Understanding the disease burden of hypophosphatasia in Chinese patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a prospective, multi-center observational study enrolling Chinese patients with hypophosphatasia to document the disease burden. Data will be collected from medical records, patient-reported outcomes, functional assessments, radiographs, and healthcare costs using an electronic data capture system across approximately 30 sites. The aim is to describe quality of life, clinical manifestations, and patient-reported outcomes in real-world clinical practice. Participation requires confirmed HPP diagnosis and informed consent; gene testing or alternative markers may be used as part of diagnostic criteria. No interventions are being tested; the study observes existing care and outcomes over time.

Why it matters

Who can join: the full rules

Inclusion Criteria:

Participants are eligible to be included in the study only if all the following criteria apply:

1. 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.
2. 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.

Exclusion Criteria:

Participants will be excluded from the study if they meet any of the following criteria:

1. Prior or current use of first-generation asfotase alfa at any time before study enrollment.
2. Participation in any other interventional clinical trial for HPP or other metabolic diseases within 3 months prior to study enrollment.
3. 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.
4. Inability to comply with study follow-up procedures (e.g., geographic inaccessibility, cognitive impairment) as assessed by the treating physician.

Where it's running

• Research Site
  Beijing, China Recruiting
• Research Site
  Beijing, China Not yet recruiting
• Research Site
  Changsha, China Not yet recruiting
• Research Site
  Chengdu, China Not yet recruiting
• Research Site
  Chongqing, China Not yet recruiting
• Research Site
  Fuzhou, China Not yet recruiting

Study contacts

• Principal investigator: Weibo Xia, MD — Peking Union Medical College Hospital
• Study coordinator: AstraZeneca Clinical Study Information Center
• Email: information.center@astrazeneca.com
• Phone: 1-877-240-9479

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.