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High Risk Hematological MalignancyPhase 2NCT04103879

US Study of UM171-Expanded CB in Patients With High Risk Leukemia/Myelodysplasia

A Phase 2 study testing ECT-001-CB (UM171-Expanded Cord Blood Transplant) in people with High Risk Hematological Malignancy and Cord Blood Transplant, run by ExCellThera inc. at 3 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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… Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
ECT-001-CB (UM171-Expanded Cord Blood Transplant)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
3 sitesin 2 countries
Study dates
13 Nov 2020to 28 Feb 2026
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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.

Who cannot join

  • Allogeneic myeloablative transplant within 6 months.
  • Autologous hematopoietic stem cell transplant within 6 months.
  • 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.
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  • Liver cirrhosis.
  • Pregnancy, breastfeeding or unwillingness to use appropriate contraception.
  • Any abnormal condition or laboratory result that is considered by the principal investigator capable of altering patient condition or study outcome.
  • Active central nervous system involvement.
  • Chloroma > 2 cm.

Trial locations and contacts

3 sites in 2 countries.

3 sites in 2 countries

United States2 sites

  • University of Colorado School of Medicine. Anschutz Medical Campus

    Aurora, Colorado, 80045

  • Fred Hutchinson / University of Washington Cancer Consortium

    Seattle, Washington, 98109

Other trials in United States

Netherlands1 site

  • Erasmus Medical Center

    Rotterdam, Gelderland, 3015

Other trials in Netherlands

About this study

Official title: A Phase II Open-Label Study of UM171-Expanded Cord Blood Transplantation in Patients With High and Very High Risk Acute Leukemia/Myelodysplasia

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.

Being tested
ECT-001-CB (UM171-Expanded Cord Blood Transplant)

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ECT-001-CB (UM171-Expanded Cord Blood Transplant)Biological

    Conditioning: High dose TBI (1320 cGy TBI + Fludarabine 75 mg/m2 + Cyclophosphamide 120 mg/kg) or Intermediate Intensity regimen (400 cGy TBI + Fludarabine 150 mg/m2 + Cyclophosphamide 50 mg/kg + Thiotepa 10 mg/kg).

    Single UM171-Expanded CB transplant (CD34+: 2.5-50x10E5/kg, CD3+>1x10E6/kg)

    Immunosuppression: Tacrolimus/MMF

Study groups in NCT04103879
Study groupRoleWhat they receive
ECT-001-Expanded CBExperimentalPatients will receive a myeloablative conditioning regimen. The cord to be expanded will undergo CD34+ selection. The CD34- product is cryopreserved and will be thawed and infused on Day +1 post-transplant. The CD34+ product will be placed in a closed culture with UM171 for a 7-day expansion and is infused on Day 0. Patients will receive standard supportive care and GVHD prophylaxis (such as MMF and tacrolimus).
Receives: Biological: ECT-001-CB (UM171-Expanded Cord Blood Transplant)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse events of ECT-001-CB

    All AEs will be graded in severity according to the modified (for HSCT) CTCAE (v. 5.0)

    Measured: 100 days post-transplant

  2. Adverse events of ECT-001-CB

    All AEs will be graded in severity according to the modified (for HSCT) CTCAE (v. 5.0)

    Measured: 2 years post-transplant

  3. Relapse-free survival

    RFS will be measured from time of transplant until disease relapse, death or last follow-up

    Measured: At 1-year post-transplant

  4. Relapse-free survival

    RFS will be measured from time of transplant until disease relapse, death or last follow-up

    Measured: At 2-year post-transplant

Secondary outcomes (8)

  1. Time to Neutrophil and Platelet engraftment

    Neutrophil engraftment (the first day of attainment of an absolute neutrophil count ≥0.5 x 10E9/L for 3 consecutive days. Time to ANC ≥ 0.1 x 10E9/L will also be documented) and platelet engraftment (first day of a sustained platelet count ≥ 20 x 10E9/L with no platelet transfusion in the preceding 7 days)

    Measured: First 60 days

  2. Incidence of transplant related mortality

    TRM is defined as any death of any cause other than malignant relapse, occurring after the commencement of conditioning regimen that could be related to the transplantation procedure

    Measured: At day 100 post-transplant

  3. Incidence of transplant related mortality

    TRM is defined as any death of any cause other than malignant relapse, occurring after the commencement of conditioning regimen that could be related to the transplantation procedure

    Measured: At 1-year post-transplant

  4. Incidence of GVHD

    Acute and chronic GVHD by NIH criteria

    Measured: At 2 years post-transplant

  5. Incidence of grade 3 or higher infectious complications

    Any of infections requiring systemic therapy, e.g., invasive candidiasis, aspergillus, other invasive fungi, CMV, adenovirus, EBV, HHV-6, HSV, VZV, PCP, toxoplasmosis and mycobacterium

    Measured: At 2 years post-transplant

  6. Incidence of pre-engraftment/engraftment syndrome requiring therapy

    Measured: At 2 years post-transplant

  7. GRFS and CRFS

    GRFS and CRFS will be measured from time of transplant until disease relapse, death or last follow-up

    Measured: At 1-year post-transplant

  8. GRFS and CRFS

    GRFS and CRFS will be measured from time of transplant until disease relapse, death or last follow-up

    Measured: At 2-year post-transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Sep 2019
  2. Study start13 Nov 2020
  3. Primary completion28 Feb 2026
  4. Study completion28 Feb 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04103879
Sponsor's protocol ID
ECT-001-CB.004
Other IDs
Study 8743
Status last verified
Mar 2026
First submitted
23 Sep 2019
Last updated
23 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ECT-001-CB (UM171-Expanded Cord Blood Transplant) in people with High Risk Hematological Malignancy and Cord Blood Transplant, run by ExCellThera inc. at 3 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 23 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse events of ECT-001-CB (time frame: 100 days post-transplant). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04103879.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2026.

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