck allowed at screening (results from local laboratory may be used if within 4 weeks of screening)
Arms & Interventions
Group 1 Asfotase Alfa
Participants will be administered asfotase alfa per approved dose for 36 weeks.
Intervention: asfotase alfa
Group 2 Asfotase Alfa
Participants will be administered asfotase alfa per approved dose for 12 weeks and then asfotase alfa at a lower dose for 24 weeks.
Intervention: asfotase alfa
Outcomes
Primary Outcomes
Change From Baseline To Week 36 In Plasma Concentrations Of Inorganic Pyrophosphate (PPi) In Group 1
Time Frame: Baseline, Week 36
Secondary Outcomes
- Change From Baseline To Week 36 In Plasma Concentrations Of PPi In Group 2(Baseline, Week 36)
- Change From Baseline To Week 36 In The 36-item Short-Form Survey (SF-36) Physical Component Summary (PCS) Score In Groups 1 And 2(Baseline, Week 36)
- Change From Baseline To Week 36 In The Repeated Chair Stand Test (A Component Of The Short Physical Performance Battery [SPPB]) In Groups 1 And 2(Baseline, Week 36)
Similar Trials
Completed
Phase 4
A Phase 4, Open-label Study of KRYSTEXXA® (Pegloticase) Co-administered With Methotrexate (MTX) in Patients With Uncontrolled Gout (FORWARD OL)Uncontrolled GoutChronic GoutNCT04762498Amgen50
Completed
Phase 4
Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Optimark (Gadoversetamide) in Pediatric PatientsPathological ProcessesNCT01092754Guerbet100
Completed
Phase 2
Safety, Tolerability, Pharmacokinetic, and Efficacy Study of SPD489 in Preschool Children With Attention-deficit/Hyperactivity DisorderAttention Deficit Hyperactivity Disorder (ADHD)NCT02402166Shire24
Completed
Phase 4
Safety, Tolerability, Pharmacokinetics and Efficacy of ARCOPlasmodium FalciparumMalaria, FalciparumNCT01930331Ifakara Health Institute60
Recruiting
Phase 2
Pharmacokinetic Study to Evaluate Anti-mycobacterial Activity of TMC207 in Combination With Background Regimen (BR) of Multidrug Resistant Tuberculosis (MDR-TB) Medications for Treatment of Children/Adolescents With Pulmonary MDR-TBNCT02354014Janssen Research & Development, LLC60