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New transplant approach aims to tame bone marrow failure

NCT ID NCT06412497

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2024

Expected to finish

May 2036

An estimate. End dates often move.

Lead sponsor

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

0 to 75 years

Sex

Anyone

Healthy volunteers

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

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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • University of Minnesota Masonic Cancer Center

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

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