Hypophosphatasia (HPP)NCT07905287
Clarify HPP: Disease Burden of HPP Patients in China
An observational study of Hypophosphatasia (HPP), run by AstraZeneca at 22 sites in China.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hypophosphatasia (HPP)
- How many people
- 200 participants
- Where
- 22 sitesChina
- Study dates
- 26 Sep 2026to 31 Oct 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 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).
Who may be able to join
- 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:
- At least 1 skeletal or non-skeletal manifestation consistent with HPP;
- Persistently low age- and sex-adjusted ALP: at least two independent tests with an interval of ≥14 days, both results below the local laboratory's age-/sex-specific reference lower limit;
- Positive ALPL gene test result (pathogenic or likely pathogenic variant, or variant of uncertain significance [VUS]) - gene testing will be provided by the study if not already available for the participant; OR Elevated pyridoxal 5'-phosphate (PLP) level, if this test is available at the clinic.
- Participant or parent (or legal guardian) must provide written informed consent prior to the performance of any study-related procedures and must be willing to comply with study procedures.
Who cannot join
- Participants will be excluded from the study if they meet any of the following criteria:
- Prior or current use of first-generation asfotase alfa at any time before study enrollment.
- Participation in any other interventional clinical trial for HPP or other metabolic diseases within 3 months prior to study enrollment.
- Severe comorbid conditions (e.g., end-stage organ failure, advanced malignancy) judged by the treating physician to interfere with the collection of valid study data or participant safety during follow-up.
- Inability to comply with study follow-up procedures (e.g., geographic inaccessibility, cognitive impairment) as assessed by the treating physician.
Trial locations and contacts
22 sites in 1 country. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center contact
22 sites in 1 country
China22 sites, 22 recruiting
- Recruiting
Research Site
Beijing
- Not yet recruiting
Research Site
Beijing
- Not yet recruiting
Research Site
Changsha
- Not yet recruiting
Research Site
Chengdu
- Not yet recruiting
Research Site
Chongqing
- Not yet recruiting
Research Site
Fuzhou
- Not yet recruiting
Research Site
Guangzhou
- Not yet recruiting
Research Site
Hangzhou
- Not yet recruiting
Research Site
Huai'an
- Not yet recruiting
Research Site
Jinan
- Not yet recruiting
Research Site
Nanchang
- Not yet recruiting
Research Site
Qingdao
- Not yet recruiting
Research Site
Shanghai, 200233
021-64369181
- Not yet recruiting
Research Site
Shanghai, 201102
021-64931990
- Not yet recruiting
Research Site
Shanghai
- Not yet recruiting
Research Site
Shenyang
- Not yet recruiting
Research Site
Shenzhen
- Not yet recruiting
Research Site
Suzhou
- Not yet recruiting
Research Site
Tianjin
- Not yet recruiting
Research Site
Wuhan
- Not yet recruiting
Research Site
Xi'an
- Not yet recruiting
Research Site
Zhengzhou
About this study
Official title: A Multi-Center, Prospective, Observational Study of the Disease Burden of Hypophosphatasia (HPP) Patients in China
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.
Read the detailed description
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.
- Conditions
- Hypophosphatasia (HPP)
- Medical subject headings
- Hypophosphatasia
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: Baseline, Month 3, Month 6, Month 9, and Month 12
Secondary outcomes (5)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline, Month 6, and Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2026
- Study start26 Sep 2026
- Primary completion31 Oct 2028 (estimated)
- Study completion31 Oct 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypophosphatasia (HPP)Studies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Weibo Xia, MD, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07905287
- Sponsor's protocol ID
- D8590R00003
- Status last verified
- Oct 2026
- First submitted
- 7 Sep 2026
- Last updated
- 29 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, Prospective, Observational Study of the Disease Burden of Hypophosphatasia (HPP) Patients in China
- ClinicalTrials.gov ID
- NCT07905287
- Acronym / sponsor ID
- D8590R00003
- Conditions
- Hypophosphatasia (HPP)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 26 Sep 2026 (actual)
- Primary completion
- 31 Oct 2028 (estimated)
- Study completion
- 31 Oct 2028 (estimated)
- First posted
- 29 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 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).
Key inclusion criteria (6 listed)
- 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:
- Participant or parent (or legal guardian) must provide written informed consent prior to the performance of any study-related procedures and must be willing to comply with study procedures.
Key exclusion criteria (5 listed)
- Prior or current use of first-generation asfotase alfa at any time before study enrollment.
- Participation in any other interventional clinical trial for HPP or other metabolic diseases within 3 months prior to study enrollment.
- Severe comorbid conditions (e.g., end-stage organ failure, advanced malignancy) judged by the treating physician to interfere with the collection of valid study data or participant safety during follow-up.
- Inability to comply with study follow-up procedures (e.g., geographic inaccessibility, cognitive impairment) as assessed by the treating physician.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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).
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
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.
5-18 years pediatric participants: SF-10 absolute values at baseline, 3, 6, 9, 12 months (including PCS, MCS)
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
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.
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)
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
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.
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).
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
To evaluate the quality of life in Chinese pediatric patients aged 13-24 months with HPP using PedsQL Infant Scales (ages 13-24 months).
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)
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
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.
5 secondary outcomes
Adult participants: BPI-SF absolute values at baseline, 3, 6, 9, 12 months (total pain severity score and total pain interference 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)
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
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
Time frame: Baseline, Month 3, Month 6, Month 9, and Month 12
30 seconds STS absolute values at baseline, 6 and 12 months
Time frame: Baseline, Month 6, and Month 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
AstraZeneca Clinical Study Information Center contact
Weibo Xia, MD principal investigator
Peking Union Medical College Hospital
22 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hypophosphatasia (HPP), run by AstraZeneca at 22 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact AstraZeneca Clinical Study Information Center (1-877-240-9479, information.center@astrazeneca.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). (time frame: Baseline, Month 3, Month 6, Month 9, and Month 12). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2026.