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New treatment option for Severe Aplastic Anemia
Official title 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.
ClinicalTrials.gov ID: NCT06412497
What this study is testing
What is Rituximab?
Rituximab is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for severe aplastic anemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and/or clonal hematopoiesis.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 75
You may be able to join if
- Idiopathic Severe Aplastic Anemia (SAA), characterized by one of the following:
- Refractory cytopenia(s), with 1+ of the following:
- Platelets \<20,000/uL or transfusion dependent
- Absolute neutrophil count \<500/uL without hematopoietic growth factor support
- Absolute reticulocyte count \<60,000/uL AND bone marrow cellularity \<50% (with \< 30% residual hematopoietic cells)
You likely can't join if
- Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test...
- 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...
See the full eligibility criteria
- Idiopathic Severe Aplastic Anemia (SAA), characterized by one of the following:
- Refractory cytopenia(s), with 1+ of the following:
- Platelets \<20,000/uL or transfusion dependent
- Absolute neutrophil count \<500/uL without hematopoietic growth factor support
- Absolute reticulocyte count \<60,000/uL AND bone marrow cellularity \<50% (with \< 30% residual hematopoietic cells)
- Early myelodysplastic features (bone marrow (BM) blasts \<5%), without history of MDS/AML pre-treatment.
- 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:
- Refractory cytopenia(s), with 1+ of the following:
- Platelets \<20,000/uL or transfusion dependent
- Absolute neutrophil count \<500/uL without hematopoietic growth factor support
- Absolute reticulocyte count \ /= 10%
- Early myelodysplastic features (bone marrow (BM) blasts \<5%) without history of MDS/AML pre-treatment.
- 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
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Minneapolis, Minnesota, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Minneapolis, Minnesota, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.