The primary objective of this study is to assess the efficacy of ALXN1850 versus placebo on functional outcomes in adolescent and adult participants with HPP who have not previously been treated with asfotase alfa.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
124
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 30-second Sit to Stand (STS) Test Score at the end of the Randomized Evaluation Period (Day 169)
Time frame: Baseline, Day 169
Change from Baseline in Lower Extremity Functional Scale (LEFS) Score at the end of the Randomized Evaluation Period (Day 169)
Time frame: Baseline, Day 169
Change from Baseline in BPI-SF pain severity score at the end of the Randomized Evaluation Period (Day 169) (adult cohort only)
Time frame: Baseline, Day 169
Change from Baseline in FACIT-Fatigue score at the end of the Randomized Evaluation Period (Day 169) (adult cohort only)
Time frame: Baseline, Day 169
Change from Baseline in Timed Up-and-Go (TUG) 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
Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)
Time frame: 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
Indianapolis, Indiana, United States
Research Site
Garden City, New York, United States
Research Site
Durham, North Carolina, United States
Research Site
Columbus, Ohio, United States
Research Site
Nashville, Tennessee, United States
Research Site
Ciudad de Buenos Aires, Argentina
Research Site
Clayton, Australia
Research Site
Herston, Australia
Research Site
Parkville, Australia
Research Site
St Leonards, Australia
...and 55 more locations
Change from Baseline in Rickets Severity Score (RSS) at the end of the Randomized Evaluation Period (Day 169)
Time frame: Baseline, Day 169