MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Launched by MASONIC CANCER CENTER, UNIVERSITY OF MINNESOTA · May 9, 2024

Keywords

ClinConnect Summary

This clinical trial, titled MT2023-20, is exploring a new treatment approach for patients with severe aplastic anemia and other related conditions that affect the bone marrow, such as paroxysmal nocturnal hemoglobinuria and acquired pure red cell aplasia. The study is testing a type of stem cell transplant that uses a milder form of treatment before the transplant and a specific medication afterward to help the body accept the new cells. Researchers hope this method will improve outcomes for patients who have not responded well to traditional treatments.

To be eligible for the trial, participants should have been diagnosed with severe aplastic anemia or one of the other specified conditions and have certain blood counts that indicate their disease is severe. The trial is open to individuals of all ages, and while many can participate, there are some exclusions, such as those with active infections or severe liver disease. Participants will receive careful monitoring and tailored treatment based on their individual needs. This trial aims to provide new hope for patients struggling with serious blood disorders.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• * Idiopathic Severe Aplastic Anemia (SAA), characterized by one of the following:
• 1. Refractory cytopenia(s), with 1+ of the following:
• • 1. Platelets \<20,000/uL or transfusion dependent
• • 2. Absolute neutrophil count \<500/uL without hematopoietic growth factor support
• • 3. Absolute reticulocyte count \<60,000/uL AND bone marrow cellularity \<50% (with \< 30% residual hematopoietic cells)
• • 2. Early myelodysplastic features (bone marrow (BM) blasts \<5%), without history of MDS/AML pre-treatment.
• • 3. Idiopathic SAA with post-HCT graft failure (blood/marrow donor chimerism \<5%) requiring a 2nd allogeneic HCT
• * Paroxysmal Nocturnal Hemoglobinuria (PNH), including AA-PNH overlap syndrome, acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT), characterized by one of the following:
• 1. Refractory cytopenia(s), with 1+ of the following:
• • 1. Platelets \<20,000/uL or transfusion dependent
• • 2. Absolute neutrophil count \<500/uL without hematopoietic growth factor support
• • 3. Absolute reticulocyte count \<60,000/uL or red cell transfusion dependent AND Bone marrow evidence of 1 to 3-lineage aplasia OR peripheral blood PNH clone \>/= 10%
• • 2. Early myelodysplastic features (bone marrow (BM) blasts \<5%) without history of MDS/AML pre-treatment.
• • 3. Idiopathic PNH, aPRCA, or aAT with post-HCT graft failure (blood/marrow donor chimerism \<5%) requiring a 2nd allogeneic HCT
• • Adequate organ function within 30 days of conditioning regimen
• Exclusion Criteria:
• • Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 7 days of the start of treatment
• • Uncontrolled infection
• • Evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
• • Known allergy to any of the study components
• • Prior radiation therapy deemed excessive by radiation therapist for proposed low dose TBI exposure on this protocol
• • Diagnosis of an inherited bone marrow failure disorder such as Fanconi anemia, Telomere biology disorder, or Schwachman-Diamond syndrome, unless reviewed by the principal investigator and deemed appropriate for this approach (e.g. GATA2 deficiency)
• • Advanced myelodysplastic syndrome (MDS; BM blasts \>5%) or acute myeloid leukemia
• • Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements
• • Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study