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

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Hypophosphatasia

In brief

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.

Key facts

Study ID: NCT06079359
Run by: Alexion Pharmaceuticals, Inc.
People needed: 30
Starts: 2024-05-14
Expected to finish: 2028-08-24
Last updated by the study team: 2026-05-28

Who can join

Age: 2 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Diagnosis of HPP documented in the medical records, and the following criteria fulfilled without other probable cause than HPP:
Presence of HPP-related rickets on skeletal X-rays during the Screening Period, with a minimum Rickets Severity Score (RSS) of 1.0 AND
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:
Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a CLIA certified laboratory (Section 8.7)
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

You may not qualify if…

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

Where it is running

Research Site — São Paulo, Brazil
Research Site — São Paulo, Brazil
Research Site — Calgary, Alberta, Canada
Research Site — Winnepeg, Manitoba, Canada
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
Research Site — Baltimore, Maryland, United States
Research Site — Kansas City, Missouri, United States
Research Site — Durham, North Carolina, United States

Full record on ClinicalTrials.gov

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