RecruitingPhase 2

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.

United States60 participantsStarted 2024-06-05

Plain-language summary

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.

Who can participate

Age range0 Years – 75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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
✓. Refractory cytopenia(s), with 1+ of the following:
✓. Platelets \<20,000/uL or transfusion dependent

What they're measuring

Incidence of grade 3-4 acute GvHD

Timeframe: 1 year post HCT

Incidence of chronic GvHD-free, failure-free survival (GFFS)

Timeframe: 1 year post HCT

Incidence of chronic GvHD-free survival

Timeframe: 1 year post HCT

Trial details

NCT IDNCT06412497

SponsorMasonic Cancer Center, University of Minnesota

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2035-05-01

Contact for this trial

Christen Ebens, MD, MPH

612-624-1791 ebens012@umn.edu

View on ClinicalTrials.gov More Severe Aplastic Anemia trials

Plain-language summary

Who can participate

Age range0 Years – 75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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
✓. Refractory cytopenia(s), with 1+ of the following:
✓. Platelets \<20,000/uL or transfusion dependent