Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humansInterventional

A Phase 1/2 Study of REGN7257 (Anti-Interleukin 2 Receptor Subunit Gamma [IL2RG] Monoclonal Antibody) in Patients with Severe Aplastic Anemia that is Refractory to or Relapsed on Immunosuppressive Therapy

This research study is looking at a new medicine, REGN7257, for a serious blood condition called severe aplastic anaemia (SAA). This condition means your body doesn't make enough new blood cells. The study is for people whose SAA has not responded well to standard treatments or has returned. The main goals are to check the safety of REGN7257 and to see how effective it is in helping patients. Researchers will carefully watch for any side effects and see if the medicine helps improve blood counts and reduce the need for blood transfusions. This is a first-time-in-humans study, meaning it’s an early stage to gather important information about this new potential treatment.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Regeneron Pharmaceuticals Inc.

Enrolment target

Start

14 Jun 2024

Severe aplastic anemia (SAA)

What is this study about?

This research study is investigating a new medicine called REGN7257 for people living with severe aplastic anaemia (SAA). SAA is a rare but serious condition where your bone marrow, the sponge-like material inside your bones, doesn't produce enough healthy blood cells. This can lead to problems like tiredness, infections, and bleeding.

The study is specifically for individuals whose SAA hasn't gotten better with common treatments (called 'immunosuppressive therapy') or has come back after treatment. The new medicine, REGN7257, is a type of 'monoclonal antibody' which is a special protein designed to target specific cells in the body. In this case, it aims to affect certain parts of your immune system that might be involved in SAA.

This is an early-stage study, called Phase 1/2. This means it's one of the first times this medicine is being given to people. The main goals are to understand if REGN7257 is safe to use and to see if it has a positive effect on severe aplastic anaemia. Researchers will be looking closely at any side effects and how patients' blood counts change, including their need for blood transfusions.

Key takeaways

Tests a new medicine (REGN7257) for severe aplastic anaemia (SAA).
For adults whose SAA hasn't responded to usual treatments.
Aims to check safety and if the medicine helps improve blood counts.
It's an early-stage study, monitoring new treatments carefully.

Who may be eligible?

This study is looking for adults aged 18 and over, with no upper age limit, who have severe aplastic anaemia.

To be considered for this study, your severe aplastic anaemia must not have improved with standard treatments that suppress the immune system, or it must have returned after you previously had those treatments.

Both men and women can take part. There will be other specific health checks and medical history reviews to make sure the study is a good fit for you and that taking part would be safe.

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 severe aplastic anaemia (SAA)?
Has your SAA not gotten better with other treatments, or has it come back?
Are you able to attend regular clinic visits and have blood tests?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the new medicine, REGN7257. Your participation will involve regular visits to the clinic for medical check-ups, blood tests, and to discuss any changes in your health or any side effects you might experience. These assessments will help the researchers understand how the medicine is affecting your body and your condition. You will be closely monitored throughout the study period. The total duration of your involvement, including follow-up, will be determined by the study design, but generally, early-stage studies like this involve frequent monitoring over several months.

Potential risks and benefits

Participating in this study might offer a potential benefit if the new medicine helps your severe aplastic anaemia, especially if other treatments haven't worked for you. However, as with any new medicine, there are potential risks, including side effects that are not yet fully known. The research team will carefully explain all known risks and monitor you closely for any unwanted effects. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
France

Common questions

What is severe aplastic anaemia?

It's a serious blood condition where your body doesn't produce enough new blood cells, leading to tiredness, infections, and bleeding.

What is REGN7257?

It's a new medicine being tested, a special protein that targets parts of your immune system to help with SAA.

Why is this study being done?

Researchers want to see if REGN7257 is safe and effective for people with SAA who haven't responded to other treatments.

Who can join this study?

Adults (18 and over) with severe aplastic anaemia that hasn't improved with standard treatments or has come back.

What does 'Phase 1/2' mean?

It means this is an early study, one of the first times the medicine is being given to people, to check safety and initial effects.