NCT04103879CompletedPhase 2
US Study of UM171-Expanded CB in Patients With High Risk Leukemia/Myelodysplasia
A Phase II Open-Label Study of UM171-Expanded Cord Blood Transplantation in Patients With High and Very High Risk Acute Leukemia/Myelodysplasia
Source: ClinicalTrials.gov · Last updated 23 Mar 2026
Brief summary (registry, in English)
Cord blood (CB) transplants are an option for patients lacking an HLA identical donor but are hampered by low cell dose, prolonged aplasia and high transplant related mortality. UM171, a novel and potent agonist of hematopoietic stem cell self renewal could solve this major limitation, allowing for CB's important qualities as lower risk of chronic GVHD and relapse to prevail. In a previous trial (NCT02668315), the CB expansion protocol using the ECT-001-CB technology (UM171 molecule) has proven to be technically feasible and safe. UM171 expanded CB was associated with a median neutrophil recovery at day (D)+18 post transplant. Amongst 22 patients who received a single UM171 CB transplant with a median follow-up of 18 months, risk of TRM (5%) and grade 3-4 acute GVHD (10%) were low. There was no moderate-severe chronic GVHD. Thus, overall and progression free survival at 12 months were impressive at 90% and 74%, respectively. The UM171 expansion protocol allowed access to smaller, better HLA matched CBs as \>80% of patients received a 6-7/8 HLA matched CB. Interestingly there were patients with high-risk hematologic malignancies and multiple comorbidities (5 patients who had already failed an allogeneic transplant and 5 patients with refractory/relapsed acute leukemia/aggressive lymphoma). Despite this high risk population, progression was 20% at 12 months. This new study seeks to test a similar strategy in a group of patients with high risk acute leukemia/myelodysplasia.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 30 (actual)
- First submitted
- 23 Sept 2019
- Start date
- 13 Nov 2020
- Primary completion date
- 28 Feb 2026
- Lead sponsor
- ExCellThera inc.
- Diseases
- Myelodysplastic syndromes
- NCT number
- NCT04103879
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 30
- From 13 Nov 2020 to the primary completion date, 28 Feb 2026
- 3 sites in 2 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Allogeneic myeloablative transplant within 6 months.
- Autologous hematopoietic stem cell transplant within 6 months.
Other illness
- Active or recent invasive fungal infection.
- Presence of a malignancy other than the one for which the UCB transplant is being performed and the expected survival related to the malignancy is estimated to be less than 75% at 5 years.
- HIV positivity.
- Hepatitis B or C infection with measurable viral load.
Lab values
- Any abnormal condition or laboratory result that is considered by the principal investigator capable of altering patient condition or study outcome.
Other
- Liver cirrhosis.
- Pregnancy, breastfeeding or unwillingness to use appropriate contraception.
- Active central nervous system involvement.
- Chloroma \> 2 cm.
Who the study is looking for
- High and very high-risk hematologic malignancy defined as:
- Acute Myeloid Leukemia (Primary induction failure, Chemorefractory relapse, Relapse after allogeneic or autologous transplant, High risk AML in CR1, ≥ CR2)
- Acute Lymphoid leukemia (Primary induction failure, High risk ALL in CR1, ≥ CR2, Chemorefractory relapse, Relapse after allogeneic or autologous transplant)
- Myelodysplastic syndrome (Relapse after allogeneic or autologous transplant, ≥10% blasts within 30 days of start of conditioning regimen, Poor and very poor cytogenetics abnormalities, CMML with HCT-specific CPSS score high or intermediate-2, Stable disease, Progressive disease while on azacitidine).
- Chronic myelogenous leukemia (Patients who progressed to blast crisis)
- Availability of 2 CBs ≥ 4/6 HLA match with pre-freeze CD34+ cell count ≥0.5 x 10E5/kg and TNC≥1.5 x 10E7/kg
- Karnofsky ≥70.
- LVE fraction ≥ 40% or fractional shortening \>22%
- FVC, FEV1 and DLCOc ≥ 50% of predicted
- Bilirubin \< 2 x ULN; AST and ALT ≤ 2.5 x ULN; alkaline phosphatase ≤ 5 x ULN.
- Creatinine \< 2.0 mg/dl.
- HCT-CI ≤3 if patients have ≥5% blasts in the bone marrow and HCT-CI ≤5 if 60-65 years old.
Age in the registry: 18 years to 70 years · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Erasmus Medical Center Coordinator of this site? Confirm its status | Rotterdam | Netherlands | Unknown status |
| University of Colorado School of Medicine. Anschutz Medical Campus Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| Fred Hutchinson/University of Washington Cancer Consortium Coordinator of this site? Confirm its status | Seattle | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.