Last updated 7 days ago

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa

114 patients around the world

Available in Spain, Brazil, Argentina, United States

Alexion Pharmaceuticals, Inc.

114Patients around the world

This study is for people with

Hypophosphatasia

To 130 Years

All Gender

: Diagnosis of HPP documented in the medical records
Must meet 1 of the following criteria: 1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory (Section 8.7 ) 2. Plasma PLP above the upper limit of normal (ULN) during the Screening Period (central or local laboratory results allowed per local regulations)
Serum ALP activity below the age- and sex-adjusted normal range during the screening period, without a probable cause other than HPP
Two separate 6MWTs at below 85% of the predicted distance (for age, sex, weight, and height) during the Screening Period without a probable cause other than HPP

: History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, neurological disorders, or any other disorders that are capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study intervention; or interfering with the interpretation of data as determined by the Investigator
Diagnosis of primary or secondary hyperparathyroidism
Hypoparathyroidism, unless secondary to HPP
Any new fracture within 12 weeks before Day 1 (excluding pseudofractures)
Planned surgical intervention which may impact the results of study assessments (in the opinion of the Investigator) during the Randomized Evaluation Period
History of allergy or hypersensitivity to any ingredient contained in ALXN1850 or the placebo comparator (Table 9)

StudyHICKORY

SponsorAlexion Pharmaceuticals, Inc.

Conditions

Requirements

To 130 YearsAll Gender

Unique study IDclinicaltrials.gov

NCT06079281