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

• Status: Recruiting
• Trial end date: 2027-10-15
• Target enrollment: 0
• Participant gender: All

Summary

The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Criteria

Inclusion Criteria:

• Diagnosis of HPP documented in the medical records

• Presence of open growth plates by X-ray during Screening Period

• Tanner stage 2 or less during the Screening Period

• 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 ≥ 6 months before Day 1

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 asfotase alfa or ALXN1850

• Body weight < 10 kg during the Screening Period