In short
Currently, there is 1 ongoing clinical trial investigating new treatments for aplastic anaemia. This trial is exploring an investigational medication called REGN7257 for adults with severe aplastic anaemia who have not responded to or have relapsed after standard immunosuppressive therapy. The trial is being conducted in France and aims to evaluate both the safety and effectiveness of this new treatment approach.
Clinical trial locations
Study of REGN7257 for Adults with Severe Aplastic Anemia Unresponsive to or Relapsed After Immunosuppressive Therapy
This clinical trial is investigating a new treatment for severe aplastic anaemia, a serious blood disorder where the bone marrow fails to produce enough blood cells. The condition can lead to fatigue, increased risk of infections, and easy bruising or bleeding due to deficiencies in red blood cells, white blood cells, and platelets.
Main inclusion criteria:
- Patients must have severe aplastic anaemia that either did not improve with immunosuppressive therapy (IST) or returned after initially responding to this treatment
- Hematopoietic stem cell transplantation (a procedure to replace damaged bone marrow with healthy stem cells) must not be available, suitable, or must have been declined by the patient
- The liver and kidneys must be functioning adequately as defined by the study protocol
- Both male and female patients can participate
Main exclusion criteria:
- Patients who have blood conditions other than severe aplastic anaemia
- Patients who fall outside the specified age range
- Those who do not meet the specific clinical trial requirements
Focus and goals:
The trial is divided into two parts. Part A focuses on understanding how safe and tolerable the treatment is for patients whose condition is resistant to immunosuppressive therapy or has relapsed. Part B also evaluates effectiveness in patients whose condition has returned after previous treatments.
Throughout the study, participants undergo regular monitoring to assess safety and any side effects. The researchers track changes in blood cell counts, including lymphocytes, neutrophils, haemoglobin, reticulocytes, and platelets. They also examine changes in immune cell subsets such as T cells, B cells, and natural killer cells. The study measures the overall response rate, complete response, and partial response to treatment. The trial is expected to conclude by July 2026.
Investigational drug:
The treatment being tested is REGN7257, a monoclonal antibody. Monoclonal antibodies are proteins designed to target specific parts of the immune system. REGN7257 specifically targets the interleukin 2 receptor subunit gamma (IL2RG), which plays a role in the body's immune response. The medication is given as an intravenous infusion, meaning it is delivered directly into the bloodstream through a vein. This drug is currently being studied in clinical trials and is not yet widely available in medical practice.
Summary
There is currently one active clinical trial for aplastic anaemia, taking place in France. This trial focuses specifically on patients with severe aplastic anaemia who have limited treatment options due to either lack of response to or relapse after standard immunosuppressive therapy. The study is investigating REGN7257, a novel monoclonal antibody that targets a specific part of the immune system. The trial represents an important step in exploring new treatment options for patients whose condition has not improved with existing therapies and for whom stem cell transplantation is not an option. The study is expected to continue until mid-2026, providing valuable information about both the safety and potential effectiveness of this new approach.
