New transplant approach aims to tame bone marrow failure
NCT ID NCT06412497
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tests a bone marrow transplant using lower doses of chemotherapy and radiation to prepare the body, along with a drug called cyclophosphamide after transplant to prevent graft-versus-host disease. It enrolls 60 people with severe aplastic anemia or related bone marrow failure conditions. The goal is to see if this approach reduces serious complications while still helping the body produce healthy blood cells.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- hematopoietic cell transplant with reduced intensity conditioning and post-transplant cyclophosphamide
- What this could lead to
- If successful, this could offer a safer transplant option for people with severe bone marrow failure, reducing serious side effects like graft-versus-host disease.
- What could go wrong
- This is an early phase 2 trial with only 60 participants, so results may not apply to everyone. The transplant still carries risks of infection, organ damage, and graft failure.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 60 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2024
- Expected to finish
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May 2036
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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0 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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University of Minnesota Masonic Cancer Center
RECRUITINGMinneapolis, Minnesota, 55455, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Half-Matched stem cell transplants tested as a cure path for older aplastic anemia patients
- A pill that could replace infusions for a rare blood disease?
- Can a gentler chemo-radiation combo make stem cell transplants safer for blood cancers?
- Every-8-Week shot could simplify PNH treatment
- Half-Matched stem cell transplant offers hope for children with blood disorders