Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase 2, Open-label, Multicenter Study of Mitapivat in Subjects With Sickle Cell Disease and Nephropathy

This study is for adults with Sickle Cell Disease who also have kidney problems. The new medicine, Mitapivat, is being tested to see if it can help improve kidney function and overall health. Researchers will closely watch participants for six months to see if the medicine reduces a specific marker of kidney damage called ACR by at least 30%. They will also track changes in kidney health over time, how often people need to visit the emergency room, and how many days they spend in the hospital. Safety is very important, so any side effects will be carefully recorded to understand the medicine's overall impact.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Agios Pharmaceuticals Inc.

Enrolment target

Start

07 Oct 2024

Sickle Cell Disease (SCD) and Nephropathy

What is this study about?

This study is looking into a new medicine called Mitapivat for adults who have both Sickle Cell Disease (SCD) and kidney problems. SCD is a condition that affects red blood cells and can lead to various health issues, including kidney damage. The main goal of this research is to see if Mitapivat can help to improve kidney health.

Researchers will be focusing on a specific measure called ACR, which stands for Albumin to Creatinine Ratio. A high ACR can indicate kidney damage. They want to find out if taking Mitapivat can reduce the ACR level by at least 30% after six months. This would suggest that the medicine is helping to protect the kidneys from further damage.

Beyond just the ACR, the study will also look at other important health indicators. This includes how kidney function changes over time, how often people need to go to the emergency room, and how many days they spend in the hospital. They will also carefully record any side effects that people experience from the medicine. By gathering all this information, doctors hope to learn if Mitapivat is a safe and effective treatment for people with Sickle Cell Disease and kidney issues.

Key takeaways

This study tests a new medicine, Mitapivat, for Sickle Cell Disease and kidney damage.
It aims to see if Mitapivat can improve kidney health over six months.
Researchers will monitor kidney function, hospital visits, and any side effects.
Only adults aged 18+ with both conditions can join.
Participation involves taking the medicine and regular clinic visits.

Who may be eligible?

This study is open to adults aged 18 and older. Both men and women can take part.

To join, you must have been diagnosed with Sickle Cell Disease and also have kidney problems. The doctors will check your medical history to make sure the study medicine is right for you and won't cause any unexpected issues with other health conditions you might have.

There might be other specific health checks or conditions that would mean you couldn't take part, but these would be discussed in detail by the study doctor.

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 18 years old or older?
Do you have a diagnosis of Sickle Cell Disease?
Have you been told you have kidney problems?
Are you willing to attend regular clinic appointments?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be given the study medicine, Mitapivat. This is an "open-label" study, meaning both you and the study team will know you are receiving Mitapivat. You will take the medicine for six months. During this time, you'll have several visits to the clinic so the research team can monitor your health closely.

At these visits, you can expect to have blood and urine tests to check your kidney function and other health markers. The team will also ask you how you're feeling and if you've experienced any changes or side effects. After the six months of treatment, there will likely be follow-up appointments to continue monitoring your health, but the study description doesn't specify the exact duration of follow-up.

Potential risks and benefits

Taking part in this study might offer potential benefits, such as receiving a new medicine that could improve your kidney health and overall well-being. However, there's no guarantee the medicine will work for everyone, and it might not be better than current treatments. As with any medicine, there are potential risks, including side effects that could range from mild to serious. The study team will carefully monitor you for any side effects and will explain all known risks before you join. Remember, taking part is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (2)

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
Ireland

Common questions

What is Mitapivat?

Mitapivat is a new medicine being tested to see if it can help people with Sickle Cell Disease and kidney problems.

What are 'kidney problems' in this study?

In this study, 'kidney problems' are identified by a specific measure in your urine called ACR, which indicates kidney damage.

How long will I be in the study?

You'll take the study medicine for at least six months, and there will be follow-up visits after that.

Will I know if I'm getting the actual medicine?

Yes, this is an 'open-label' study, so you and the doctors will know you are receiving Mitapivat.

Can I stop the study if I want to?

Yes, you can choose to leave the study at any time, and it won't affect your medical care.