An observational study in Hypophosphatasia (HPP), sponsored by AstraZeneca. Recruiting at 22 sites in China. Per ClinicalTrials.gov, last updated 2026-10-29.
Sponsored by AstraZeneca · Observational
A multi-Center, prospective, observational study of the disease burden of Hypophosphatasia (HPP) patients in China. The study aims to prospectively collect real-world outcome data on HPP, including QoL, clinical manifestations, patient-reported outcomes (PROs) among Chinese HPP patients.
A multi-Center, prospective, observational study of the disease burden of Hypophosphatasia (HPP) patients in China. The study aims to prospectively collect real-world outcome data on HPP, including QoL, clinical manifestations, patient-reported outcomes (PROs) among Chinese HPP patients. Primary data collection via electronic data capture (EDC) system is expected to be conducted across 30 clinical sites in China, including patient medical records, patient-reported outcomes, functional assessments, radiographic examinations and healthcare cost data.
46 studies on the registry are indexed under Hypophosphatasia; 11 are open to participants now.
This study's planned enrollment of 200 is above the median of 58 across 24 observational studies indexed under Hypophosphatasia.
Browse Hypophosphatasia studies →AstraZeneca is the lead sponsor of 3,499 studies on the registry; 270 are open to participants now.
Of its 368 completed or terminated interventional studies of FDA-regulated products, 178 (48%) have results posted.
Counted across the registry records on this site, refreshed daily.
Participants with confirmed HPP diagnosis meeting inclusion criteria (at least one HPP-consistent clinical manifestation, persistently low ALP, positive ALPL gene test or elevated PLP) and no exclusion criteria (prior asfotase alfa use, recent interventional trial participation, severe comorbidities, poor compliance to study procedure).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participants are eligible to be included in the study only if all the following criteria apply:
Confirmed diagnosis of HPP, defined by meeting all the following:
Exclusion Criteria:
Participants will be excluded from the study if they meet any of the following criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Adults: SF-36 V2.0 absolute values at baseline and months 3, 6, 9 and 12, including Physical and Mental Component Summary scores and 8 domains (physical functioning/role, pain, general health, vitality, social functioning/role, mental health).
To evaluate the quality of life in Chinese adult patients with HPP using SF-36 V2.0. Raw scores of the 8 domains and the two summary scores are converted to a standardized 0-100 scale, with higher scores indicating better overall physical and mental quality of life.
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
5-18 years pediatric participants: SF-10 absolute values at baseline, 3, 6, 9, 12 months (including PCS, MCS)
To evaluate the quality of life in Chinese pediatric patients aged 5-18 years with HPP using SF-10. It assesses core physical and mental health dimensions, with results summarized into a continuous total score (0-100 scale) reflecting overall physical and mental health, higher total scores correspond to better pediatric health-related quality of life.
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
2-4 years pediatric participants: PedsQL 4.0 Toddler Version absolute values at baseline, 3, 6, 9, 12 months (total score and 4 domain summary scores including physical functioning, emotional functioning, social functioning, school functioning)
To evaluate the quality of life in Chinese pediatric patients aged 2-4 years with HPP using PedsQL 4.0 Toddler Version. All items are completed on a 5-point Likert scale, and scores are transformed to a 0-100 scale where higher scores represent better health-related quality of life.
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
Pediatric participants aged 13-24 months: PedsQL Infant Scales absolute values at baseline and months 3, 6, 9 and 12, including total score and 5 domains (physical functioning/symptoms, emotional, social and cognitive functioning).
To evaluate the quality of life in Chinese pediatric patients aged 13-24 months with HPP using PedsQL Infant Scales (ages 13-24 months).
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
1-12 months pediatric participants: PedsQL Infant Scales ages 1-12 months (total score and 5 domain summary scores including physical functioning, physical symptoms, emotional functioning, social functioning, cognitive functioning)
To evaluate the quality of life in Chinese pediatric patients aged 1-12 months with HPP using PedsQL Infant Scales (ages 1-12 months). All items are completed on a 5-point Likert scale, and scores are transformed to a 0-100 scale where higher scores represent better health-related quality of life.
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
Adult participants: BPI-SF absolute values at baseline, 3, 6, 9, 12 months (total pain severity score and total pain interference score)
To assess the severity of pain in Chinese adult HPP patients using BPI-SF. BPI-SF includes 4 items that assess pain severity (11-point scale: 0 = no pain, 10 = worst pain you can imagine) and 7 items that assess pain interference with functioning (11- point scale: 0 = does not interfere, 10 = completely interferes) in the preceding 24 hours. A total pain severity score (BPI-SF-PS; range: 0-40) is calculated from the 4 items of pain intensity, with each item weighted equally in the final score. A total pain interference score (BPI-SF-PI; range: 0-70) is calculated from the 7 items on pain interference, each contributing the same weight to the final score.
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
Pediatric participants: PODCI Pain/Comfort subscale absolute values at baseline, 3, 6, 9, 12 months (PODCI Pain/Comfort score)
To assess the severity of pain in Chinese pediatric HPP patients using PODCI Pain/Comfort subscale. The PODCI includes functional subscales, in this study, only the Pain/Comfort subscale will be used. Standardized scores for the Pain/Comfort subscale range from 0 to 100, with higher scores indicating less pain and better comfort/health status.
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
Adult participants: FACIT-Fatigue absolute values at baseline, 3, 6, 9, 12 months
To evaluate the level of fatigue in Chinese adult HPP patients using FACIT-Fatigue. The FACIT-Fatigue comprises 13 items, each rated on a 5-point Likert scale from 0 (not at all) to 4 (very much). Higher scores indicate less severe fatigue and better functional status.
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
Pediatric participants (8-18 years): Peds FACIT-Fatigue absolute values at baseline, 3, 6, 9, 12 months
To evaluate the level of fatigue in Chinese pediatric HPP patients using Peds FACIT-Fatigue. Scores will be calculated in accordance with the applicable Peds FACIT-Fatigue scoring manual, with higher scores indicating less fatigue and better functioning.
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
30 seconds STS absolute values at baseline, 6 and 12 months
To assess the functional status of Chinese HPP patients using 30 seconds STS. The test result is recorded as the number of complete sit-to-stand repetitions completed within 30 seconds, more repetitions indicate better lower limb muscle strength, endurance, and functional mobility.
Time frame: Baseline, Month 6, and Month 12
Plan to share: Yes — Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Time frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Access criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
Data sharing details ↗ · vivli.org
No publications or documents are linked to this record.
Started Sep 2026 (actual). Primary completion is estimated for Oct 2028, 25 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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