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Active (not recruiting) Phase 3 NCT06079281

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa

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

Sponsor: Alexion Pharmaceuticals, Inc.

trial.available_in: БГ
Overview
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.
Who can participate
Inclusion Criteria: * Diagnosis of HPP documented in the medical records * Must meet 1 of the following criteria: 1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 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) * Must meet 1 of the following criteria without a probably cause other than HPP: 1. Serum ALP activity below the age- and sex-adjusted normal range during the screening period as measured by the Central Laboratory 2. Two 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 CLIA or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval. * Two separate 6MWTs at below 85% of the predicted distance (for age, sex, weight, and height) during the Screening Period without a probable cause other than HPP 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 (Table 9)
Locations 17
Argentina (1)
Research Site
Ciudad de Buenos Aires
Australia (1)
Research Site
Clayton
Brazil (1)
Research Site
Belo Horizonte
Canada (1)
Research Site
Calgary , Alberta
China (1)
Research Site
Beijing
France (1)
Research Site
Paris
Germany (1)
Research Site
Bad Reichenhall
Israel (1)
Research Site
Ashkelon
Italy (1)
Research Site
Florence
Japan (1)
Research Site
Hiroshima
Poland (1)
Research Site
Lodz
South Korea (1)
Research Site
Seoul
Spain (1)
Research Site
Barcelona
Taiwan (1)
Research Site
Taipei
Turkey (1)
Research Site
Ankara
United Kingdom (1)
Research Site
London
United States (1)
Research Site
Indianapolis , Indiana
Technical details
Status
Active (not recruiting)
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male and female
Minimum age
12 Years
Maximum age
130 Years
Healthy volunteers
No
Start date
03.01.2024
Completion date
29.03.2028
Registry ID
NCT06079281
Source
anzctr
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