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)
Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169) — Day 169
Trial sites (32)
Facility
City
Region
Status
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 the UK
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.
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