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Multiple MyelomaPhase 1/2NCT03441958

ECT-001 (UM171) Expanded Cord Blood Transplant to Treat High-risk Multiple Myeloma

A Phase 1/2 study testing ECT-001 (UM171) expanded cord blood in people with Multiple Myeloma, run by Ciusss de L'Est de l'Île de Montréal at 1 site in Canada.

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

Multiple Myeloma (MM) is a morbid disease associated with a poor outcome and while current therapies with new drugs have improved survival, MM still remains incurable in most patients. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Multiple Myeloma
What is being tested?
ECT-001 (UM171) expanded cord blood
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
1 siteCanada
Study dates
7 Mar 2018to 17 Oct 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 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age 18-65 years.
  • Newly diagnosed multiple myeloma using the International Myeloma Working Group criteria with measurable disease and any of the following:
  • t(4;14), t(14;16), t(14;20), del(17p13), chromosome 1 abnormalities with ISS II or III; ii. Revised-ISS 3; iii. Primary plasma cell leukemia; iv. Refractory to first line triplet Bortezomib-based induction treatment. v. ≥ 2 cytogenetics abnormalities as defined above regardless of ISS stage
  • Received a first line triplet Bortezomib-induction regimen for a minimum of 4 cycles with achievement of at least partial response; or received a doublet or triplet Lenalidomide-based second line induction treatment with at least partial response for patients refractory to Bortezomib in first line.
  • Received high-dose Melphalan ≥ 140 mg/m2 followed by ASCT.
  • Availability of a cord blood with an HLA match ≥ 5/8 and < 8/8 meeting the following requirements: CD34+ cell count ≥ 0.5 x 105/kg and nucleated cell count >= 1.5 x 107/kg.

Who cannot join

  • Having previously received two ASCT.
  • Having previously received autologous-allogeneic tandem transplantation.
  • Having received more than 4 months of maintenance with Lenalidomide or Bortezomib after ASCT.
  • Poor organ function defined as either: forced vital capacity, forced expiratory volume in 1 second or lung diffusing capacity of carbon monoxide corrected for hemoglobin < 50%, left ventricular ejection fraction < 40% (evaluated by either echocardiogram or MUGA), uncontrolled arrhythmia or symptomatic cardiac disease, creatinine clearance < 60 mL/minute.
  • Karnofsky score < 70% or comorbidity index HCT-CI > 3.
  • Bilirubin > 2 x upper limit of normal (ULN) unless felt to be related to Gilbert's disease or hemolysis; AST and ALT > 2.5 x ULN; alkaline phosphatase > 5 x ULN; liver cirrhosis.
Show all 17Show fewer
  • Non secretory disease or non-measurable disease in serum or urine at time of diagnosis.
  • Uncontrolled infection.
  • Active infection with any of the following viruses: HIV, HTLV-1 or 2, hepatitis B or C.
  • Presence of another malignancy with an expected survival estimated < 75% at 5 years.
  • Suspicion of cardiac amyloidosis.
  • Current history of drug and/or alcohol abuse.
  • Availability of a matched sibling donor.
  • Pregnancy, breastfeeding or unwillingness to use appropriate contraception.
  • Participation in a trial with an investigational agent within 30 days prior to entry in the study.
  • Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up and tests.
  • Any abnormal condition or laboratory result that is considered by the principal investigator capable of altering patient's condition or study outcome.

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • CIUSSS de l'Est-de-l'île-de-Montréal, Installation Hôpital Maisonneuve Rosemond

    Montreal, Quebec, H1T2M4

Other trials in Canada

About this study

Official title: A Phase I-II Open-label Study of Reduced Intensity-allogeneic Transplant of ECT-001 (UM171/ Fed-batch Culture System) Expanded Cord Blood in Patients With High-risk Multiple Myeloma

Multiple Myeloma (MM) is a morbid disease associated with a poor outcome and while current therapies with new drugs have improved survival, MM still remains incurable in most patients. The only potential curative treatment remains allogeneic Hematopoietic stem cell transplant (HSCT), as shown by our cohort of 92 newly diagnosed patients who received a sibling tandem auto-allo (HSCT) with an estimated 10-year progression free survival (PFS) of 43%. However, the high incidences of toxicities including chronic graft-versus-host-disease (GVHD) (up to 79%) and disease progression (up to 49%) impair improvement in cure rate. Using umbilical cord blood (CB) as an alternative source of hematopoietic stem cells (HSC) could be superior biologically because of their increased proliferative capacity, greater number of progeny with longer telomeres and better anti-tumor efficacy in presence of positive residual disease. Moreover, using CB has been shown to decrease incidence of chronic GVHD. However, CBs have the disadvantage of having a limited HSC dose leading to prolonged cytopenia and higher risk of infections.

In a first in-human trial using CB expanded with the ECT-001 (UM171) molecule (clinicaltrial.gov # NCT02668315), the median net expansion of HSC was 36 fold, which allows for the selection of better HLA matched CB regardless of their lower HSC dose. Moreover, the ECT-001 expanded CBs have a different cell composition than regular CBs, with more than 25% of dendritic cell precursors. This, combined to better HLA matched CBs, may reduce chronic GVHD incidence and improve immune reconstitution. To date, 22 patients received an ECT-001 expanded CB and the procedure proved to be safe and feasible.

In this new trial, the goal is to evaluate the safety and efficacy of ECT-001 expanded CB transplant in high risk MM patients.

Read the detailed description

This is a single institution, prospective, phase I/II open-label study in a maximum of 20 patients evaluating a novel treatment strategy in NDMM patients with high-risk disease who do not have a 6/6 compatible sibling donor. Participating patients will be from Hôpital Maisonneuve-Rosemont (HMR) or referred to HMR for this protocol. Newly diagnosed multiple myeloma patients will be evaluated for eligibility before or during the autologous stem cell transplant (ASCT) period. After a Bortezomib-based induction treatment (VTD, CyBorD, RVD or PAD [in patients with plasma cell leukemia]) for a minimum of 4 cycles, followed by Melphalan ≥ 140 mg/m2 and ASCT, eligible patients who accept to participate will undergo screening evaluation to receive a Reduced Intensity (RIC) allogeneic HSCT with ECT-001 expanded CB. It is estimated that 18 months will be necessary to enroll the targeted sample size.

Once eligibility has been confirmed, study treatment will begin. After an ASCT, eligible patients will receive a conditioning regimen before receiving a RIC allogeneic HSCT with an ECT-001 expanded CB on day 0. Patients will be followed at least every week for the first 3 months, then every month, in the absence of GVHD, for disease evaluation and adverse events. Occurrence and severity of acute GVHD will be evaluated using the modified Glucksberg176 and IBMTR177 criteria, while chronic GVHD will be evaluated using the NIH178 criteria.

The trial will be terminated when all patients have been followed for 5 years after allogeneic HSCT.

Being tested
ECT-001 (UM171) expanded cord blood
Keywords
hematopoietic stem cell transplant, Expanded cord blood
Medical subject headings
Multiple Myeloma

What participants receive

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

  • ECT-001 (UM171) expanded cord bloodBiological

    ECT-001 expanded cord-blood will be produced and infused on site

Study groups in NCT03441958
Study groupRoleWhat they receive
ECT-001 (UM171) expanded cord bloodExperimental1. Patients will receive a reduced intensity conditioning regimen containing Cyclophosphamide 50 mg/kg, Fludarabine 40 mg/m2 x 5 days and total body irradiation 200 cGy. 2. The cord to be expanded is thawed 7 days prior to transplant and undergoes CD34+ selection. The CD34+ product will be placed in the fed-batch culture with UM171 for a 7-day expansion and is infused fresh on Day 0. The CD34- product is cryopreserved and will be thawed and infused on Day +1. 3. Patients will receive standard supportive care and GVHD prophylaxis with Mycophenolate mofetil and Tacrolimus.
Receives: Biological: ECT-001 (UM171) expanded cord blood

What is being measured

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

  1. Safety of ECT-001 expanded CB expansion as measured by toxicity evaluation

    AEs with a CTCAE grade ≥ 3 (non hematologic) and with a grade ≥ 4 (hematologic) will be reported from the beginning of the conditioning regimen up to 5 years after CB transplant.

    Measured: 5 years

  2. Feasibility of ECT-001 expanded CB expansion

    Number of successful expansion and infusions in an outpatient nonmyeloablative transplant condition for high-risk myeloma patients

    Measured: 5 years

  3. Measure of the kinetics of donor lymphoid cells recovery

    Donor lymphocytes cells recovery assessed by chimerism analysis.

    Measured: 2 years

  4. Measure of the kinetics of donor myeloid cells recovery

    Time to neutrophils and platelets engraftment will be measured

    Measured: 2 years

  5. Incidence of chronic GVHD by grade at 1 years by NIH criteria.

    The incidence of chronic GVHD will be evaluated at 1 years using the recommendations of the NIH Consensus Conference and recently updated. Analysis by cumulative incidence

    Measured: 1 year

  6. Incidence of chronic GVHD by grade at 2 years by NIH criteria.

    The incidence of chronic GVHD will be evaluated at 1 years using the recommendations of the NIH Consensus Conference and recently updated. Analysis by cumulative incidence

    Measured: 2 years

Secondary outcomes (25)

  1. Correlation between neutrophil and CD34+ doses infused

    Regression analysis

    Measured: 2 years

  2. Correlation between neutrophil and CD34+CD45RA+ doses infused

    Regression analysis. Time to neutrophil and platelet engraftment will be correlated to the CD34+ and the CD34+CD45RA- dose (which includes all human hematopoietic stem cells (HSCs): long term and short term repopulating cells) contained in the expanded graft. Expansion and cultures might change the characteristics and behaviour of CD34+ cells, hence the need to look at the correlation between primitive CD34+CD45RA- subpopulation and engraftment.

    Measured: 2 years

  3. Incidence of graft failure

    Cumulative incidence of graft failure by type (primary or secondary). For Primary engraftment failure will be defined as non-achieving a T lymphocyte chimerism of ≥30% at D+28 and ≥70% at D+180. Secondary graft failure will be defined as followed : ANC drops below 0.5x109/L for 14 consecutive days, unresponsive to G-CSF without any identifiable cause (medication, viral infection, vitamin deficiency or other) or <5% donor chimerism at any time point beyond initial engraftment in the absence of relapse and graft dominance by 2nd infused cord or other stem cell source.

    Measured: 2 years

  4. Evaluation of T Cells reconstitution

    Evaluation at several levels :

    • Multiparametric flow cytometry to quantify the proportion of naïve (CD45RA+/CD27+) and memory (CD45RA-/CD27+) cells.
    • TREC to measure thymic output
    • Diversity of the T cell repertoire (deep sequencing)
    • T-cell function (Elispot assays)

    Measured: 3 years

  5. Evaluation of B cells reconstitution

    B cell evaluation will be carried out prospectively using flow cytometry (CD19+), immunoglobulin (Ig) measurements, and PCR for donor B cell chimerism .

    Measured: 3 years

  6. Evaluation of NK Cells reconstitution

    NK cell evaluation will be performed by flow cytometry (CD 16+/56+) and chimerism analysis.

    Measured: 3 years

  7. Evaluation of expanded HSC activity in vivo

    Standard in vitro (long term culture-initiating cells and colony forming cells) and in vivo (the NSG mouse model) assays

    Measured: 3 years

  8. Incidence of acute GVHD at day +120

    Analysis by cumulative incidence

    Measured: 4 months

  9. Incidence of acute GVHD at 6 month

    Analysis by cumulative incidence

    Measured: 6 months

  10. Incidence of acute GVHD at 1 year

    Analysis by cumulative incidence

    Measured: 1 year

  11. Incidence of grade >=3 infectious complications

    Analysis by cumulative incidence

    Measured: 5 years

  12. Incidence of engraftment syndrome requiring therapy

    Analysis by cumulative incidence

    Measured: 2 years

  13. Duration of hospitalization

    Number of days of hospitalization during the first 180 days post-transplant

    Measured: 6 months

  14. Non relapse mortality at day +120

    Analysis by cumulative incidence

    Measured: 4 months

  15. Non relapse mortality at 1 year

    Analysis by cumulative incidence

    Measured: 1 year

  16. Non relapse mortality at 2 year

    Analysis by cumulative incidence

    Measured: 2 years

  17. Progression free survival at 2 years

    Kaplan Meier analysis

    Measured: 2 years

  18. Overall survival at 2 years

    Kaplan Meier analysis

    Measured: 2 years

  19. Response to treatment at 1 year after allogeneic transplant

    Evaluation of response categories according to the International Myeloma Working Group (IMWG)

    Measured: 1 years

  20. Response to treatment at 2 year after allogeneic transplant

    Evaluation of response categories according to the International Myeloma Working Group (IMWG)

    Measured: 2 years

  21. Best response achieve at 1 year after allogeneic transplant

    Evaluation of the best response during the 1 st year post-transplant

    Measured: 1 years

  22. Best response achieve at 2 year after allogeneic transplant

    Evaluation of the best response during the 2 years post-transplant

    Measured: 2 years

  23. Minimal residual disease post transplant

    Next Generation flow cytometry to determine how efficient an expanded CB is at reducing MM burden

    Measured: 5 years

  24. Patient's quality of life

    Assessment through Quality of Life questionnaires

    Measured: 5 years

  25. Pharmaco-economic evaluation of the proposed treatment

    Developement of an analysis model to determine if ECT-001 expanded CB to treat MM has a better cost-efficiency ratio than conventional chemotherapy-based treatment

    Measured: 5 years

Timeline

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

  1. First posted22 Feb 2018
  2. Study start7 Mar 2018
  3. Primary completion28 Oct 2023
  4. Study completion17 Oct 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
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03441958
Sponsor's protocol ID
HMR007
Status last verified
Mar 2026
First submitted
2 Feb 2018
Last updated
18 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Roy J, Cohen S, Sauvageau G, Ahmad I, Fournier V, Terra R, Caudrelier P, Thiant S, Thauvette G, Bambace N, Delisle JS, Lachance S, Kiss T, Bernard L, Roy DC, Veilleux O, LeBlanc R. A Pilot Study of UM171-Expanded Cord Blood Grafts for Tandem Auto/Allogeneic Hematopoietic Cell Transplant in High and Ultra-High-Risk Myeloma Patients. Transplant Cell Ther. 2025 Jan;31(1):34.e1-34.e14. doi: 10.1016/j.jtct.2024.10.008. Epub 2024 Oct 16.

    PubMed 39419177

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing ECT-001 (UM171) expanded cord blood in people with Multiple Myeloma, run by Ciusss de L'Est de l'Île de Montréal at 1 site in Canada.

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 6 inclusion and 17 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 18 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 Canada. 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?

Safety of ECT-001 expanded CB expansion as measured by toxicity evaluation (time frame: 5 years). The study lists 6 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 NCT03441958, which also lists 1 linked publication.

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 18 Mar 2026.

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