BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Primary Hyperoxaluria Type 1

Sex eligibility

All sexes

Study type

Observational

Primary location

Clinical Trial Site, Ghent, Belgium

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Documented diagnosis of PH1, per physician's determination

Exclusion criteria

Currently enrolled in a clinical trial for any investigational agent

Treatment and study plan

Primary outcomes

Incidence of Adverse Events in Lumasiran Treated Patients

Time frame: Up to 7 years

Secondary outcomes

Incidence of Selected Events of Interest in PH1 Patients

Time frame: 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)

Time frame: 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

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

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Acronym: BONAPH1DE

Important dates

Study start: 2021
Primary completion: 2028
Study completion: 2028
First posted: Jul 29, 2021
Registry last updated: Apr 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Active, Not Recruiting