AuthorisedTherapeutic confirmatory (Phase III)Interventional

Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3

This study is looking at a new medicine called stiripentol for people aged 6 and older who have primary hyperoxaluria (PH). PH is a rare condition that affects the kidneys and can lead to kidney stones. The main goal is to see if stiripentol helps lower the amount of a substance called oxalate in the urine over 6 months. Researchers also want to check if the medicine is safe and if it improves kidney health and overall well-being. Participants will receive either the active medicine or a placebo (a dummy pill) to compare the effects.

At a glance

Status

Authorised

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Biocodex

Enrolment target

Start

16 May 2024

Primary Hyperoxaluria (PH) of all subtypes (PH1 PH2 and PH3)

What is this study about?

This study is a phase 3 clinical trial, which means it's one of the final steps in testing a new medicine before it can be considered for wider use. It's focused on a condition called primary hyperoxaluria (PH). This is a rare, inherited disorder where the body produces too much oxalate, a natural chemical. This excess oxalate can build up and form painful kidney stones, or even lead to serious kidney damage over time.

The medicine being tested is called stiripentol, and it's thought to help reduce these high oxalate levels. Researchers will be carefully comparing stiripentol to a 'placebo' – a pill that looks the same but contains no active medicine. This helps them understand if any changes they see are truly due to the stiripentol or something else.

The main thing the study wants to find out is how much stiripentol can lower the amount of oxalate in people's urine over six months. They'll also be looking at other important things, like how well the kidneys are working, if stiripentol reduces kidney stone problems, and how people's quality of life changes while taking the medicine. This is a crucial step to see if stiripentol could become a helpful treatment for people living with primary hyperoxaluria.

Key takeaways

This study is for people aged 6 and over with primary hyperoxaluria (PH).
It's testing a medicine called stiripentol to lower oxalate in urine.
Participants will take either stiripentol or a dummy pill (placebo).
The study lasts 6 months and includes regular clinic visits.
Potential benefits include access to a new medicine, but there are also potential risks.
You can stop participating at any time.

Who may be eligible?

This study is looking for both adults and children aged 6 years and older who have been diagnosed with primary hyperoxaluria (this includes types 1, 2, or 3). Both men and women can take part. There is no upper age limit for adults wanting to join the study.

To ensure the study is safe and the results are clear, there will be specific health checks and medical history questions to see if the study is suitable for you or your child. For example, certain other medical conditions or medicines you are currently taking might mean you can't participate.

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.

Are you or your child aged 6 years or older?
Do you have a confirmed diagnosis of primary hyperoxaluria (PH1, PH2, or PH3)?
Are you able to attend regular clinic appointments for 6 months?
Are you willing to take study capsules and have blood and urine tests?
Do you understand that you might receive a placebo?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will be given either the study medicine (stiripentol) or a placebo (a dummy pill) in capsule form. You will need to take these pills regularly as instructed. Over the course of the study, you will have several visits to the clinic. These visits will involve various tests and assessments, such as urine collections over 24 hours to measure oxalate levels, blood tests to check kidney function, and possibly questions about your general health and how you are feeling.

The study will last for a total of 6 months. During this time, the researchers will carefully monitor your health and how the treatment is affecting you. There will also be follow-up appointments to ensure everything is going well and to collect all necessary information. Be prepared for regular contact with the study team and committing to the scheduled appointments.

Potential risks and benefits

Taking part in a study like this might offer potential benefits, such as gaining access to a new medicine that could help manage your primary hyperoxaluria before it's widely available. You'll also receive close medical attention and monitoring from a specialist team. However, there are potential risks, including side effects from the medicine – though these will be carefully monitored. There's also a chance you might receive the placebo and not the active medicine. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

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

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Unverified
France
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Unverified
Belgium
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Unverified
Italy

Common questions

What is primary hyperoxaluria (PH)?

It's a rare inherited kidney condition where your body makes too much oxalate, which can lead to kidney stones and kidney damage.

What is stiripentol?

It's the name of the medicine being tested in this study to see if it can help reduce high oxalate levels.

What does 'placebo' mean?

A placebo is a pill that looks like the study medicine but doesn't contain any active ingredients. It helps researchers compare the effects accurately.

How long will the study last?

The main part of the study will last for 6 months.

Will I know if I'm getting the real medicine or the placebo?

No, during the study, neither you nor your doctor will know whether you are receiving the active medicine or the placebo. This is standard practice to ensure fair results.