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

Primary Hyperoxaluria Type 1 Clinical Trial

— BONAPH1DE  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04982393
• Other study ID #: ALN-GO1-007
• Status: Recruiting
• Start date: December 13, 2021
• Est. completion date: September 2028

Study information

• Verified date: May 2024
• Source: Alnylam Pharmaceuticals
• Contact: Alnylam Clinical Trial Information Line
• Phone: 1-877-ALNYLAM
• Email: clinicaltrials[@]alnylam.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: September 2028
• Est. primary completion date: September 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Documented diagnosis of PH1, per physician's determination

Exclusion Criteria:

• Currently enrolled in a clinical trial for any investigational agent

Related Conditions & MeSH terms

• Hyperoxaluria, Primary
• Primary Hyperoxaluria Type 1

Locations

Country	Name	City	State
Belgium	Clinical Trial Site	Gent
Belgium	Clinical Trial Site	Liège
Canada	Clinical Trial Site	Hamilton	Ontario
Canada	Clinical Trial Site	Laurier	Quebec
Canada	Clinical Trial Site	Toronto	Ontario
France	Clinical Trial Site	Bordeaux
France	Clinical Trial Site	Lyon
France	Clinical Trial Site	Paris
Germany	Clinical Trial Site	Berlin
Germany	Clinical Trial Site	Cologne
Germany	Clinical Trial Site	Hamburg
Israel	Clinical Trial Site	Jerusalem
Italy	Clinical Trial Site	Orbassano	Torino
Spain	Clinical Trial Site	Barcelona
Switzerland	Clinical Trial Site	Bern
United Kingdom	Clinical Trial Site	London	England
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Cincinnati	Ohio
United States	Clinical Trial Site	Dallas	Texas
United States	Clinical Trial Site	Houston	Texas
United States	Clinical Trial Site	Phoenix	Arizona
United States	Clinical Trial Site	Pittsburgh	Pennsylvania
United States	Clinical Trial Site	Rochester	Minnesota
United States	Clinical Trial Site	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  France,  Germany,  Israel,  Italy,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Adverse Events in Lumasiran Treated Patients		Up to 7 years
Secondary	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.	Up to 7 years
Secondary	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.	Up to 7 years
Secondary	Change in Urinary Oxalate Excretion		Baseline and every 12 months for up to 7 years
Secondary	Change in Plasma Oxalate		Baseline and every 12 months for up to 7 years