Ongoing Clinical Trials for Pure Red Cell Aplasia
There is currently 1 ongoing clinical trial investigating treatment options for Pure Red Cell Aplasia, specifically in patients who develop this condition after stem cell transplantation with a major ABO blood type mismatch. The trial is testing a targeted antibody therapy called isatuximab and is being conducted in France.
Clinical trial locations
Study on Isatuximab for Treating Pure Red Cell Aplasia in Patients with Major ABO Mismatch After Stem Cell Transplantation
This clinical trial focuses on a specific complication that can occur after allogeneic hematopoietic stem cell transplantation—a condition where the body stops producing red blood cells properly. This happens when there is a major mismatch in blood types between the donor and the recipient, causing the immune system to attack and destroy red blood cell precursors in the bone marrow.
Who can participate:
- Patients aged 15 years or older
- Those who have received an allogeneic stem cell transplant with a major ABO blood type mismatch
- Individuals diagnosed with Pure Red Cell Aplasia, meaning they require regular red blood cell transfusions 60 days after transplant, with a reticulocyte count under 10 G/L
- Patients whose donor cells are working well and whose white blood cells and platelets have recovered
- Those with no return or worsening of their original disease
- Participants must be willing to use effective contraception during treatment and for at least 5 months after the last dose
- Must have health insurance coverage and provide signed informed consent (for those under 18, both parents must sign)
Who cannot participate:
- Patients who have not experienced a major ABO mismatch after stem cell transplant
- Those who have not been diagnosed with Pure Red Cell Aplasia
- Individuals outside the specified age range for the study
- Those unwilling or unable to follow study procedures and requirements
- Patients unable to provide informed consent
What the trial involves:
The study is testing isatuximab, a monoclonal antibody that targets a specific protein called CD38 on the surface of certain cells. This medication helps the immune system identify and destroy these cells, potentially reducing the time it takes for the body to start producing red blood cells again.
Participants are randomly assigned to either receive isatuximab treatment or standard supportive care, which may include blood transfusions. The treatment group receives isatuximab through an intravenous infusion directly into a vein. This randomization typically occurs around six months after the transplant.
Throughout the study, researchers closely monitor several factors including the number of blood transfusions needed, ferritin levels in the blood, and any side effects experienced. Quality of life is evaluated at specific time points: 60 days, 100 days, and several months after transplant. The main goal is to determine how quickly patients can become independent of transfusions and to assess overall health outcomes.
The study is expected to continue until March 20, 2027, and aims to provide valuable insights into whether isatuximab can improve outcomes for patients experiencing this complication after stem cell transplantation.
Summary
Currently, only one clinical trial is actively recruiting patients with Pure Red Cell Aplasia following stem cell transplantation. This trial is based in France and represents an important effort to find more effective treatments for this challenging complication. The focus on isatuximab, a targeted antibody therapy, reflects a modern approach to managing immune-related complications after transplantation. Patients who have undergone stem cell transplantation with major ABO mismatches and are experiencing difficulty producing red blood cells may wish to discuss this trial with their healthcare team to determine if participation might be appropriate for their situation.
