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Active (not recruiting) Phase 3 NCT06079359

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

Phase 3 – large-scale trial before approval
Conditions: Hypophosphatasia

Sponsor: Alexion Pharmaceuticals, Inc.

trial.available_in: БГ
Overview
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.
Who can participate
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
Locations 16
Australia (1)
Research Site
Nedlands
Belgium (1)
Research Site
Brussels
Brazil (1)
Research Site
Brasília
Canada (1)
Research Site
Calgary , Alberta
China (1)
Research Site
Beijing
Finland (1)
Research Site
Helsinki
Israel (1)
Research Site
Ashkelon
Mexico (1)
Research Site
Chihuahua City
Poland (1)
Research Site
Lodz
Romania (1)
Research Site
Bucharest
Spain (1)
Research Site
Madrid
Sweden (1)
Research Site
Stockholm
Taiwan (1)
Research Site
Taipei
Turkey (1)
Research Site
Ankara
United Kingdom (1)
Research Site
Birmingham
United States (1)
Research Site
Baltimore , Maryland
Technical details
Status
Active (not recruiting)
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male and female
Minimum age
2 Years
Maximum age
11 Years
Healthy volunteers
No
Start date
14.05.2024
Completion date
24.08.2028
Registry ID
NCT06079359
Source
anzctr
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