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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
30
Research Site
Baltimore, Maryland, United States
Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)
Time frame: Day 169
Change from Baseline in Rickets Severity Score (RSS) at the end of the Randomized Evaluation Period (Day 169)
Time frame: Baseline, Day 169
Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169)
Time frame: Baseline, Day 169
Change from Baseline in Percent Predicted 6MWT at the end of the Randomized Evaluation Period (Day 169)
Time frame: Baseline, Day 169
Change from Baseline in Bruininks Oseretsky Test of Motor Proficiency, Second Edition (BOT2) Score at the end of the Randomized Evaluation Period (Day 169)
Time frame: Baseline, Day 169
Change from Baseline in Peabody Developmental Motor Scales, Third Edition (PDMS-3) Score at the end of the Randomized Evaluation Period (Day 169)
Time frame: Baseline, Day 169
RGI-C Responder at the end of the Randomized Evaluation Period (Day 169)
Time frame: Day 169
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Research Site
Kansas City, Missouri, United States
Research Site
Durham, North Carolina, United States
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
...and 22 more locations