A Phase 2, Two-Part, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of Subcutaneously Administered REGN7999 (An Inhibitor of TMPRSS6) in Participants with Iron Overload Due to Non-Transfusion Dependent β-Thalassemia.
This research study is looking at a new medicine called REGN7999. It's for adults (18 years and older) who have a condition called non-transfusion dependent beta-thalassemia (NTDT), which means they don't need regular blood transfusions but still have symptoms. People with NTDT can sometimes build up too much iron in their body, which can be harmful. This study wants to find out if REGN7999 can help reduce these iron levels and if it's safe to use. Half the participants will get the new medicine, and the other half will get a placebo (a dummy medicine) to compare the effects. The main things we'll be checking are how much iron is in the liver and if there are any side effects.
At a glance
What is this study about?
This study is designed to learn more about a new medicine called REGN7999. It's for people who have a condition called beta-thalassemia, specifically the type known as non-transfusion dependent beta-thalassemia (NTDT). This means that people with NTDT often don't need regular blood transfusions, but their bodies can still produce red blood cells differently, leading to various health issues.
One common problem for people with NTDT is having too much iron build up in their body, especially in organs like the liver. This can happen over time and can cause serious health problems if not managed. REGN7999 is a new medicine that aims to help the body control these iron levels. We want to see if this medicine can effectively reduce the iron stored in the liver and how safe it is for people to use.
During the study, some participants will receive the new medicine, REGN7999, while others will receive a placebo – which looks exactly like the medicine but contains no active drug. This comparison helps us truly understand the effects of REGN7999. Researchers will be carefully monitoring changes in iron levels in the liver using special scans and also keeping a close eye on participants' overall health and any potential side effects. The goal is to find better ways to manage iron overload in people with NTDT and hopefully improve their quality of life.
Key takeaways
- This study is testing a new medicine (REGN7999) for non-transfusion dependent beta-thalassemia (NTDT).
- It aims to reduce too much iron build-up in the liver.
- Participants will receive either the new medicine or a placebo (dummy medicine).
- Iron levels in the liver will be measured using special MRI scans.
- Safety and side effects will be carefully monitored.
- The study is for adults aged 18 and over who have NTDT and iron overload.
Who may be eligible?
This study is for adult men and women, meaning you need to be at least 18 years old to take part. There isn't an upper age limit mentioned.
To join, you must have a specific type of blood condition called non-transfusion dependent beta-thalassemia (NTDT). This generally means you have beta-thalassemia but don't need regular blood transfusions every few weeks or months.
Crucially, you must also be experiencing iron overload, which means you have too much iron built up in your body, particularly in your liver. The study will likely check your iron levels to confirm this.
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 non-transfusion dependent beta-thalassemia (NTDT)?
- Do you have too much iron in your body (iron overload)?
- Are you able to attend regular clinic visits and have MRI scans?
What does participation involve?
If you decide to join this study, you would receive either the new medicine (REGN7999) or a placebo, which looks identical but has no active drug. The medicine would be given as an injection under the skin, similar to a flu jab. Since it's a 'double-blind' study, neither you nor your study doctor will know whether you're getting the active medicine or the placebo until the study is over.
You would have regular visits to the study clinic. These visits would involve health checks, blood tests to monitor your iron levels and general health, and special MRI scans to measure the iron concentration in your liver. Researchers will also be checking for any side effects or changes in your health.
The study involves two main parts, and the total duration for each participant, including follow-up, would be decided by the study doctors. You would receive all study-related medicine and tests at no cost.
Potential risks and benefits
Locations (2)
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Common questions
What is non-transfusion dependent beta-thalassemia (NTDT)?
It's a type of inherited blood condition where your body doesn't make enough healthy red blood cells, but you don't usually need regular blood transfusions to manage it.
What does 'iron overload' mean?
It means you have too much iron built up in your body, which can happen with certain conditions like NTDT and can damage organs like the liver if not treated.
What is a 'placebo'?
A placebo is a 'dummy' medicine that looks like the real drug but doesn't contain any active ingredients. It helps researchers compare the effects of the actual medicine fairly.
How will they check my iron levels?
They will use special MRI scans (a type of body scan) to measure how much iron is stored in your liver, as well as regular blood tests.
Can I stop participating if I want to?
Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your standard medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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