Active not recruitingOBSERVATIONAL

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

This study, called BONAPH1DE, is observing people who have been diagnosed with a rare inherited kidney condition called Primary Hyperoxaluria Type 1 (PH1). The main goal is to understand the natural journey of this condition as it progresses over many years. Researchers also want to see how a medicine called lumasiran, which is used to treat PH1, performs in everyday situations, specifically looking at how safe it is and how well it helps patients in the long term. This is an observational study, meaning participants will be monitored, but no new treatments are being tested as part of the study itself.

At a glance

Status

Active not recruiting

Sponsor

Alnylam Pharmaceuticals

Enrolment target

207

Start

13 Dec 2021

Estimated completion

01 Sep 2028

Primary Hyperoxaluria Type 1

What is this study about?

Imagine a study that acts like a detailed diary of how a health condition affects people's lives over time. That's essentially what the BONAPH1DE study is doing for a rare kidney disease called Primary Hyperoxaluria Type 1 (PH1).

PH1 is a condition where the body produces too much of a chemical called oxalate, which can build up and damage the kidneys. This study aims to gather information on how PH1 typically progresses, from when it's first diagnosed through many years. Researchers want to understand the different ways the condition can affect people, what symptoms they experience, and how their health changes over time — all in real-world settings, not just in a controlled laboratory.

Another important part of this study is to see how a specific medicine, lumasiran, works for people with PH1 in their daily lives. Lumasiran is already approved for use, and this study will help doctors understand its long-term safety and how effective it is outside of very strict clinical trial environments. This kind of information is crucial for health professionals to give the best possible care to patients with PH1.

Key takeaways

This study is about a rare kidney condition called Primary Hyperoxaluria Type 1 (PH1).
It aims to understand how PH1 develops over many years.
Researchers will also observe how an existing medicine, lumasiran, works in daily life.
It's an 'observational' study, meaning no new treatments are being tested.
People of all ages with a PH1 diagnosis may be eligible to participate.

Who may be eligible?

To be part of this study, you must have a confirmed diagnosis of Primary Hyperoxaluria Type 1 (PH1) from your doctor. This is the main requirement to join.

There are also some reasons why you might not be able to join. For example, if you are already taking part in another study that is testing a new medicine, you would not be eligible for this one. This helps make sure that the results of this study are clear and not influenced by other experimental treatments.

People of all ages, from children to adults, and of any gender can potentially participate, as long as they meet the diagnosis criteria for PH1 and are not in another trial for an experimental drug.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do I have a confirmed diagnosis of Primary Hyperoxaluria Type 1 (PH1) from my doctor?
Am I currently taking part in another clinical trial that is testing a new, experimental drug?
Am I willing to allow my medical information related to my PH1 to be used for research?
Am I comfortable with researchers gathering information during my routine doctor's visits?

Answer every question to see your result.

What does participation involve?

As an observational study, taking part in BONAPH1DE means researchers will collect information about your health and your PH1 condition over a period of time. This usually involves reviewing your existing medical records, and potentially collecting information during your routine doctor's appointments. You won't be given any new or experimental medications as part of this study; instead, researchers will simply observe how your condition progresses and how any treatments you are already receiving, like lumasiran, are affecting you in your normal life. The exact number of visits or assessments will depend on your individual healthcare plan and how often you see your doctor for your PH1. You will be asked if your medical information can be used for the study research.

Potential risks and benefits

The main benefit of taking part in this study is contributing to a better understanding of PH1, which could help improve care for future patients. Because this is an observational study, you will not be given any new treatments, so there are generally no additional risks beyond those associated with your usual medical care for PH1. You will continue to receive care from your own doctors as normal. You are free to withdraw from the study at any time without it affecting your medical care.

Locations (30)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Clinical Trial Site
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Phoenix, United States
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Washington D.C., United States
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Boston, United States
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Rochester, United States
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Cincinnati, United States
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Pittsburgh, United States
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Dallas, United States
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Houston, United States
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Ghent, Belgium
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Liège, Belgium
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Hamilton, Canada
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Toronto, Canada

Common questions

What is Primary Hyperoxaluria Type 1 (PH1)?

PH1 is a very rare inherited condition where your body produces too much oxalate, a substance that can build up and harm your kidneys.

Is this study testing a new medicine?

No, this study is not testing a new medicine. It's observing how an already approved medicine, lumasiran, works in real-life situations for people with PH1.

What does 'observational study' mean?

It means researchers will just gather information about your health and how your condition progresses over time, without giving you any experimental treatments or changing your usual medical care.

How long will I be in the study?

The study aims to look at how PH1 progresses over the long term, so participation could involve watching your health records over several years. Your doctors will be able to give you more specific details.

Will I have extra doctor's appointments?

Generally, no. Information for the study will be collected during your regular visits to the doctor for your PH1 care, not requiring additional appointments just for the study.