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Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

30 patients around the world

Available in Spain, Brazil, Argentina, Mexico, United States

Alexion Pharmaceuticals, Inc.

30Patients around the world

This study is for people with

Hypophosphatasia

To 11 Years

All Gender

: Diagnosis of HPP documented in the medical records, and the following criteria fulfilled during the Screening Period without other probable cause than HPP: Presence of HPP-related rickets on skeletal X-rays, with a minimum Rickets Severity Score (RSS) of 1.0 AND Serum ALP activity below the age- and sex-adjusted normal range
Must meet 1 of the following criteria: Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory (Section 8.7) OR Plasma PLP above the upper limit of normal (ULN) during the Screening Period (central or local laboratory results allowed per local regulations)
Tanner stage 2 or less during the Screening Period

: 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

StudyMULBERRY

SponsorAlexion Pharmaceuticals, Inc.

Study typeInterventional

Conditions

Hypophosphatasia

Requirements

To 11 YearsAll Gender

Unique study IDclinicaltrials.gov

NCT06079359