BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
- Conditions
- Primary Hyperoxaluria Type 1
- Registration Number
- NCT04982393
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
The purpose of this study is to describe the natural history and progression of patients diagnosed with PH1, and to characterize the long-term real-world safety and effectiveness of lumasiran.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 200
- Documented diagnosis of PH1, per physician's determination
- Currently enrolled in a clinical trial for any investigational agent
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Incidence of Adverse Events in Lumasiran Treated Patients Up to 7 years
- Secondary Outcome Measures
Name Time Method 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version) Up to 7 years SF-12 V2 is a 12-question measure capturing global quality of life and overall health status and evaluates the following 8 domains: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health.
Incidence of Selected Events of Interest in PH1 Patients Up to 7 years Selected events of interest are defined as hepatic events, kidney stones, acute kidney injury events, nephrocalcinosis, chromic kidney disease, kidney failure, and any cardiac, bone, skin, eye, hematological, or neuropathic manifestations due to oxalosis.
Change in Urinary Oxalate Excretion Baseline and every 12 months for up to 7 years Change in Plasma Oxalate Baseline and every 12 months for up to 7 years
Trial Locations
- Locations (1)
Clinical Trial Site
🇬🇧London, United Kingdom