Cord blood transplants: a new hope for Hard-to-Treat blood diseases?
NCT ID NCT03016806
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing whether umbilical cord blood stem cells from unrelated donors can safely and effectively restore blood cell production in people with serious conditions like leukemia, immune deficiencies, and inherited blood disorders. The main goal is to see how quickly the new cells start working (engraftment) and how often the transplant fails. Researchers are also tracking side effects like graft-versus-host disease, where donor cells attack the patient's body.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- umbilical cord blood stem cells
- What this could lead to
- If successful, this could improve the success rate of cord blood transplants for people with serious blood or immune disorders, offering a potential path to disease control.
- What could go wrong
- This is a small, early-phase study (30 participants) focused on feasibility and safety. The transplant may fail to engraft, or patients may develop severe graft-versus-host disease, a serious complication.
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.
- Participants
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About 30 people
The number the study aims to enrol. It can still change while the study runs.
- Start date
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Jun 2015
- Expected to finish
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Jun 2027
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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2 months 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: * Appropriate diagnosis: Patients must have a disease or syndrome amenable to therapy with hematopoietic stem cell transplantation. Diagnoses include, but are not limited to: * Congenital and Other Non-malignant Disorders: * Immunodeficiency disorders (e.g. Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome) * Congenital hematopoietic stem cell defects (e.g. Chediak-Higashi Syndrome, Congenital Osteopetrosis, Osteogenesis Imperfecta) * Metabolic disorders (e.g. Hurler's Syndrome) * Severe aplastic anemia * High-Risk Leukemia: * Acute Myelogenous Leukemia * Refractory to standard induction therapy (more than 1 cycle required to achieve remission) * Recurrent (in CR ≥ 2) * Treatment-related AML or MDS * Evolved from myelodysplastic syndrome * Presence of FLT3 abnormalities * FAB M6 or M7 * Adverse cytogenetics * Myelodysplastic Syndrome * Acute Lymphoblastic Leukemia including T lymphoblastic leukemia: * Refractory to standard induction therapy (time to CR \>4 weeks) * Recurrent (in CR ≥ 2) * WBC count \>30,000/mcL at diagnosis * Age \>30 at diagnosis * Adverse cytogenetics, such as t(9:22), t(1:19), t(4:11), and other MLL rearrangements. * Chronic Myelogenous Leukemia in accelerated phase or blast crisis * Biphenotypic or undifferentiated leukemia * Burkitt's leukemia or lymphoma * Lymphoma: * Large cell, Mantle cell, Hodgkin lymphoma refractory or recurrent, chemo-sensitive, and ineligible for an autologous stem cell transplant or previously treated with autologous SCT * Marginal zone or follicular lymphoma that is progressive after at least two prior therapies * Multiple Myeloma, recurrent following high-dose therapy and autologous SCT or ineligible for an autologous HSCT * Solid tumors, with efficacy of allogeneic HSCT demonstrated for the specific disease and disease status * Adequate organ function: * Cardiac - LVEF \>45%, or shortening fraction \>25%, Absence of congestive heart failure or conduction disturbances with high risk for sudden death * Pulmonary - DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted; * Renal - serum Cr \< 1.5 times the upper limit of normal for age or GFR ≥ 50 ml/min/1.73m2 * Hepatic - total bilirubin level \< 2 times the upper limit of normal (except for patients with Gilbert's syndrome or hemolysis); if the primary disease process is causal, this criterion will be reconsidered. ALT, AST, and Alkaline phosphatase ≤ 5 times upper limit of normal. * Performance Status Karnofsky or Lansky score ≥ 70%. * Informed Consent must be obtained prior to initiating conditioning therapy. * Receipt of viable cord blood product(s), single or dual, must be confirmed with the stem cell processing laboratory prior to initiating conditioning therapy. Exclusion Criteria: * Availability of 10/10 or 9/10 HLA-matched related or unrelated donor within a reasonable timeframe dictated by the clinical urgency of the transplant * Autologous HSCT \< 6 months prior to proposed UCB transplant * Pregnant or breast feeding * Current uncontrolled infection * Evidence of HIV infection or positive HIV serology
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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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Wilmot Cancer Institute
RECRUITINGRochester, New York, 14642, United States
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