Hypophosphatasia - Activity Trackingin a Rare, Inherited Musculoskeletal Condition(HATRIC)
- Conditions
- E83.38
- Registration Number
- DRKS00032056
- Lead Sponsor
- Klinische Studieneinheit König Ludwig Haus
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 21
Confirmed clinical diagnosis of HPP based on one of the following:
- ALP activity (age- and gender-adjusted) below lower limit of normal, measured at least two times within a minimum four-week interval
- Genetic confirmation of the disease by documented variant within the ALPL Gene
- Experience HPP-associated functional deficits requiring professional treatment (ie. Pain medication, physiotherapy)
- Elevated substrates above upper limit of normal for PLP and/or urinary PEA
- Symptoms of the disease
- Inability to walk, or require the use of assistive devices for walking
- Regular use of a wheelchair even for distances < 50m
- Inability / unwillingness to comply with repeated testing requirements and wearing the
activity tracking device
- Currently enrolled in another clinical trial
- Patient on enzyme replacement therapy with asfotase alfa
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To evaluate the feasibility of and the adherence to activity tracking by utilizing<br>a wearable tracking device in patients with hypophosphatasia. Data will be collected via activity tracker which will be worn 3x2 weeks.
- Secondary Outcome Measures
Name Time Method To assess the validity and reliability of activity tracking using a wearable device in patients with<br>hypophosphatasia.