Clinical trial NCT04982393: BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
Official title: BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Primary Hyperoxaluria Type 1
Keywords: PH1
Lumasiran
Primary Hyperoxaluria Type 1
Lead sponsor: Alnylam Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2021-07-29
Enrollment: 200
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients diagnosed with PH1 will be managed and treated per routine clinical practice.

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.


Eligibility criteria: Inclusion Criteria:

* Documented diagnosis of PH1, per physician's determination

Exclusion Criteria:

* Currently enrolled in a clinical trial for any investigational agent

Primary outcomes:
measure: Incidence of Adverse Events in Lumasiran Treated Patients
timeFrame: Up to 7 years
Secondary outcomes:
measure: Incidence of Selected Events of Interest in PH1 Patients
timeFrame: Up to 7 years
measure: 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version)
timeFrame: Up to 7 years
measure: Change in Urinary Oxalate Excretion
timeFrame: Baseline and every 12 months for up to 7 years
measure: Change in Plasma Oxalate
timeFrame: Baseline and every 12 months for up to 7 years
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Clinical Trial Site
state: Arizona
city: Washington
country: United States
facility: Clinical Trial Site
state: District of Columbia
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Rochester
country: United States
facility: Clinical Trial Site
state: Minnesota
city: Cincinnati
country: United States
facility: Clinical Trial Site
state: Ohio
city: Pittsburgh
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Dallas
country: United States
facility: Clinical Trial Site
state: Texas
city: Houston
country: United States
facility: Clinical Trial Site
state: Texas
city: Gent
country: Belgium
facility: Clinical Trial Site
city: Liège
country: Belgium
facility: Clinical Trial Site
city: Hamilton
country: Canada
facility: Clinical Trial Site
state: Ontario
city: Toronto
country: Canada
facility: Clinical Trial Site
state: Ontario
city: Laurier
country: Canada
facility: Clinical Trial Site
state: Quebec
city: Bordeaux
country: France
facility: Clinical Trial Site
city: Lyon
country: France
facility: Clinical Trial Site
city: Paris
country: France
facility: Clinical Trial Site
city: Berlin
country: Germany
facility: Clinical Trial Site
city: Cologne
country: Germany
facility: Clinical Trial Site
city: Hamburg
country: Germany
facility: Clinical Trial Site
city: Jerusalem
country: Israel
facility: Clinical Trial Site
city: Orbassano
country: Italy
facility: Clinical Trial Site
state: Torino
city: Milan
country: Italy
facility: Clinical Trial Site
city: Verona
country: Italy
facility: Clinical Trial Site
city: Amsterdam
country: Netherlands
facility: Clinical Trial Site
city: Santa Cruz de Tenerife
country: Spain
facility: Clinical Trial Site
state: Canary Islands
city: Barcelona
country: Spain
facility: Clinical Trial Site
city: Las Palmas
country: Spain
facility: Clinical Trial Site
city: Bern
country: Switzerland
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
state: England
city: London
country: United Kingdom
facility: Clinical Trial Site
Study officials:
affiliation: Alnylam Pharmaceuticals
name: Medical Director
role: STUDY_DIRECTOR
References: