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Autoimmune haemolytic anaemia – Trials in Disease

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Clinical Trials for Autoimmune Haemolytic Anaemia

There are currently 6 ongoing clinical trials investigating new treatments for autoimmune haemolytic anaemia, a rare condition where the immune system mistakenly destroys the body’s own red blood cells. These trials are testing several promising medications across multiple European countries, offering hope for patients who have not responded well to standard treatments or cannot tolerate conventional therapies.

Clinical trial locations

Study of rilzabrutinib tablets compared to placebo in adults with warm autoimmune hemolytic anemia

This trial is investigating rilzabrutinib, a medication taken as a tablet, for adults with warm autoimmune hemolytic anemia (wAIHA). The study aims to determine if this medication can help maintain stable blood levels in patients whose disease has not responded well to standard treatments.

Main inclusion criteria: Participants must be adults (18 years or older) who have been diagnosed with warm autoimmune hemolytic anemia for at least 3 months. They must have previously tried corticosteroid treatment without maintaining a sustained response, or be unable to take corticosteroids. Their physical condition should be assessed as ECOG Grade 2 or lower, meaning they are able to walk and handle self-care. Both men and women can participate, and appropriate contraception methods must be used during the study.

Main exclusion criteria: People with current Evans syndrome (where the body makes antibodies against both red blood cells and platelets) cannot participate. Also excluded are those with a history of blood clotting disorders or recent significant blood clots, recent major surgery within 4 weeks, active hepatitis B or C, HIV, active tuberculosis, active cancer or cancer history within the past 3 years, severe heart conditions, severe kidney disease requiring dialysis, and those who are pregnant or breastfeeding.

Trial focus: The study will measure several aspects of treatment effects, including changes in hemoglobin levels (a protein in red blood cells that carries oxygen), changes in fatigue levels, and how quickly the treatment works. Participants will be randomly assigned to receive either rilzabrutinib tablets or placebo tablets by mouth. The maximum daily dose of rilzabrutinib will be 800 mg, and the treatment period will last for 224 weeks. Regular monitoring will include physical examinations, vital signs checks, heart monitoring through electrocardiograms, and tracking of any side effects.

Investigational drug: Rilzabrutinib works by targeting and inhibiting an enzyme called BTK (Bruton’s tyrosine kinase), which plays a role in the immune system response. The goal is to reduce the destruction of red blood cells and help maintain healthy hemoglobin levels in patients with this condition.

Study on the Effectiveness of Apixaban and Enoxaparin Sodium in Preventing Blood Clots in Patients with Autoimmune Hemolytic Anemia

This French clinical trial focuses on preventing blood clots (venous thromboembolism) in patients with autoimmune hemolytic anemia using a combination approach. The study uses enoxaparin sodium initially during hospitalization, followed by apixaban (Eliquis) taken orally.

Main inclusion criteria: Participants must be 18 years or older and have provided written informed consent. They must have a confirmed diagnosis of autoimmune hemolytic anemia (either primary or secondary to other conditions), with hemoglobin levels less than 12 g/dL, haptoglobin levels less than 0.4 g/L, and a positive direct antiglobulin test. Patients can be newly diagnosed or experiencing a relapse of the condition. Life expectancy must be more than 6 months.

Main exclusion criteria: The trial excludes patients who are not diagnosed with autoimmune hemolytic anemia, those outside the specified age range, and vulnerable populations such as children or elderly people requiring special protection.

Trial focus: The study evaluates the effectiveness of blood clot prevention over 24 weeks. Initially, patients receive enoxaparin sodium as a subcutaneous injection at 4000 IU per day during hospitalization. After discharge, they transition to apixaban 2.5 mg tablets taken twice daily (morning and evening) for 12 weeks. Throughout the study period, patients are monitored for any signs of blood clots, bleeding, or other complications.

Investigational drugs: Heparin (enoxaparin) is used as an injection under the skin during the initial treatment phase to help reduce the risk of blood clots during hospitalization. Apixaban is taken by mouth after the initial treatment to continue preventing blood clots over a longer period.

Long-Term Safety Study of Riliprubart in Patients with Cold Agglutinin Disease

This trial investigates the long-term safety and tolerability of riliprubart (SAR445088) in patients with cold agglutinin disease (CAD), a specific type of autoimmune hemolytic anemia where the immune system attacks red blood cells at cold temperatures.

Main inclusion criteria: Participants must be adults (18 years or older) with confirmed cold agglutinin disease. If previously treated with SAR445088, they must have completed a previous study with this drug and received a positive assessment from their doctor. If never treated with SAR445088, they must meet specific diagnostic criteria including chronic hemolysis (ongoing breakdown of red blood cells), positive direct antiglobulin tests, cold agglutinin titer of 64 or higher at 4°C, hemoglobin level of 10 g/dL or lower, and elevated bilirubin not due to liver disease. All participants must have received certain vaccinations within the last five years and at least 14 days before starting the study. Appropriate contraception methods are required for sexually active participants.

Main exclusion criteria: The study excludes patients with autoimmune hemolytic anemia that is not cold agglutinin disease, those outside the specified age range, vulnerable populations, and those who do not meet other eligibility criteria.

Trial focus: The study monitors participants over an extended period to observe any side effects or changes in their condition. Researchers track treatment-emergent adverse events and measure changes in various blood components such as bilirubin, hemoglobin, lactate dehydrogenase, and reticulocyte count. The trial also evaluates complement system pathway levels and monitors for the development of anti-SAR445088 antibodies. Regular follow-up visits assess the long-term safety and tolerability of riliprubart.

Investigational drug: SAR445088 (riliprubart) is administered as a solution through injection or infusion. It works by targeting specific pathways involved in the immune response, helping to reduce the destruction of red blood cells in patients with cold agglutinin disease.

Study for Patients with Advanced Malignancies: Continued Treatment with Parsaclisib Alone or with Itacitinib, Ruxolitinib, Ibrutinib, or Tafasitamab

This study allows patients with advanced cancers and certain immune system diseases to continue treatment with parsaclisib, either alone or in combination with other medications including itacitinib, ruxolitinib, ibrutinib, or tafasitamab.

Main inclusion criteria: Participants must be currently enrolled and receiving treatment in an Incyte-sponsored clinical study of parsaclisib. They must be tolerating the treatment well and receiving clinical benefit as determined by the investigator, with at least stable disease. Participants must demonstrate compliance with the parent study requirements, be willing to follow scheduled visits and treatment plans including preventive treatment for lung infections, and use appropriate contraception methods. They must be able to comprehend and sign an informed consent form.

Main exclusion criteria: Patients with advanced malignancies or immune-related diseases who do not meet the inclusion criteria are excluded from participation.

Trial focus: The study is open-label, meaning both participants and researchers know which treatments are being administered. Participants continue their current treatment regimen as they have been receiving it. Regular monitoring assesses safety, tracks any adverse effects or serious adverse effects, and evaluates the continued benefit of treatment. The study does not introduce new treatments but maintains the current therapeutic approach while gathering information on long-term effects.

Investigational drugs: Parsaclisib works by blocking specific proteins that help cancer cells grow. Itacitinib helps manage immune system-related conditions by interfering with signals that cause inflammation. Ruxolitinib reduces inflammation and is used for certain blood disorders. Ibrutinib targets proteins in cancer cells to slow their growth. Tafasitamab helps the immune system recognize and attack cancer cells.

Study on Nipocalimab for Adults with Warm Autoimmune Hemolytic Anemia

This trial evaluates nipocalimab, a monoclonal antibody medication given as an infusion, for treating adults with warm autoimmune hemolytic anemia. The study aims to determine if the treatment can improve hemoglobin levels without requiring additional rescue therapies.

Main inclusion criteria: Participants must be 18 years or older and have been diagnosed with warm autoimmune hemolytic anemia for at least 3 months with previous treatment history. New patients who have not been treated are not eligible. Hemoglobin levels must be less than 10 g/dL with signs of hemolysis such as high lactate dehydrogenase, low haptoglobin, or high indirect bilirubin, plus a positive Direct Antiglobulin Test. If taking corticosteroids, participants must have been on a stable dose for at least 4 weeks, or for 14 days before randomization. For immunosuppressants, a stable dose for 12 weeks is required, or they must have stopped at least 8 weeks before the study. Platelet count must be at least 30 x 109/L. Women of childbearing potential must have a negative pregnancy test and agree to avoid pregnancy using reliable birth control. Men must use condoms during the study and for 90 days after the last dose. Participants must have received vaccinations according to local guidelines, including COVID-19.

Main exclusion criteria: The study excludes individuals without warm autoimmune hemolytic anemia, those outside the specified age range, and vulnerable populations requiring special protection or care.

Trial focus: The study is designed as double-blind, meaning neither participants nor researchers know who receives nipocalimab or placebo during the initial phase. After this phase, there is a long-term extension where all participants may receive nipocalimab. Regular visits monitor health and treatment effects, with the primary goal of achieving hemoglobin concentration of at least 10 g/dL and an increase of at least 2 g/dL from baseline at three consecutive visits. The study tracks hemoglobin levels, overall health, and any side effects throughout the treatment period.

Investigational drug: Nipocalimab is given as an intravenous infusion. It works by blocking a specific receptor involved in the immune response, which helps reduce the destruction of red blood cells. The medication is designed to help reduce the immune system’s attack on red blood cells, potentially improving symptoms and increasing red blood cell counts.

Study on the Effects and Safety of Rilzabrutinib for Adults with Warm Autoimmune Hemolytic Anemia

This trial evaluates the effects and safety of rilzabrutinib tablets in adults with warm autoimmune hemolytic anemia. The study is divided into two parts: Part A evaluates initial effectiveness over 24 weeks, while Part B examines long-term effects for those who show positive results.

Main inclusion criteria: Participants must be male or female patients with confirmed diagnosis of primary warm autoimmune hemolytic anemia or systemic lupus erythematosus (SLE)-associated wAIHA without other SLE-related symptoms apart from skin and joint issues. They must have previously been unable to maintain lasting improvement after corticosteroid treatment. ECOG performance status must be grade 2 or lower, meaning they can walk and handle self-care but may be unable to work. Body mass index must be between 17.5 and 40 kg/m². Vaccination status must be up-to-date according to local guidelines. All contraceptive use must follow local regulations. For Part B only, participants must show evidence of treatment effectiveness by achieving an overall response during Part A and must have completed the 24-week Part A treatment period.

Main exclusion criteria: The study excludes patients with other serious health conditions that might interfere with the study, pregnant or breastfeeding women, those who participated in another clinical trial recently, those with a history of allergic reactions to similar medications, patients unable to follow study procedures or attend required visits, those with a history of drug or alcohol abuse, active infections requiring treatment, recent treatments for their condition, conditions affecting the immune system, and history of cancer except for certain successfully treated types.

Trial focus: During Part A, participants take rilzabrutinib tablets orally for 24 weeks. Their response is evaluated based on hemoglobin levels and other health indicators, with the primary goal being to assess the proportion of participants achieving an overall hemoglobin response. Those showing treatment efficacy during Part A may transition to Part B, which focuses on evaluating long-term effectiveness in maintaining hemoglobin response. Throughout both parts, regular assessments monitor health, safety, and changes in fatigue levels using the FACIT-Fatigue scale. The study also tracks the frequency of any additional treatments needed for wAIHA.

Investigational drug: Rilzabrutinib is taken in tablet form and works by inhibiting Bruton’s tyrosine kinase, a protein involved in the immune response. This helps reduce the destruction of red blood cells, potentially improving symptoms and overall health in patients with warm autoimmune hemolytic anemia.

Summary

The current landscape of clinical trials for autoimmune haemolytic anaemia shows a concentrated research effort across Europe, with multiple countries participating in various studies. Italy appears as a particularly active location, hosting trials for several different investigational medications. The trials span multiple European nations including Poland, Czechia, Netherlands, Austria, Greece, Denmark, Sweden, Spain, Germany, Hungary, Belgium, and France.

A notable pattern emerges in the types of treatments being studied. Rilzabrutinib appears in multiple trials, reflecting significant research interest in this BTK inhibitor as a potential treatment option. The studies focus primarily on warm autoimmune hemolytic anemia, with one trial specifically addressing cold agglutinin disease. Several trials are investigating novel approaches to managing the condition, including medications that target different aspects of the immune response, such as BTK inhibitors, complement pathway inhibitors, and FcRn blockers.

The trials generally target patients who have not responded well to standard corticosteroid therapy or who cannot tolerate conventional treatments, highlighting the need for alternative therapeutic options. Most studies require participants to be adults aged 18 or older, with confirmed diagnoses and specific laboratory criteria. Many trials incorporate long-term monitoring periods, some extending several years, to assess both safety and sustained effectiveness of the treatments.

An additional focus area includes the prevention of complications, as demonstrated by the French study on preventing blood clots in patients with autoimmune haemolytic anaemia. This addresses an important aspect of disease management beyond treating the primary condition itself.

Ongoing Clinical Trials on Autoimmune haemolytic anaemia

  • Long-Term Safety Study of Riliprubart in Patients with Cold Agglutinin Disease

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Germany Italy The Netherlands

  • Study on the Effects and Safety of Rilzabrutinib for Adults with Warm Autoimmune Hemolytic Anemia

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Denmark Italy Spain

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