ClinicalTrials.gov record NCT06412497
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.
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.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Phase 2 study testing a reduced-intensity conditioned allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for people with severe aplastic anemia, PNH, aPRCA, or aAT. Dosing of pre-transplant drugs is individualized using population pharmacokinetics and low-dose total body irradiation (TBI) is adjusted by age, myelodysplasia features, or clonal hematopoiesis. Participants receive conditioning, stem cell infusion, GVHD prophylaxis, and are followed for outcomes up to two years.
What it is studying. The study tests a transplant approach that combines reduced-intensity conditioning (including fludarabine dosed using population pharmacokinetic guidance), cyclophosphamide, rabbit ATG, and low-dose total body irradiation when indicated, followed by stem cell infusion and post-transplant cyclophosphamide for graft-versus-host disease (GvHD) prevention. It compares two parallel groups defined by age and presence of clonal hematopoiesis and measures transplant-related outcomes.
What the researchers hope to learn. Scientific goals are to measure transplant outcomes, including incidence of grade 3–4 acute GvHD at 1 year, chronic GvHD-free, failure-free survival (GFFS) at 1 year, chronic GvHD-free survival at 1 year, and secondary outcomes such as neutrophil and platelet recovery, overall survival at 1 and 2 years, and incidence of chronic GvHD at 1 and 2 years.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Rituximab
- Rabbit ATG
- Cyclophosphamide
- Fludarabine
- Post-Transplant G-CSF
- Tacrolimus
- Mycophenolate Mofetil
- Biological
- Cell Infusion
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion 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 <60,000/uL or red cell transfusion dependent AND Bone marrow evidence of 1 to 3-lineage aplasia OR peripheral blood PNH clone >/= 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
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
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants receive a reduced-intensity conditioning course (drugs such as rabbit ATG, cyclophosphamide, model-based fludarabine dosing) and low-dose total body irradiation (dose depends on age and clonal hematopoiesis), followed by a donor stem-cell infusion on day 0. Post-transplant care includes post-transplant cyclophosphamide, G-CSF from about day +5 until blood counts recover, and graft-versus-host disease (GvHD) prophylaxis with tacrolimus (through about day +180) and a shorter course of mycophenolate (typically stopped around day +35 or after engraftment). Procedures include IV drug infusions, outpatient rituximab if indicated for EBV, radiation treatments, clinical monitoring for engraftment and GvHD, and tests such as pregnancy tests and bone marrow evaluation when required by eligibility or follow-up. Supportive care and follow-up assessments continue through two years after the transplant.
How the study is structured. Interventional Phase 2, non-randomized, two parallel arms (based on age and whether clonal hematopoiesis is present). Participants receive a tailored pre-transplant conditioning regimen, possible low-dose TBI, stem cell infusion, and post-transplant GvHD prophylaxis including cyclophosphamide; follow-up and supportive care continue through two years. The study is open-label (no masking).
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on paroxysmal nocturnal hemoglobinuria
Important dates
- Study start
- Jun 5, 2024
- Primary completion
- May 1, 2035 (estimated)
- Study completion
- May 1, 2036 (estimated)
- Target enrollment
- 60 participants (estimated)
- Last updated on registry
- Jun 3, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Meera Srikanthan, MD 📞 (612) 626-2961 ✉ srika038@umn.edu
Participating sites (1)
- University of Minnesota Masonic Cancer CenterRecruiting
Minneapolis, Minnesota, 55455 · United States
- Meera Srikanthan, MD · srika038@umn.edu
Terms used on this page · explanations by MedicalUpdates.ai
- pharmacokinetic
- How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
- Refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- Absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- biopsy
- Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
- principal investigator
- The doctor or scientist responsible for running the study at a site or overall.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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