Study of ALXN1850 in Participants With Hypophosphatasia (HPP)

Alexion Pharmaceuticals, Inc.15 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

BASIC_SCIENCE

Masking

NONE

Enrollment

Conditions

Hypophosphatasia

Interventions

ALXN1850BIOLOGICAL

ALXN1850 will be administered as an IV infusion and via the SC route.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 64 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Confirmed clinical diagnosis of HPP * Not anticipated to require further treatment with enzyme replacement therapy to treat participant's HPP after study completion * Willing and able to follow protocol-specified contraception requirements * Willing and able to give informed consent Exclusion Criteria: * Primary or secondary hyperparathyroidism or hypoparathyroidism * Fracture within 12 weeks of screening * Current or relevant history of unstable physical or psychiatric illness * Significant allergies * Asfotase alfa use within 6 months and/or positive for asfotase alfa antidrug antibody/neutralizing antibodies

Locations (4)

Research Site

Las Vegas, Nevada, United States

Research Site

Columbus, Ohio, United States

Research Site

Nashville, Tennessee, United States

Research Site

Austin, Texas, United States

Outcomes

Primary Outcomes

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)

TEAEs were defined as any adverse events (AEs) that began or worsened on or after the first dose of treatment until the final follow-up visit. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. TESAEs were defined as any serious AEs that began or worsened on or after the first dose of treatment until the final follow-up visit. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Time frame: Day 1 up to Day 85

Secondary Outcomes

Maximum Observed Plasma Concentration (Cmax) of ALXN1850 Following Intravenous (IV) Dose, Subcutaneous (SC) Dose 1, SC Dose 2 and SC Dose 3

Time frame: Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29

Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity Following IV Dose of ALXN1850

Time frame: Predose, 2, 6 and 12 hours postdose on Days 1, 15, 22 and 29

Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Following SC Dose 1, SC Dose 2 and SC Dose 3

Time frame: Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29

Area Under the Plasma Concentration Versus Time Curve From Time 0 to Dosing Interval (AUCtau) Values of the First SC Versus IV Administration of ALXN1850

Time frame: Predose, 2, 6 and 12 hour postdose on Days 1, 15, 22 and 29

Data from ClinicalTrials.gov

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