MedPath

Phase 3 Study of ALXN1850 in Pediatric Participants with HPP Previously Treated with Asfotase Alfa

Phase 3
Conditions
Hypophosphatasia
Registration Number
JPRN-jRCT2051230131
Lead Sponsor
Wuebbenhorst Hiroaki
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Pending
Sex
All
Target Recruitment
4
Inclusion Criteria

1. Participant must be 2 or more and < 12 years of age at Day 1.
2. Diagnosis of HPP documented in the medical records
3. Presence of open growth plates by X-ray during Screening Period
4. Tanner stage 2 or less during the Screening Period
5. Must have been treated with 6 mg/kg/ week of asfotase alfa via SC injection administered as either 2mg/kg 3 times per week or 1 mg/kg 6 times per week for more than 6 months before Day 1

Exclusion Criteria

1. 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.
2. Diagnosis of primary or secondary hyperparathyroidism
3. Hypoparathyroidism, unless secondary to HPP
4. Any new fracture within 12 weeks before Day 1 (excluding pseudofractures)
5. Planned surgical intervention which may impact the results of study assessments (in the opinion of the Investigator) during the Randomized Evaluation Period
6. History of allergy or hypersensitivity to any ingredient contained in asfotase alfa or ALXN1850
7. Body weight < 10 kg during the Screening Period

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
umber of Participants with Treatment-emergent Adverse Events (TEAEs)
Secondary Outcome Measures
NameTimeMethod
© Copyright 2025. All Rights Reserved by MedPath