Clinical Trial TitleEfficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia
National Clinical Trial Number:NCT04119050
Clinical Trial Protocol Description:
Warm autoimmune hemolytic anemia (wAIHA) is a rare disease that affects the red blood cells and causes anemia. It can occur as a primary disease or secondary to such diseases as systemic lupus erythematosus (SLE), lymphoma, chronic lymphocytic leukemia or after use of certain drugs. It is considered an autoimmune disorder because the body’s natural defenses destroy healthy tissue (red blood cells) by autoantibodies for unknown reasons. The process leads to premature destruction of healthy red blood cells and low levels of circulating red blood cells causing anemia. Anemia may cause fatigue, a pale skin color, yellowing of the skin and whites of the eyes (jaundice), and enlargement of the spleen and liver. The range of clinical presentation can vary from well-controlled to severe and life-threatening.
M281, an investigative drug, is designed to block a receptor called FcRn, a receptor that is involved in recycling of antibodies. Blocking this receptor results in a reduction of the antibodies that cause warm autoimmune hemolytic anemia and may potentially treat the disease. M281 has been tested in animals and also in healthy human subjects for safety evaluation.
The purpose of this study is to determine whether M281 is safe in the treatment of this disease, along with the standard treatment.
Participants will be asked to make a total of 15 visits to the study site over 36 weeks; each visit will take approximately 2 hours. This will include up to 4 weeks of the screening (qualification) period, 24 weeks of study treatment, and a follow-up visit 8 weeks after the final study treatment.
The study treatments will be administered as an intravenous infusion (the infusion will take approximately 15 minutes) and will be given every two weeks no matter what treatment group to which the participant is assigned. The needle will deliver the study drug, M281, or placebo (a sugar water solution).
Clinical Trial Eligibility Criteria:
In order to participate you must meet the following criteria:
- Are 18 years of age or older.
- Have been diagnosed with active primary or secondary wAIHA for at least 3 months and have received standard of care treatment for wAIHA, but it is not currently under adequate control.
- Have sufficient venous access to allow drug administration by IV infusion.
You will be excluded from the study if any of the following criteria apply to you:
- Have received a blood transfusion within 30 days prior to entering the study.
- Have received rituximab within 6 months prior to entering the study.
- Have a severe infection , such as, pneumonia, biliary tract infection, diverticulitis, or Clostridium difficile infection that requires parenteral anti-infectives and/or hospitalization, and/or is assessed as serious/clinically significant by the Investigator, within 8 weeks prior to screening for the study.
- Have a chronic infection or require chronic treatment with anti-infectives (antibiotics, antivirals).
This is a partial list of eligibility requirements. To inquire about your eligibility, please call the contact number provided. If you wish to inquire via email, please include the title of the study in your message.