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Regn7257

A groundbreaking clinical trial is underway to evaluate the potential of REGN7257, an experimental drug designed to treat severe aplastic anemia (SAA). This study focuses on patients whose condition has not responded well to previous immunosuppressive therapies. The trial aims to assess the safety, tolerability, and effectiveness of REGN7257 in improving blood cell production and reducing the need for transfusions in SAA patients.

Table of Contents

What is REGN7257?

REGN7257 is an experimental drug that is currently being researched for the treatment of severe aplastic anemia (SAA). It is classified as an anti-interleukin 2 receptor subunit gamma (IL2RG) monoclonal antibody. This means it’s a type of protein designed to target and block a specific part of the immune system that may be involved in the development of SAA[1].

Target Condition: Severe Aplastic Anemia (SAA)

Severe aplastic anemia (SAA) is a serious bone marrow disorder. In this condition, the bone marrow fails to produce enough blood cells, including red blood cells, white blood cells, and platelets. This leads to various health problems such as fatigue, increased risk of infections, and bleeding issues[1].

Purpose of the Clinical Study

The main goal of this study is to investigate REGN7257 in patients with SAA who have not responded well to other immunosuppressive therapies (ISTs) or have experienced a relapse. The research aims to answer several important questions[1]:

  • How safe and tolerable is REGN7257 for patients with SAA?
  • What side effects might patients experience when taking REGN7257?
  • How does REGN7257 work in the body?
  • How much REGN7257 remains in the blood after dosing?
  • Can REGN7257 increase certain blood cell counts after treatment?
  • How quickly does REGN7257 work to raise blood cell counts?
  • In patients who respond positively, how long does the response last throughout the study?
  • Can REGN7257 reduce the need for platelet and red blood cell transfusions?
  • How does REGN7257 affect immune cell counts and composition?
  • Does the body produce antibodies against REGN7257 (anti-drug antibodies)?

Study Design and Administration

The clinical trial for REGN7257 is designed in two parts[1]:

  1. Part A: This involves single ascending dose (SAD) escalation cohorts. This means that different groups of patients will receive a single dose of REGN7257, with each group receiving a higher dose than the previous one.
  2. Part B: This part involves multiple REGN7257 dosages, likely to assess the effects of repeated treatments.

In both parts of the study, REGN7257 is administered through intravenous (IV) infusion. This means the drug is given directly into the patient’s vein[1].

Primary Outcomes Being Measured

The study will primarily focus on the safety and effectiveness of REGN7257. Key measurements include[1]:

  • Adverse events (AEs) and serious adverse events (SAEs): These are any undesirable experiences associated with the use of REGN7257. They will be monitored for 12 months post-treatment (approximately 52 weeks) and through the end of the study (approximately 78 weeks).
  • Treatment-emergent adverse events (TEAEs): These are adverse events that appear or worsen after starting the treatment. They will be monitored for their incidence and severity.
  • Overall response rate (ORR): This measures how many patients show improvement in their condition after 6 months (approximately 26 weeks) of treatment.

Secondary Outcomes Being Evaluated

The study will also look at several secondary outcomes to further understand the effects of REGN7257[1]:

  • Complete response (CR) and partial response (PR): These measure the degree of improvement in the patient’s condition after 3 months (approximately 12 weeks) of treatment.
  • Time to best response and time to first response: These measure how quickly patients show improvement.
  • Any clinical response: This will be monitored until the end of the study (approximately 52 or 78 weeks).
  • Transfusion needs: The study will track how many platelet and red blood cell transfusions patients need over time.
  • Blood cell counts: Changes in various blood cell counts (lymphocytes, neutrophils, hemoglobin, reticulocytes, and platelets) will be monitored.
  • Immune cell subsets: Changes in T cells, B cells, and Natural Killer (NK) cells will be observed.
  • Drug concentration: The amount of REGN7257 in the blood over time will be measured.
  • Anti-drug antibodies (ADA): The study will check if patients develop antibodies against REGN7257 over time.
Aspect Details
Drug Name REGN7257
Condition Studied Severe Aplastic Anemia (SAA)
Study Design Phase 1/2, two-part study (Part A and Part B)
Administration Method Intravenous (IV) infusion
Primary Outcomes Incidence of adverse events, serious adverse events, treatment-emergent adverse events, and overall response rate
Secondary Outcomes Time to response, transfusion requirements, changes in blood cell counts, immune cell subsets, drug concentrations, and anti-drug antibody formation
Study Duration Approximately 78 weeks

FAQ

  • What is REGN7257? REGN7257 is an experimental drug being studied for the treatment of severe aplastic anemia. It is a monoclonal antibody that targets the interleukin 2 receptor subunit gamma (IL2RG).
  • What is the main purpose of this clinical trial? The main purpose of this two-part study is to test how safe and tolerable REGN7257 is in patients with severe aplastic anemia (SAA) who have not responded well to other immunosuppressive therapies.
  • How is REGN7257 administered? REGN7257 is administered through intravenous (IV) infusion in both Part A and Part B of the study.
  • What are the primary outcomes being measured in this trial? The primary outcomes include the incidence of adverse events, serious adverse events, and treatment-emergent adverse events. Additionally, the overall response rate at 6 months is being evaluated.
  • How long does the study last? The study lasts approximately 78 weeks, with various measurements taken at different time points throughout the trial, including at 3 months, 6 months, and 12 months post-treatment.

Ongoing Clinical Trials on Regn7257

  • Study of REGN7257 for Adults with Severe Aplastic Anemia Unresponsive to or Relapsed After Immunosuppressive Therapy

    Not recruiting

    1 1
    Investigated drugs:
    France

Glossary

  • Severe Aplastic Anemia (SAA): A serious condition where the bone marrow fails to produce enough blood cells, leading to a decrease in all types of blood cells.
  • Immunosuppressive Therapy (IST): A treatment that suppresses or reduces the strength of the body's immune system, often used in autoimmune diseases or to prevent rejection of transplanted organs.
  • Monoclonal Antibody: A type of protein made in the laboratory that can bind to substances in the body, including cancer cells. They are used to treat various diseases, including some types of cancer.
  • Interleukin 2 Receptor Subunit Gamma (IL2RG): A protein component of certain immune cell receptors that plays a crucial role in the development and function of various immune cells.
  • Intravenous (IV) Infusion: A method of delivering medications or fluids directly into a vein using a needle or tube.
  • Adverse Events (AEs): Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.
  • Serious Adverse Events (SAEs): An adverse event that results in death, is life-threatening, requires hospitalization, causes a significant incapacity, or requires intervention to prevent permanent impairment.
  • Treatment-Emergent Adverse Events (TEAEs): Adverse events that appear or worsen during treatment in a clinical study.
  • Overall Response Rate (ORR): The proportion of patients who have a partial or complete response to treatment.
  • Complete Response (CR): The disappearance of all signs of cancer in response to treatment.
  • Partial Response (PR): A decrease in the size of a tumor, or in the extent of cancer in the body, in response to treatment.
  • Anti-Drug Antibodies (ADA): Antibodies that the body produces against a therapeutic drug, which can potentially reduce the drug's effectiveness or cause adverse reactions.

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