Ignite Creation Date:
2025-12-24 @ 4:31 PM
Ignite Modification Date:
2025-12-26 @ 3:29 PM
Study NCT ID:
NCT07301866
Status:
RECRUITING
Last Update Posted:
2025-12-24
First Post:
2025-11-27
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study Testing an Improved Dose of UM171 to Help Make Cord Blood Transplants More Effective and Safe.
Sponsor:
Ciusss de L'Est de l'Île de Montréal
Organization:
Study Overview
Official Title:
Single-site, Prospective, Open-label Phase I-II Study on Transplantation Using Cord Blood Treated With an Optimized Dose of UM171: Optimized ECT-001-CB.
Status:
RECRUITING
Status Verified Date:
2025-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ECT-001-CB-013
Brief Summary:
This clinical study is testing a new way to improve stem cell transplants for adults with high-risk blood cancers, such as leukemia or myelodysplasia, who do not have a suitable donor. The transplant uses stem cells from umbilical cord blood that have been expanded in the lab using a molecule called UM171. Previous studies showed that UM171 helps these cells grow and work better, leading to faster blood count recovery and fewer complications.
In this study, researchers are testing whether increasing the dose of UM171 during the lab expansion process can make the transplant less toxic. The hypothesis is that using a higher dose of UM171 to expand cord blood stem cells will help patients recover blood counts faster after transplant by improving the growth and function of the cells. This may lead to better immune recovery, fewer infections, shorter hospital stays, and improved overall outcomes.
Only seven patients will be enrolled, and they will be followed for one year after their transplant.
In this study, researchers are testing whether increasing the dose of UM171 during the lab expansion process can make the transplant less toxic. The hypothesis is that using a higher dose of UM171 to expand cord blood stem cells will help patients recover blood counts faster after transplant by improving the growth and function of the cells. This may lead to better immune recovery, fewer infections, shorter hospital stays, and improved overall outcomes.
Only seven patients will be enrolled, and they will be followed for one year after their transplant.
Detailed Description:
This is a single-center, prospective, open-label, dose-escalating Phase I/II clinical trial designed to evaluate the safety, feasibility, and efficacy of ECT-001-CB cord blood transplantation using an optimized dose of UM171, a small molecule that enhances hematopoietic stem cell (HSC) expansion.
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for patients with high-risk hematologic malignancies. However, some patients lack a suitable donor. Cord blood (CB) is a valuable alternative donor source due to its tolerance for HLA mismatches and lower incidence of chronic graft-versus-host disease (GVHD). Despite these advantages, CB transplantation has declined due to limitations such as low cell dose, delayed engraftment, and increased non-relapse mortality (NRM).
UM171 is a proprietary molecule that has demonstrated the ability to expand CD34⁺ HSCs ex vivo, thereby addressing the cell dose limitation of CB transplants. Previous trials using UM171-expanded CB units (ECT-001-CB) showed promising results, including faster engraftment, improved immune reconstitution, and reduced NRM.
This study builds on prior findings by investigating whether a higher dose of UM171 (125nM vs. the conventional 35nM) can further accelerate neutrophil engraftment and enhance immune recovery. Preclinical data suggest that higher UM171 concentrations may increase the number of long-term repopulating HSCs and promote balanced lymphoid and myeloid differentiation, potentially improving clinical outcomes.
Seven patients with high-risk acute leukemia or myelodysplasia, or other hematologic malignancies requiring HSCT and lacking a suitable donor, will be enrolled over a 12-month period. Patients will receive a single ECT-001-CB transplant following an intermediate or high-intensity conditioning regimen. The CB unit will be expanded ex vivo with escalating doses of UM171 (starting at 70nM, potentially increasing to 125nM based on engraftment outcomes).
Primary endpoints include safety (grade ≥3 adverse events), feasibility of manufacturing and infusion, and time to neutrophil engraftment. Secondary endpoints include NRM, platelet engraftment, graft failure, progression-free survival (PFS), overall survival (OS), incidence of acute and chronic GVHD, immune reconstitution, and time to hospital discharge. Exploratory endpoints include infectious complications, relapse incidence, GVHD-free relapse-free survival (GRFS), advanced immunologic profiling, and comparisons with historical controls.
Safety will be monitored by an independent Data Safety Monitoring Board (DSMB). The study includes predefined stopping rules to ensure patient safety, including thresholds for engraftment delay, graft failure, NRM, and severe GVHD.
This trial aims to optimize CB transplantation by enhancing the efficacy of UM171-expanded grafts, potentially offering a safer and more effective treatment option for patients with limited donor availability.
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for patients with high-risk hematologic malignancies. However, some patients lack a suitable donor. Cord blood (CB) is a valuable alternative donor source due to its tolerance for HLA mismatches and lower incidence of chronic graft-versus-host disease (GVHD). Despite these advantages, CB transplantation has declined due to limitations such as low cell dose, delayed engraftment, and increased non-relapse mortality (NRM).
UM171 is a proprietary molecule that has demonstrated the ability to expand CD34⁺ HSCs ex vivo, thereby addressing the cell dose limitation of CB transplants. Previous trials using UM171-expanded CB units (ECT-001-CB) showed promising results, including faster engraftment, improved immune reconstitution, and reduced NRM.
This study builds on prior findings by investigating whether a higher dose of UM171 (125nM vs. the conventional 35nM) can further accelerate neutrophil engraftment and enhance immune recovery. Preclinical data suggest that higher UM171 concentrations may increase the number of long-term repopulating HSCs and promote balanced lymphoid and myeloid differentiation, potentially improving clinical outcomes.
Seven patients with high-risk acute leukemia or myelodysplasia, or other hematologic malignancies requiring HSCT and lacking a suitable donor, will be enrolled over a 12-month period. Patients will receive a single ECT-001-CB transplant following an intermediate or high-intensity conditioning regimen. The CB unit will be expanded ex vivo with escalating doses of UM171 (starting at 70nM, potentially increasing to 125nM based on engraftment outcomes).
Primary endpoints include safety (grade ≥3 adverse events), feasibility of manufacturing and infusion, and time to neutrophil engraftment. Secondary endpoints include NRM, platelet engraftment, graft failure, progression-free survival (PFS), overall survival (OS), incidence of acute and chronic GVHD, immune reconstitution, and time to hospital discharge. Exploratory endpoints include infectious complications, relapse incidence, GVHD-free relapse-free survival (GRFS), advanced immunologic profiling, and comparisons with historical controls.
Safety will be monitored by an independent Data Safety Monitoring Board (DSMB). The study includes predefined stopping rules to ensure patient safety, including thresholds for engraftment delay, graft failure, NRM, and severe GVHD.
This trial aims to optimize CB transplantation by enhancing the efficacy of UM171-expanded grafts, potentially offering a safer and more effective treatment option for patients with limited donor availability.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: