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.
Recruiting
Phase 2
Interventional Study
Severe Aplastic Anemia
Acquired Amegakaryocytic Thrombocytopenia
Acquired Pure Red Cell Aplasia
Paroxysmal Nocturnal Hemoglobinuria
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 0 – 75
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 60 (estimated)
- Sponsor
- Masonic Cancer Center, University of Minnesota · Other
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About This Trial
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 o…
Trial Locations
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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 suppo…
Contacts