Clinical Trials List
Protocol NumberALXN1850-HPP-305
NCT Number(ClinicalTrials.gov Identfier)NCT06079359
Active
2023-12-01 - 2028-06-30
Phase III
Not yet recruiting1
Terminated1
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 Versus Placebo Administered Subcutaneously in Pediatric (2 to < 12 years of age) Participants with Hypophosphatasia Who Have Not Received Previous Treatment with Asfotase Alfa
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Sponsor
ALEXION PHARMA TAIWAN LTD.
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Trial scale
Multi-Regional Multi-Center
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Update
2025/12/01
Investigators and Locations
Co-Principal Investigator
- 徐瑞聲 Division of General Internal Medicine
- I-JUNG TSAI Division of Pediatrics
- Ta-Ching Chen Division of Ophthalmology
- 謝正宜 Division of Rehabilitation Medicine
- 陳薈安 Division of Pediatrics
- Yin-Hsiu Chien Division of General Internal Medicine
- WUH-LIANG HWU Division of Pediatrics
- 彭信逢 Division of Radiology
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Principal Investigator
Ru-Li Lin
Co-Principal Investigator
- 蔡安黎 Division of Pediatrics
- Geng-Guo Lin Division of Ophthalmology
- 洪國烜 Division of Ophthalmology
- Min-Hua Tseng Division of Pediatrics
The Actual Total Number of Participants Enrolled
0 Terminated
Condition/Disease
Hypophosphatasia
Objectives
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.
Test Drug
ALXN1850
Active Ingredient
ALXN1850
Dosage Form
subcutaneous injection
Dosage
100 mg/ml
Endpoints
Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)
Inclution Criteria
Inclusion Criteria:
• Diagnosis of HPP documented in the medical records, and the following criteria fulfilled without other probable cause than HPP:
1. Presence of HPP-related rickets on skeletal X-rays during the Screening Period, with a minimum Rickets Severity Score (RSS) of 1.0 AND
2. Serum ALP activity below the age- and sex-adjusted normal range during the Screening Period as measured by the Central Laboratory OR 2 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 Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval.
• Must meet 1 of the following criteria:
1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a CLIA 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)
• Tanner stage 2 or less during the Screening Period
• Diagnosis of HPP documented in the medical records, and the following criteria fulfilled without other probable cause than HPP:
1. Presence of HPP-related rickets on skeletal X-rays during the Screening Period, with a minimum Rickets Severity Score (RSS) of 1.0 AND
2. Serum ALP activity below the age- and sex-adjusted normal range during the Screening Period as measured by the Central Laboratory OR 2 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 Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval.
• Must meet 1 of the following criteria:
1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a CLIA 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)
• Tanner stage 2 or less during the Screening Period
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
• 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
The Estimated Number of Participants
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Taiwan
4 participants
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Global
30 participants
