A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body
This study is looking into a new experimental treatment called REGN7999 for adults who have a condition called non-transfusion dependent beta-thalassemia (NTDT) and also have too much iron in their bodies. NTDT means you have a blood disorder but don't need regular blood transfusions. The study will test an injection given under the skin. We want to find out if this new drug is safe to use and if it can effectively reduce the extra iron. We'll be using special scans called MRIs to measure iron levels and checking for any side effects. This research could help people with NTDT better manage their iron levels and improve their health.
At a glance
What is this study about?
This research study is focused on understanding a new medicine, called REGN7999, for people with a blood condition called non-transfusion dependent beta-thalassemia (NTDT). NTDT is a type of thalassemia where your body doesn't make enough healthy red blood cells, but you don't typically need frequent blood transfusions. A common problem for people with NTDT is that their bodies can build up too much iron, which can be harmful to organs over time. This new medicine is given as an injection under the skin.
The main goals of this study are to see how safe the new medicine is and how well it works. This means we'll be carefully watching for any unwanted effects (side effects) and also checking if the medicine helps to lower the extra iron in the body. We'll use special medical scans called MRI to measure iron levels in different parts of the body. We're also curious to understand how the body handles the medicine – how much is in the blood and if the body's immune system reacts to it.
Finding better ways to manage iron overload in NTDT is very important because too much iron can lead to health problems. This study is a 'Phase 2' study, which means it's one of the earlier steps in testing new medicines on a larger group of people to get a clearer idea of their safety and effectiveness.
Key takeaways
- This study is for adults (18-65) with non-transfusion dependent beta-thalassemia (NTDT) and too much iron.
- It tests a new under-the-skin injection called REGN7999 to see if it's safe and lowers iron levels.
- MRI scans will be used to measure iron in the body.
- You might receive the new drug or a placebo (inactive substance).
- Participation involves clinic visits, blood tests, and scans.
- Your health and safety are closely monitored during the study.
Who may be eligible?
To be considered for this study, you must be an adult between 18 and 65 years old and have a confirmed diagnosis of non-transfusion dependent beta-thalassemia (NTDT). It's also important that you have extra iron in your body, which will be checked with a special MRI scan and blood tests. Your iron levels need to be above certain amounts to be included.
There are also some reasons why you might not be able to join. For instance, if your haemoglobin (a measure of red blood cells) is too low, or if you've recently had a blood transfusion. If you're currently taking other medicines to remove iron from your body (called iron chelation therapy), there are specific rules about how long you need to have been on a stable dose, or if you need to have stopped them for a certain period before joining. Also, if you have severe kidney or liver problems, or can't have an MRI scan, you wouldn't be able to take part. Other specific medical conditions or recent treatments could also mean you're not suitable.
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 between 18 and 65 years old?
- Do you have a diagnosis of non-transfusion dependent beta-thalassemia (NTDT)?
- Have you been told you have too much iron in your body (iron overload)?
- Have you had a blood transfusion recently (within the last 12 weeks)? (If yes, you may not be eligible)
- Do you have severe kidney or liver problems?
- Can you have an MRI scan?
What does participation involve?
If you join this study, it will involve several visits to the clinic so the medical team can monitor your health closely. You will receive either the study drug, REGN7999, or a placebo (an inactive substance that looks like the study drug). Neither you nor your study doctor will know which one you are receiving. The study involves regular blood tests, check-ups, and special MRI scans to measure your iron levels. There will also be tests to see how your body reacts to the drug and if it produces any antibodies against it. The total duration of your involvement in the study will be explained in detail by the study team, and it involves consistent medication administration, regular health checks, and follow-up to ensure your safety and monitor the drug's effects.
Potential risks and benefits
Locations (26)
- Weill Cornell MedicineVerified postcodeNew York, United States· Recruiting
- K Eristavi National Center of Experimental and Clinical SurgeryVerified postcodeTbilisi, Georgia· Recruiting
- Medinvest Institute of Hematology and Transfusiology LimitedVerified postcodeTbilisi, Georgia· Recruiting
- Children's Hospital Agia SophiaVerified postcodeAthens, Greece· Recruiting
- Laiko General Hospital of AthensVerified postcodeAthens, Greece· Recruiting
- University Hospital of IoanninaVerified postcodeIoannina, Greece· Recruiting
- Hemato-Oncology Clinic Ahmedabad PVT. LTD.Verified postcodeAhmedabad, India· Recruiting
- Nirmal Hospital Pvt. Ltd - SuratVerified postcodeSurat, India· Recruiting
- Amrita Institute of Medical Sciences and Research Centre (AIMS)Verified postcodeKochi, India· Recruiting
- K J Somaiya Super Specialty Hospital & Research CentreVerified postcodeMumbai, India· Recruiting
- All India Institute of Medical Sciences, New DelhiVerified postcodeNew Delhi, India· Recruiting
- Indraprastha Apollo HospitalsVerified postcodeNew Delhi, India· Recruiting
Common questions
What is 'non-transfusion dependent beta-thalassemia'?
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.
What does 'iron overload' mean?
It means you have too much iron built up in your body, which can be harmful to your organs over time.
What is REGN7999?
It's an experimental drug being tested in this study, given as an injection under the skin, to see if it can help reduce iron levels.
What is an MRI scan?
An MRI is a special type of scan that uses strong magnets and radio waves to create detailed pictures inside your body, and in this study, it's used to measure iron levels.
Can I choose not to take part in the study?
Yes, taking part is completely voluntary. You can decide not to join, or you can leave the study at any time, without it affecting your medical care.
How to find out more
Clinical Trials Administrator
Always speak to your GP or specialist before deciding to take part in a study.
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Discussion
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