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Clinical Trials in China / NCT06079359
Active, not recruiting Phase 3

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

NCT06079359 · tracked via the Priya Life Science China tracker
Sponsor
Alexion Pharmaceuticals, Inc.
Phase
Phase 3
Started
2024-05-14
Last updated
2026-05-28

Condition(s) studied

Hypophosphatasia

Investigational drug(s) / intervention(s)

ALXN1850Placebo

ALXN1850: Participants will receive a ALXN1850 via subcutaneous (SC) injection.

Placebo: Participants will receive placebo via SC injection.

Study summary

The primary purpose of this study is to evaluate the efficacy of ALXN1850 versus placebo on radiographic outcomes in pediatric participants with HPP who have not previously been treated with asfotase alfa.

Eligibility

Sex
ALL
Min age
2 Years
Max age
11 Years
Healthy volunteers
No
Inclusion Criteria: * Diagnosis of HPP documented in the medical records, and the following criteria fulfilled without other probable cause than HPP: 1. Presence of HPP-related rickets on skeletal X-rays during the Screening Period, with a minimum Rickets Severity Score (RSS) of 1.0 AND 2. Serum ALP activity below the age- and sex-adjusted normal range during the Screening Period as measured by the Central Laboratory OR 2 documented serum ALP activity results, at least 15 days apart, below the age- and sex-adjusted local laboratory normal range during the 24 months before the Day 1 Visit. Note: Local laboratories need to be Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval. * Must meet 1 of the following criteria: 1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a 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) * Tanner stage 2 or less during the Screening Period Exclusion Criteria: * 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

Primary outcome measure(s)

Trial sites (32)

FacilityCityRegionStatus
Research Site Baltimore Maryland
Research Site Kansas City Missouri
Research Site Durham North Carolina
Research Site Nedlands Australia
Research Site Parkville Australia
Research Site Brussels Belgium
Research Site Brasília Brazil
Research Site Porto Alegre Brazil
Research Site Recife Brazil
Research Site Salvador Brazil
Research Site São Paulo Brazil
Research Site São Paulo Brazil
Research Site Calgary Alberta
Research Site Winnepeg Manitoba
Research Site Beijing China
Research Site Guangzhou China
Research Site Shanghai China
Research Site Shenzhen China
Research Site Helsinki Finland
Research Site Ashkelon Israel
Research Site Chihuahua City Mexico
Research Site Lodz Poland
Research Site Bucharest Romania
Research Site Madrid Spain
Research Site Vitoria-Gasteiz Spain
Research Site Stockholm Sweden
Research Site Taipei Taiwan
Research Site Ankara Turkey (Türkiye)
Research Site Bursa Turkey (Türkiye)
Research Site Erzurum Turkey (Türkiye)
Research Site Istanbul Turkey (Türkiye)
Research Site Birmingham United Kingdom

More Alexion Pharmaceuticals, Inc. trials in China

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06079359 on ClinicalTrials.gov ↗ ← All trials in China