Clinical Trials List
Protocol NumberALXN1850-HPP-301
NCT Number(ClinicalTrials.gov Identfier)NCT06079281
Active
2023-11-21 - 2028-06-30
Phase III
Recruiting2
ICD-10E83.30
Disorder of phosphorus metabolism, unspecified
ICD-10E83.31
Familial hypophosphatemia
ICD-10E83.32
Hereditary vitamin D-dependent rickets (type 1) (type 2)
ICD-10E83.39
Other disorders of phosphorus metabolism
ICD-9275.3
Disorders of phosphorus metabolism
A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 (Recombinant Alkaline Phosphatase) Administered Subcutaneously in Adolescent (12 to < 18 years of age) and Adult Participants with Hypophosphatasia Who Have Not Previously Been Treated with Asfotase Alfa
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Sponsor
ALEXION PHARMA TAIWAN LTD.
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Trial scale
Multi-Regional Multi-Center
-
Update
2026/03/01
Investigators and Locations
Co-Principal Investigator
- WUH-LIANG HWU Division of Pediatrics
- 彭信逢 無
- Ta-Ching Chen Division of Ophthalmology
- 謝正宜 無
- 徐瑞聲 Division of General Internal Medicine
- I-JUNG TSAI 無
- 陳薈安 無
- Yin-Hsiu Chien 無
The Actual Total Number of Participants Enrolled
0 Recruiting
Principal Investigator
Ru-Li Lin
Co-Principal Investigator
- Min-Hua Tseng Division of Pediatrics
- Geng-Guo Lin Division of Ophthalmology
- 劉顥筌 無
- 蔡安黎 Division of Pediatrics
- 洪國烜 Division of Ophthalmology
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Hypophosphatasia
Objectives
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.
Test Drug
ALXN1850
Active Ingredient
ALXN1850
Dosage Form
Subcutaneous injection
Dosage
MG
Endpoints
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.
Inclution Criteria
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
• 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
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)
• 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)
The Estimated Number of Participants
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Taiwan
4 participants
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Global
114 participants
