Ongoing Clinical Trials for Cold Type Haemolytic Anaemia
There are currently 2 clinical trials investigating new treatment approaches for Cold Type Haemolytic Anaemia. These studies are taking place across several European countries and are testing medications that work by modifying how the immune system affects blood cells. (Also known as: Cold Agglutinin Disease, CAD)
Clinical trial locations
- Austria
- Belgium
- Denmark
- Germany
- Italy
- Netherlands
- Norway
- Spain
Study on Povetacicept for Patients with Autoimmune Cytopenias (wAIHA, CAD, ITP)
This trial is investigating povetacicept, also known as ALPN-303, for patients with autoimmune cytopenias. These are conditions where the immune system mistakenly attacks and destroys blood cells. The study specifically includes three types of blood disorders: warm autoimmune hemolytic anemia, cold agglutinin disease, and immune thrombocytopenia.
Main inclusion criteria: To participate in this study, you must have one of the three specific blood disorders. For cold agglutinin disease specifically, you must have had the condition for at least 12 weeks with ongoing breakdown of red blood cells. You need positive tests for certain antibodies and a cold agglutinin titer of 64 or more at 4°C, with no obvious cancer present. Your hemoglobin level must have been 9 or less at some point, and you must have at least one sign of red blood cell breakdown, such as low haptoglobin or high lactate dehydrogenase. You must have tried at least one treatment that did not work or where the condition returned. Both men and women within the specified age range can participate.
Main exclusion criteria: You cannot participate if you have other serious health conditions that might interfere with the study, are pregnant or breastfeeding, have had recent major surgery, are in another clinical trial, have severe allergic reaction history, have an active infection requiring treatment, have a history of drug or alcohol abuse, have received certain medications that might affect results, have a history of cancer except certain skin cancers, or have uncontrolled high blood pressure or diabetes.
Study focus: The trial aims to evaluate the safety and tolerability of povetacicept in patients with these autoimmune conditions. Participants will receive the medication as an injection under the skin and will be closely monitored through regular check-ups. The study will assess any side effects, measure the medication’s effectiveness on blood counts and health markers, and gather information on how the body responds to treatment over time. The study is expected to conclude by July 30, 2026.
Investigational drug: Povetacicept is given as a subcutaneous injection, meaning it is injected under the skin. It works as an immunomodulatory agent, aiming to balance the immune response and reduce the abnormal destruction of blood cells in these specific blood disorders.
Study on Zanubrutinib for Patients with Primary Cold Agglutinin Disease
This clinical trial is studying zanubrutinib, a medication taken as an oral capsule, for treating primary cold agglutinin disease. This is a rare blood disorder where the immune system mistakenly attacks and destroys red blood cells, especially in cold temperatures, causing them to clump together.
Main inclusion criteria: To join this study, you must have a confirmed diagnosis of cold agglutinin disease that includes chronic breakdown of red blood cells for more than 3 months with low haptoglobin levels, a cold agglutinin titer of 64 or higher at 4°C, and specific positive blood tests. You must also have evidence of a clonal B-cell disorder in your bone marrow. You need treatment either because your hemoglobin level is 10.5 g/dL or lower, or you have significant cold-induced symptoms. You must have either not responded to at least one previous treatment or relapsed, or be newly diagnosed and not eligible for current treatment options. You must be 18 years or older, have adequate bone marrow function with specific blood count requirements, and have ferritin levels above normal. Women of childbearing potential must use effective contraception.
Main exclusion criteria: You cannot participate if you have other serious health conditions that might interfere with the study, are currently in another clinical trial, have had or are planning major surgery during the study, are pregnant or breastfeeding, have allergic reactions to similar medications, have an active infection requiring treatment, have a history of drug or alcohol abuse, or cannot follow study procedures or attend required visits.
Study focus: The trial evaluates how effective zanubrutinib is in treating patients with primary cold agglutinin disease. Participants will receive the medication over 6 treatment cycles, with each cycle lasting 28 days. Throughout treatment, doctors will closely monitor the response of the disease to the medication, including changes in blood parameters and symptoms. The study will also assess improvements in cold-induced symptoms, quality of life, and fatigue levels. The primary assessment will occur at the end of cycle 6, between days 22 and 28, to determine the treatment’s effectiveness.
Investigational drug: Zanubrutinib is taken orally in capsule form. It works by inhibiting Bruton’s tyrosine kinase, a protein that plays a crucial role in the signaling pathways leading to antibody production that causes red blood cells to clump together. This medication belongs to the class of Bruton’s tyrosine kinase inhibitors.
Summary
Two clinical trials are currently underway for cold type haemolytic anaemia across Europe. The trials are concentrated primarily in Northern and Central European countries, with Norway hosting both studies, suggesting a regional research focus. Each study tests a different approach to managing the condition: povetacicept works by modulating the overall immune response, while zanubrutinib targets a specific enzyme involved in antibody production. The povetacicept trial includes patients with cold agglutinin disease alongside other autoimmune blood disorders, while the zanubrutinib study focuses exclusively on primary cold agglutinin disease. Both studies require participants to have tried previous treatments without success, reflecting the need for new therapeutic options for patients whose condition has not responded to existing therapies.