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Hypophosphatasia clinical trials

View clinical trials related to Hypophosphatasia.

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NCT ID: NCT04222452 Active, not recruiting - Hypophosphatasia Clinical Trials

The PORTRAIT Study

PORTRAIT
Start date: July 12, 2021
Phase:
Study type: Observational

Clinical Consequences of Adults Presenting with Hypophosphatasia with Special Focus on Gait, Bone Microstructure and Cognition: The PORTRAIT study Hypophosphatasia (HPP) is an inherited condition that leads to weak bones. Early childhood forms are severe and easily recognized. Adult forms can vary in severity. HPP is often missed by doctors or confused with osteoporosis. This is important because the usual osteoporosis treatments may be harmful to patients with HPP and increase the risk of broken bones. One of the reasons it is missed is a lack of research describing the typical features of HPP, so doctors don't recognize the signs, and don't know when or how to test for it. The PORTRAIT Study will help increase understanding of the burden of disease of HPP on patients. The aim is to examine the effects of HPP on bone structure and strength, physical functioning, cognition, and quality of life. Researchers will study adults with HPP and healthy age- and gender-matched individuals. Blood samples will be collected after an overnight fast. Researchers will use these samples to measure markers of HPP and bone health. Medical history and lifestyle, quality of life and cognitive function will be assessed using questionnaires. Bone mineral density, body composition and bone structure and strength will be measured using dual energy x-ray absorptiometry and high resolution peripheral quantitative computed tomography. Physical functioning will be assessed as participants perform a series of physical performance and gait tests. Magnetic resonance images of the lower limbs will be matched-up with the physical functioning data to create patient-specific musculoskeletal models. Cognitive function tests will be performed to assess cognition and mental health. To reveal the burden of disease of HPP, the data collected from patients with HPP will be compared to that collected from healthy controls.

NCT ID: NCT04195763 Active, not recruiting - Hypophosphatasia Clinical Trials

Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)

Start date: November 6, 2019
Phase:
Study type: Observational

This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support program managed by OneSourceā„¢.

NCT ID: NCT04181164 Active, not recruiting - Clinical trials for Hypophosphatasia (HPP)

Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)

BABS
Start date: October 1, 2019
Phase:
Study type: Observational

The study aims to evaluate the bone architecture and bone strength in adults with Hypophosphatasia (HPP).

NCT ID: NCT04018287 Active, not recruiting - Hypophosphatasia Clinical Trials

Circulating miRNAs and Bone Microstructure in Adults With Hypophosphatasia

Start date: August 1, 2017
Phase:
Study type: Observational

The aim of the study is to accomplish a complete bone status of patients with HPP using new approaches to assess bone quality.