Patient prognosis evaluation with retrospective data in severe perinatal, benign perinatal or infantile HPP: A pooled analysis from the post-marketing study and perinatal findings surveillance

Patient prognosis evaluation with retrospective data in severe perinatal, benign perinatal or infantile HPP: A pooled analysis from the post-marketing study and perinatal findings surveillance - HPP-PERINATAL study

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-jRCT1050220128

Enrollment

Registered

2022-12-08

Start date

2022-12-26

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

severe perinatal, benign perinatal or infantile HPP HPP

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. At the time of the PMS data lock in July 2021, Strensiq Specified drug use-results survey (post-marketing survey) Patients should have have provided data to PMS prior the data base lock date above. 2. Patients diagnosed with perinatal severe hypophosphatasia, perinatal benign hypophosphatasia, or infantile hypophosphatasia 3. A patient whose legal representative or patient can give written consent to provide patient information and a patient whose mother can give written consent to provide maternal information.

Exclusion criteria

Exclusion criteria: 1. Patients diagnosed with non-perinatal and non-infantile hypophosphatasia 2. Patients in whom fetal ultrasound image data including the number of weeks of gestation before and after 28 ~ 31 weeks of gestation cannot be obtained

Design outcomes

Primary

Measure	Time frame
-To verify the relationship between fetal ultrasound findings and postnatal motor development data by HPP disease type (Severe perinatal, benign perinatal, and infantile) and to perform an exploratory evaluation for the presence of ultrasound findings that affect motor development in patients. -To verify the relationship between fetal ultrasound findings and postnatal respiratory status data by HPP disease type (Severe perinatal, benign perinatal, and infantile) and to perform an exploratory evaluation for ultrasound imaging data that affect respiratory status. -Describe what are the baseline characteristics of perinatal/infantile HPP at the time of the abnormality detection from ultrasound imaging data -Asses what are the identifiable predictors of prognosis of HPP from ultrasound imaging data	—

Measure

Time frame

-To verify the relationship between fetal ultrasound findings and postnatal motor development data by HPP disease type (Severe perinatal, benign perinatal, and infantile) and to perform an exploratory evaluation for the presence of ultrasound findings that affect motor development in patients. -To verify the relationship between fetal ultrasound findings and postnatal respiratory status data by HPP disease type (Severe perinatal, benign perinatal, and infantile) and to perform an exploratory evaluation for ultrasound imaging data that affect respiratory status. -Describe what are the baseline characteristics of perinatal/infantile HPP at the time of the abnormality detection from ultrasound imaging data -Asses what are the identifiable predictors of prognosis of HPP from ultrasound imaging data

—

Secondary

Measure	Time frame
-To verify the relationship between fetal ultrasound imaging findings and patient disease types and to evaluate the predictability of HPP disease types after birth based on fetal ultrasound imaging findings. -To compare fetal ultrasound findings with the presence or absence of postnatal skeletal lesions and assess these associations descriptively. -To explore the therapeutic effect of asfotase alfa by HPP subtypes and assess these associations descriptively. -Asses if there is a correlation between clinical symptoms in the fetal period and post-natal motor function development grouped by the type of HPP -Assess prenatal and postnatal information on patients diagnosed with perinatal or infantile hypophosphatasia with or without enzyme replacement therapy and to examine the impact of prenatal information on postnatal HPP type, skeletal lesions, and asphotase alfa therapy	—

Measure

Time frame

-To verify the relationship between fetal ultrasound imaging findings and patient disease types and to evaluate the predictability of HPP disease types after birth based on fetal ultrasound imaging findings. -To compare fetal ultrasound findings with the presence or absence of postnatal skeletal lesions and assess these associations descriptively. -To explore the therapeutic effect of asfotase alfa by HPP subtypes and assess these associations descriptively. -Asses if there is a correlation between clinical symptoms in the fetal period and post-natal motor function development grouped by the type of HPP -Assess prenatal and postnatal information on patients diagnosed with perinatal or infantile hypophosphatasia with or without enzyme replacement therapy and to examine the impact of prenatal information on postnatal HPP type, skeletal lesions, and asphotase alfa therapy

—

Contacts

Public ContactTomoaki Sakaguchi

Linical Co., Ltd.

PERINATAL-study@linical.co.jp+81-3-6215-8005

Outcome results