New hope for rare blood disorder: Half-Matched transplants show promise
NCT ID NCT02828592
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is for people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells, leading to serious infections and bleeding. It tests a bone marrow transplant from a half-matched family donor, followed by a drug to reduce complications. The goal is to see if this approach can safely restore normal blood cell production in patients who don't have a fully matched donor.
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 20 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Sep 2016
- Expected to finish
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Aug 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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1 year 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.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction * Age \<= 65 years for previously treated and \<= 75 years for previously treated patients * KPS \>= 70% * Aplastic Anemia that meets the following criteria: Peripheral Blood (must fulfill 2 of 3): * \<500 PMN/mm3 * \<20,000 platelets * absolute reticulocyte count \<40,000/microL Bone Marrow (must be either): * markedly hypocellular (\<25% of normal cellularity) * moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above Exclusion Criteria: * poor cardiac function (LVEF \<40%) * poor pulmonary function (FEV1 \& FVC \<50% predicted) * poor liver function (bili \>= 2mg/dL) * poor renal function (creatinine \>= 2.0mg/dL or creatinine clearance \<40mL/min) * prior allogeneic transplant
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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
2 sites. 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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Blood and Marrow Transplant Group of Georgia
RECRUITINGAtlanta, Georgia, 30342, United States
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Northside Hospital
RECRUITINGAtlanta, Georgia, 30342, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cell transplants tested as a cure path for older aplastic anemia patients
- Can a gentler chemo-radiation combo make stem cell transplants safer for blood cancers?
- Half-Matched stem cell transplant offers hope for children with blood disorders
- New transplant approach aims to tame bone marrow failure
- New hope for older adults with rare blood disorder: safer transplant regimen under study
- New drug combo shows promise for rare bone marrow failure