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Observing patients with Primary Hyperoxaluria Type 1 and lumasiran treatment

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

Alnylam Pharmaceuticals · NCT04982393

This study looks at how Primary Hyperoxaluria Type 1 affects patients over time and checks how well the treatment lumasiran works for them in the real world.

Quick facts

Study typeObservational
Enrollment200 (estimated)
SexAll
SponsorAlnylam Pharmaceuticals (industry)
Locations30 sites (Phoenix, Arizona and 29 other locations)
Trial IDNCT04982393 on ClinicalTrials.gov

What this trial studies

This observational study aims to describe the natural history and progression of patients diagnosed with Primary Hyperoxaluria Type 1 (PH1). It will also characterize the long-term real-world safety and effectiveness of the treatment lumasiran. By collecting data from various clinical sites, the study seeks to provide insights into how PH1 affects patients over time and the impact of lumasiran on their condition.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals with a documented diagnosis of Primary Hyperoxaluria Type 1.

Not a fit: Patients currently enrolled in a clinical trial for any investigational agent may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable information on the long-term safety and effectiveness of lumasiran for patients with PH1.

How similar studies have performed: While this study focuses on observational data, similar studies have shown success in understanding the natural history of rare diseases and the effectiveness of treatments.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Documented diagnosis of PH1, per physician's determination

Exclusion Criteria:

* Currently enrolled in a clinical trial for any investigational agent

Where this trial is running

Phoenix, Arizona and 29 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Primary Hyperoxaluria Type 1, PH1, Lumasiran

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.