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High Risk Myeloid MalignanciesPhase 1/2NCT04990323

US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies

A Phase 1/2 study testing ECT-001-CB (UM171-Expanded Cord Blood Transplant) in people with High Risk Myeloid Malignancies and Cord Blood Transplant, run by ExCellThera inc. at 1 site in United States.

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 0 to 21 years
What is being tested?
ECT-001-CB (UM171-Expanded Cord Blood Transplant)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
1 siteUnited States
Study dates
1 Dec 2021to 31 Dec 2025
Phase
Phase 1/2
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 0 to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Acute Myeloid Leukemia
  • Chemo-refractory relapse (MRD+)
  • Primary induction failure (no CR or CRi after >= 2 courses of intensive induction therapy): < 30% blasts in evaluable marrow.
  • Relapse after previous allogeneic (or autologous) transplant (>4 months)
  • Secondary or therapy-related MDS/AML
  • Poor response to induction (5-30% blasts) or MDR+ after induction
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  • Myelodysplastic syndrome (MDS)
  • Relapse after allogeneic or autologous transplant (>4 months)
  • ≥10 % blasts within 30 days of start of conditioning regimen
  • Poor and very poor cytogenetics abnormalities
  • Chronic myelogenous leukemia: Patients who progressed to blast crisis
  • Mixed Phenotype Acute Leukemia: MRD+ or relapse after previous transplant (>4 months).
  • JMML (Juvenile Myelo-Monocytic Leukemia)
  • Availability of 2 ≥ 4/8 HLA matched CBU (allele level: A, B, C and DRB1)
  • Cord to be expanded: CD34+ cell count ≥ 0.5 x 10^5/kg and TNC ≥ 1.5 x 10^7/kg (pre-cryo)
  • 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.
  • Lansky / Karnofsky >60%
  • 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
  • Estimated or measured creatinine clearance ≥ 50ml/min/1.73m2
  • Left ventricular ejection fraction of ≥ 40%
  • FVC, FEV1 and DLCO ≥ 50% of predicted
  • Signed written informed consent
  • 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.

Who cannot join

  • Previous allogeneic transplantation within 4 months.
  • Uncontrolled infection.
  • 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
  • Seropositive for HIV.
  • Hep B and C infection with measurable viral load.
  • Liver cirrhosis.
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  • Active CNS disease.
  • Chloroma > 2cm.
  • >30% blasts in marrow in evaluable marrow sample.
  • Pregnancy, breastfeeding, or unwillingness to use appropriate contraception
  • Participation in a trial with an investigational agent within 30days prior to entry in the study.
  • Any abnormal condition or lab result that is considered by the PI capable or altering patient's condition or study outcome.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

Other trials in United States

About this study

Official title: A Phase I/II Open-Label Study of ECT-001-Expanded Cord Blood Transplantation in Pediatric and Young Adult (<21year) Patients With High-Risk and Very High-Risk Myeloid Malignancies

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 previous trials (NCT02668315, NCT03913026, NCT04103879, and NCT03441958), the CB expansion protocol using the ECT-001-CB technology (UM171 molecule) has proven to be technically feasible and safe in adults.

UM171 expanded CB was associated with a prompt (D+17), robust (98%) and durable neutrophil recovery. Amongst patients who received a single UM171 CB transplant with a median follow-up of 18 months, risk of TRM (10%), grade 3-4 acute GVHD (13%) and moderate-severe chronic GVHD (2%) was low at 1 year post-transplant. Incidence of severe viral and bacterial infections was reduced and immunosuppression could be discontinued in 77% of patients at 1 year. Thus, PFS and GRFS were very promising, 72% and 59% at 12 months, 69% and 53% at 24 months, respectively, in particular accounting for a large proportion of very high-risk patients. By a 10-fold increase of CB accessibility, ECT-001-CB allowed access to smaller, better HLA matched CBs.

This new study seeks to test a similar strategy in a group of pediatric and young adult patients with high risk myeloid malignancies. 12 patients will be enrolled in the first stage of this 2-stage design protocol. If intervention is considered promising (<= 3 relapses in the first 12 patients), this study will open multicenter and be extended to a second stage (16 additional patients for a total accrual 28).

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

    Also called Conditioning Regimen (Preferred): clofarabine:30 mg/m2/day IV x 4 days, fludarabine:10 mg/m2/day IV x 4 days, busulfan: daily for 4 days with cumm Bu target = 90mg h/L, Conditioning Regimen (Alternative): MIDI: fludarabine: 30 mg/m2/day IV x 5 days, cyclophosphamide: 50 mg/kg IV , thiotepa: 5 mg/kg/day IV x 2 days , TBI: 400 cGy, GVHD Prophylaxis: Tacrolimus/MMF

    Single UM171-Expanded CB transplant (CD34+: 2.5-50x10^5/kg, CD3+>1x10^6/kg)

Study groups in NCT04990323
Study groupRoleWhat they receive
ECT-001-Expanded CBExperimentalPatients will receive a myeloablative conditioning regimen (Preferred: Clo/Flu/Bu90, Alternative: MIDI) 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

    Incidence and severity of AEs according to the modified (for HSCT) CTCAE (v. 5.0)

    Measured: 100 days

  2. Relapse

    Incidence of relapse will be measured from time of transplant

    Measured: 1 year post-transplant

Secondary outcomes (7)

  1. Leukemia-free survival

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

    Measured: 1- and 2-year post-transplant

  2. Non-Relapse Mortality

    NRM 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: 1 year post-transplant

  3. GVHD

    Incidence of acute and chronic GVHD will be measured by NIH criteria

    Measured: 1- and 2-year post-transplant

  4. Grade 3 Infections

    Incidence and severity 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: 2-year post-transplant

  5. Hematologic engraftment

    Time to 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 time to platelet engraftment (first day of a sustained platelet count ≥ 50 x 10E9/L with no platelet transfusion in the preceding 7 days)

    Measured: 42 and 100 days

  6. Pre-engraftment/engraftment syndrome

    Incidence of pre-engraftment/engraftment syndrome requiring therapy

    Measured: 2-year post-transplant

  7. Hospitalization events

    Duration of transplant admission and number of days in hospital in 1st 100 days, and last day of fever (>38°C) prior to engraftment

    Measured: 100 days

Timeline

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

  1. First posted4 Aug 2021
  2. Study start1 Dec 2021
  3. Primary completion31 Dec 2025
  4. Study completion31 Dec 2025

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
13 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04990323
Sponsor's protocol ID
ECT-001-CB.007
Status last verified
Mar 2026
First submitted
27 Jul 2021
Last updated
24 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 1/2 study testing ECT-001-CB (UM171-Expanded Cord Blood Transplant) in people with High Risk Myeloid Malignancies and Cord Blood Transplant, run by ExCellThera inc. at 1 site in United States.

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 23 inclusion and 12 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 24 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 1 site in United States. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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). The study lists 2 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 NCT04990323.

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 24 Mar 2026.

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