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

Study Identifier:
D8590C00002
ClinicalTrials.gov Identifier:
NCT06079281
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Recruitment Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: ALXN1850
  • Drug: Placebo
Date
Jan 2024 - Jul 2025
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 12 - 130 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Diagnosis of HPP documented in the medical records
  • Must meet 1 of the following criteria:
  • 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 )
  • 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:
  • Serum ALP activity below the age- and sex-adjusted normal range during the screening period as measured by the Central Laboratory
  • 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)
Sex
Female & Male
Age
12 - 130 Years

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: ALXN1850
  • Drug: Placebo
Date
Jan 2024 - Jul 2025
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 12 - 130 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Diagnosis of HPP documented in the medical records
  • Must meet 1 of the following criteria:
  • 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 )
  • 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:
  • Serum ALP activity below the age- and sex-adjusted normal range during the screening period as measured by the Central Laboratory
  • 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)

Protocol Summary

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.

Trial Locations

Location
Status
Location
Research Site
Indianapolis, Indiana, United States, 46202
Status
N/A
Location
Research Site
Garden City, New York, United States, 11530
Status
N/A
Location
Research Site
Durham, North Carolina, United States, 27705
Status
N/A
Location
Research Site
Columbus, Ohio, United States, 43203
Status
N/A
Location
Research Site
Nashville, Tennessee, United States, 37212
Status
N/A
Location
Research Site
Ciudad de Buenos Aires, Argentina, C1199
Status
N/A
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