Expanded Cord Blood Transplant for Multiple Myeloma

Recruiting in Palo Alto (17 mi)

Overseen byJean Roy, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Ciusss de L'Est de l'Île de Montréal

No Placebo Group

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for adults aged 18-65 with newly diagnosed high-risk multiple myeloma who've had specific initial treatments and responded at least partially. They must have undergone a stem cell transplant using Melphalan, not received more than two transplants or certain maintenance drugs for long, and have a suitable cord blood match.

Inclusion Criteria

I am between 18 and 65 years old.

I have a cord blood match that meets specific health standards.

I have been newly diagnosed with multiple myeloma with specific genetic features.

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Exclusion Criteria

I have had both autologous and allogeneic stem cell transplants.

My heart, lungs, kidneys, or liver are not working well.

My health severely limits my daily activities or I have several serious health issues.

See 12 more

Treatment Details

Interventions

ECT-001 (UM171) expanded cord blood (Stem Cell Transplant)

Trial OverviewThe trial tests ECT-001 expanded cord blood transplant's safety and effectiveness in treating high-risk multiple myeloma patients. It aims to improve survival rates by potentially reducing chronic graft-versus-host disease and enhancing immune recovery compared to traditional methods.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: ECT-001 (UM171) expanded cord bloodExperimental Treatment1 Intervention

1. 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.