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

May 9, 2024 updated by: Alnylam Pharmaceuticals
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 Overview

Status

Recruiting

Conditions

Study Type

Observational

Enrollment (Estimated)

200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Belgium
      • Gent, Belgium
        • Recruiting
        • Clinical Trial Site
      • Liège, Belgium
        • Recruiting
        • Clinical Trial Site
  • Canada
    • Ontario
      • Hamilton, Ontario, Canada
        • Recruiting
        • Clinical Trial Site
      • Toronto, Ontario, Canada
        • Not yet recruiting
        • Clinical Trial Site
    • Quebec
      • Laurier, Quebec, Canada
        • Recruiting
        • Clinical Trial Site
  • France
      • Bordeaux, France
        • Recruiting
        • Clinical Trial Site
      • Lyon, France
        • Recruiting
        • Clinical Trial Site
      • Paris, France
        • Recruiting
        • Clinical Trial Site
  • Germany
      • Berlin, Germany
        • Recruiting
        • Clinical Trial Site
      • Cologne, Germany
        • Recruiting
        • Clinical Trial Site
      • Hamburg, Germany
        • Recruiting
        • Clinical Trial Site
  • Israel
      • Jerusalem, Israel
        • Recruiting
        • Clinical Trial Site
  • Italy
    • Torino
      • Orbassano, Torino, Italy
        • Recruiting
        • Clinical Trial Site
  • Spain
      • Barcelona, Spain
        • Recruiting
        • Clinical Trial Site
  • Switzerland
      • Bern, Switzerland
        • Recruiting
        • Clinical Trial Site
  • United Kingdom
    • England
      • London, England, United Kingdom
        • Recruiting
        • Clinical Trial Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Recruiting
        • Clinical Trial Site
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Recruiting
        • Clinical Trial Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Clinical Trial Site
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Clinical Trial Site
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Clinical Trial Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Recruiting
        • Clinical Trial Site
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • Clinical Trial Site
      • Houston, Texas, United States, 77030
        • Recruiting
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients diagnosed with PH1 will be managed and treated per routine clinical practice.

Description

Inclusion Criteria:

  • Documented diagnosis of PH1, per physician's determination

Exclusion Criteria:

  • Currently enrolled in a clinical trial for any investigational agent

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Patients with PH1
Patients with a diagnosis of PH1 will be eligible for the study and will be managed and treated per routine clinical practice.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of Adverse Events in Lumasiran Treated Patients
Time Frame: Up to 7 years
Up to 7 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Up to 7 years
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.
Up to 7 years
Change in Urinary Oxalate Excretion
Time Frame: Baseline and every 12 months for up to 7 years
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
Baseline and every 12 months for up to 7 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Medical Director, Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 13, 2021

Primary Completion (Estimated)

September 1, 2028

Study Completion (Estimated)

September 1, 2028

Study Registration Dates

First Submitted

July 20, 2021

First Submitted That Met QC Criteria

July 20, 2021

First Posted (Actual)

July 29, 2021

Study Record Updates

Last Update Posted (Actual)

May 10, 2024

Last Update Submitted That Met QC Criteria

May 9, 2024

Last Verified

May 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-GO1-007

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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