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Clarify HPP: Disease Burden of HPP Patients in China

Recruitingn/aAges any - anyAllAstraZeneca
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In plain words

What the trial is, and whether it could suit you

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.

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๐Ÿ“‹ Eligibility rules (as published)
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 โ‰ฅ14 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.
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What it tests

  • Not listed
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Where

22 sites
Research Site
Beijing, China
Research Site
Beijing, China
Research Site
Changsha, China
Research Site
Chengdu, China
Research Site
Chongqing, China
Research Site
Fuzhou, China
Research Site
Guangzhou, China
Research Site
Hangzhou, China

โ€ฆand 14 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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