Expanded cord blood transplant shows early promise for young cancer patients
NCT ID NCT04990323
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a new method of cord blood transplantation in 13 children and young adults with high-risk myeloid cancers. The approach uses a molecule called UM171 to grow more stem cells from a single cord blood unit, aiming to speed up recovery and reduce complications. Early results suggest the treatment is safe and may lead to better outcomes, but larger studies are needed.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- UM171-expanded cord blood transplant
- What this could lead to
- If successful, this approach could make cord blood transplants safer and more available for young patients with hard-to-treat blood cancers, potentially improving survival and reducing complications.
- What could go wrong
- This is an early-phase trial with only 13 participants, so results may not apply to all patients. There are still risks of relapse, infection, and graft-versus-host disease.
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 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
13 people
The number who actually took part.
- Started
-
Dec 2021
- Finished
-
Dec 2025
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
0 to 21 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 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: 1. Acute Myeloid Leukemia 1. Chemo-refractory relapse (MRD+) 2. Primary induction failure (no CR or CRi after \>= 2 courses of intensive induction therapy): \< 30% blasts in evaluable marrow. 3. Relapse after previous allogeneic (or autologous) transplant (\>4 months) 4. Secondary or therapy-related MDS/AML 5. Poor response to induction (5-30% blasts) or MDR+ after induction 2. Myelodysplastic syndrome (MDS) 1. Relapse after allogeneic or autologous transplant (\>4 months) 2. ≥10 % blasts within 30 days of start of conditioning regimen 3. Poor and very poor cytogenetics abnormalities 3. Chronic myelogenous leukemia: Patients who progressed to blast crisis 4. Mixed Phenotype Acute Leukemia: MRD+ or relapse after previous transplant (\>4 months). 5. JMML (Juvenile Myelo-Monocytic Leukemia) 6. Availability of 2 ≥ 4/8 HLA matched CBU (allele level: A, B, C and DRB1) 1. Cord to be expanded: CD34+ cell count ≥ 0.5 x 10\^5/kg and TNC ≥ 1.5 x 10\^7/kg (pre-cryo) 2. Back up cord: Pre-freeze TNC ≥ 2 x 10\^7/kg with CD34+ cells ≥ 1.5 x 10\^5/kg. If a single cord does not meet this criterion 2 back up cords will be an acceptable alternative with a minimum for each of 1.5 x 10\^7 TNC/kg with 1.0 x 10\^5 CD34+/kg. Another acceptable HSC back up source could be a haploidentical with medical clearance prior to starting conditioning regimen. 7. Lansky / Karnofsky \>60% 8. Bilirubin \< 2 x upper limit of normal (ULN) unless felt to be related to Gilbert's disease or hemolysis; AST and ALT \< 3 x ULN; alkaline phosphatase \< 5 x ULN 9. Estimated or measured creatinine clearance ≥ 50ml/min/1.73m2 10. Left ventricular ejection fraction of ≥ 40% 11. FVC, FEV1 and DLCO ≥ 50% of predicted 12. Signed written informed consent 13. Female patients of childbearing potential must have a negative serum pregnancy test within 7 days of enrolment and mush be willing to use an effective contraceptive method while enrolled in the study. Exclusion Criteria: 1. Previous allogeneic transplantation within 4 months. 2. Uncontrolled infection. 3. Presence of other malignancy other than the one for which the CB transplant is being performed, with an expected survival to be less than 75% at 5 years 4. Seropositive for HIV. 5. Hep B and C infection with measurable viral load. 6. Liver cirrhosis. 7. Active CNS disease. 8. Chloroma \> 2cm. 9. \>30% blasts in marrow in evaluable marrow sample. 10. Pregnancy, breastfeeding, or unwillingness to use appropriate contraception 11. Participation in a trial with an investigational agent within 30days prior to entry in the study. 12. Any abnormal condition or lab result that is considered by the PI capable or altering patient's condition or study outcome.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Acute myeloid leukemia are added.
Genom att skicka in godkänner du våra Användarvillkor
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.
Contacts and locations
Locations
-
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a Platelet-Boosting drug help control a rare bone marrow disorder?
- Can an HDAC inhibitor wipe out residual leukemia cells?
- Can an experimental pill block a cancer-driving enzyme in hard-to-treat leukemia?
- Two-Drug combo targets leukemia that outsmarted its first treatment
- Tweaking donor cells may shield older transplant patients from a dangerous complication
- Can a drug and donor cells stop leukemia from returning after transplant?