Second chance: milder prep for stem cell transplants may help when first fails
NCT ID NCT01666080
First seen Jul 06, 2026 · Last updated Jul 07, 2026 · Updated 1 time
Summary
This trial tests a second stem cell transplant using a gentler conditioning regimen of busulfan, fludarabine, and low-dose total body irradiation. It is for people with blood disorders, hemoglobin diseases, or immune deficiencies whose first transplant did not achieve enough donor cell engraftment. The goal is to see if this approach can successfully establish donor cells and restore healthy blood and immune function.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Busulfan, Fludarabine, total body irradiation, and stem cell transplant
- What this could lead to
- If successful, this approach could offer a second chance at a working transplant for people whose first one failed, potentially controlling their blood disorder or immune deficiency.
- What could go wrong
- This is a small, early-stage guideline study, not a large controlled trial. Risks include graft failure, infection, and side effects from the conditioning regimen.
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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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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About 30 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2012
- Expected to finish
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Jun 2028
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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Up to 55 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: * Diagnosis of any disease for which a second or greater hematopoietic stem cell transplant is needed due to insufficient donor chimerism following hematopoietic recovery after previous HSCT. Determination of "insufficiency of donor chimerism" will be made by the treating transplant physician. Occasionally donor derived engraftment may be present, but sustained aplasia or failed recovery of sufficient hematopoiesis requires administration of a second graft. This intervention may be used for both situations. * Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program * Transplantation using sufficiently matched related donors (such as matched siblings) or unrelated donors will be considered. Both granulocyte-colony stimulating factor (GCSF) stimulated peripheral blood grafts and bone marrow grafts will be considered, although bone marrow will be the priority. * Cord blood grafts, both related and unrelated, are also eligible. As this protocol will use a reduced intensity regimen, this protocol will use the current recommendations of the University of Minnesota for choosing cord blood grafts. If a single cord blood unit cell dose is insufficient, double cord transplantation should be considered if sufficiently matched cord blood units are available. The priority of choosing cord blood donors is based on the current institutional recommendations. * Exclusion of Metabolic Disorder or other Inherited Disorder Carrier Status from related donor and unrelated cord blood grafts as appropriate for primary disease. At the discretion of the treating transplant physician, an allograft from the previous donor may be used, if available. * Age, Performance Status, Consent * Age: 0 to 55 years * Consent: voluntary written consent (adult or parental/guardian) Exclusion Criteria: * Previous irradiation that precludes the safe administration of an additional dose of 200 cGy of total body irradiation (TBI). Radiation Oncology will evaluate all patients who have had previous radiation therapy or TBI for approval to receive an additional 200 cGy of TBI * Pregnant or breastfeeding * Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted * HIV positive * While it would be advantageous to begin therapy on this second transplant regimen \> 6 months following a prior myeloablative regimen or \>2 months after a reduced intensity regimen, it is recognized that there are circumstances where this may not be practical.
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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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Masonic Cancer Center, University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55455, United States
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Other studies related to the condition(s) this trial covers.
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- Gentler transplant shows promise for kids with blood diseases
- New immune cell boost may shield kids from Post-Transplant infections