Primary Hyperoxaluria Type 1BONAPH1DENCT04982393
BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
An observational study of Primary Hyperoxaluria Type 1, run by Alnylam Pharmaceuticals at 30 sites in 11 countries.
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Hyperoxaluria Type 1
- How many people
- 207 participants
- Where
- 30 sitesin 11 countries
- Study dates
- 13 Dec 2021to 2 Jan 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients diagnosed with PH1 will be managed and treated per routine clinical practice.
Who may be able to join
- Documented diagnosis of PH1, per physician's determination
Who cannot join
- Currently enrolled in a clinical trial for any investigational agent
Trial locations and contacts
30 sites in 11 countries.
30 sites in 11 countries
United States8 sites
Clinical Trial Site
Boston, Massachusetts, 02115
Clinical Trial Site
Cincinnati, Ohio, 45229
Clinical Trial Site
Dallas, Texas, 75390
Clinical Trial Site
Houston, Texas, 77030
Clinical Trial Site
Phoenix, Arizona, 85016
Clinical Trial Site
Pittsburgh, Pennsylvania, 15213
Clinical Trial Site
Rochester, Minnesota, 55905
Clinical Trial Site
Washington D.C., District of Columbia, 20010
Canada3 sites
Clinical Trial Site
Hamilton, Ontario
Clinical Trial Site
Laurier, Quebec
Clinical Trial Site
Toronto, Ontario
France3 sites
Clinical Trial Site
Bordeaux
Clinical Trial Site
Lyon
Clinical Trial Site
Paris
Germany3 sites
Clinical Trial Site
Berlin
Clinical Trial Site
Cologne
Clinical Trial Site
Hamburg
Italy3 sites
Clinical Trial Site
Milan
Clinical Trial Site
Orbassano, Torino
Clinical Trial Site
Verona
Spain3 sites
Clinical Trial Site
Barcelona
Clinical Trial Site
Las Palmas
Clinical Trial Site
Santa Cruz de Tenerife, Canary Islands
United Kingdom2 sites
Clinical Trial Site
London, England
Clinical Trial Site
London
About this study
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.
- Conditions
- Primary Hyperoxaluria Type 1
- Keywords
- PH1, Lumasiran, Primary Hyperoxaluria Type 1
- Medical subject headings
- Primary hyperoxaluria type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events in Lumasiran Treated Patients
Measured: Up to 7 years
Secondary outcomes (4)
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.
Measured: 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.
Measured: Up to 7 years
Change in Urinary Oxalate Excretion
Measured: Baseline and every 12 months for up to 7 years
Change in Plasma Oxalate
Measured: Baseline and every 12 months for up to 7 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2021
- Study start13 Dec 2021
- Primary completion2 Jan 2029 (estimated)
- Study completion2 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Hyperoxaluria Type 1Studies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 207 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04982393
- Sponsor's protocol ID
- ALN-GO1-007
- Other IDs
- EUPAS43242
- Acronym
- BONAPH1DE
- Status last verified
- Oct 2026
- First submitted
- 20 Jul 2021
- Last updated
- 6 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
- ClinicalTrials.gov ID
- NCT04982393
- Acronym / sponsor ID
- BONAPH1DE / ALN-GO1-007
- Conditions
- Primary Hyperoxaluria Type 1
- Keywords
- PH1, Lumasiran, Primary Hyperoxaluria Type 1
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 207 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 13 Dec 2021 (actual)
- Primary completion
- 2 Jan 2029 (estimated)
- Study completion
- 2 Jan 2029 (estimated)
- First posted
- 29 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients diagnosed with PH1 will be managed and treated per routine clinical practice.
Key inclusion criteria (1 listed)
- Documented diagnosis of PH1, per physician's determination
Key exclusion criteria (1 listed)
- Currently enrolled in a clinical trial for any investigational agent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Incidence of Adverse Events in Lumasiran Treated Patients
Time frame: Up to 7 years
4 secondary outcomes
Incidence of Selected Events of Interest in PH1 Patients
Time frame: Up to 7 years
12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version)
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Alnylam Pharmaceuticals
30 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Hyperoxaluria Type 1, run by Alnylam Pharmaceuticals at 30 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 6 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, Canada, France, Germany, Italy and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Adverse Events in Lumasiran Treated Patients (time frame: Up to 7 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2026.