Primary Hyperoxaluria Type 1
Conditions
Keywords
PH1, Lumasiran, Primary Hyperoxaluria Type 1
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.
Interventions
None listed
Sponsors
Alnylam Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Documented diagnosis of PH1, per physician's determination
Exclusion criteria
* Currently enrolled in a clinical trial for any investigational agent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of Adverse Events in Lumasiran Treated Patients | Up to 7 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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 | — |
Countries
Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom, United States
Contacts
STUDY_DIRECTORMedical Director
Alnylam Pharmaceuticals
Outcome results
None listed