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

N/AActive Not RecruitingNCT04982393

Alnylam Pharmaceuticals207 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

207

Conditions

Primary Hyperoxaluria Type 1

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: * Documented diagnosis of PH1, per physician's determination Exclusion Criteria: * Currently enrolled in a clinical trial for any investigational agent

Locations (30)

Clinical Trial Site

Phoenix, Arizona, United States

Outcomes

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

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

Data from ClinicalTrials.gov

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