An observational study in Primary Hyperoxaluria Type 1, sponsored by Alnylam Pharmaceuticals. Active, not recruiting at 30 sites in 11 countries. Per ClinicalTrials.gov, last updated 2026-10-06.
Sponsored by Alnylam Pharmaceuticals · Observational
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.
Alnylam Pharmaceuticals is the lead sponsor of 82 studies on the registry; 20 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 14 (82%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients diagnosed with PH1 will be managed and treated per routine clinical practice.
Exclusion Criteria:
Patients with a diagnosis of PH1 will be eligible for the study and will be managed and treated per routine clinical practice.
Incidence of Adverse Events in Lumasiran Treated Patients
Time frame: Up to 7 years
Incidence of Selected Events of Interest in PH1 Patients
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.
Time frame: Up to 7 years
12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version)
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.
Time frame: Up to 7 years
Change in Urinary Oxalate Excretion
Time frame: Baseline and every 12 months for up to 7 years
Change in Plasma Oxalate
Time frame: Baseline and every 12 months for up to 7 years
Plan to share: No
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Primary hyperoxaluria type 1
Alnylam Pharmaceuticals