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NCT04990323 · ClinicalTrials.gov registry record · Phase 1
US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies
A Phase 1 study of High Risk Myeloid Malignancies and Cord Blood Transplant, sponsored by ExCellThera.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 1
- Study location
NCT04990323 is a Phase 1 study of High Risk Myeloid Malignancies and Cord Blood Transplant that is actively recruiting participants, run by ExCellThera. The registered enrollment target is 12 participants. The trial reports 1 study location across 1 state.
The verdict
NCT04990323, a Phase 1 study of High Risk Myeloid Malignancies and Cord Blood Transplant, is actively recruiting participants, sponsored by ExCellThera.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 1
- Study location
Study 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 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).
Conditions Studied
Interventions
- BIOLOGICAL ECT-001-CB (UM171-Expanded Cord Blood Transplant)
Study Locations (1)
New York
- Memorial Sloan Kettering Cancer Center - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2021-12-01 |
| Est. Completion | 2027-06-01 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04990323
The ClinicalTrials.gov registry entry for NCT04990323 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is ExCellThera, which has 2 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with High Risk Myeloid Malignancies appearing as the primary indexed condition, and to 1 intervention - of which ECT-001-CB (UM171-Expanded Cord Blood Transplant) is the first listed.
NCT04990323 reports 1 study location spanning 1 distinct geographic area - top geographies include New York.
Frequently Asked Questions
What is clinical trial NCT04990323 about?
NCT04990323 is a clinical study titled "US Study of ECT-001-CB in Pediatric and Young Adult Patients With 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 ...
What is the current status of trial NCT04990323?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2021-12-01. Estimated completion is 2027-06-01.
What conditions does trial NCT04990323 study?
This clinical trial studies the following conditions: High Risk Myeloid Malignancies, Cord Blood Transplant.
What interventions are being tested in trial NCT04990323?
The interventions under investigation include: ECT-001-CB (UM171-Expanded Cord Blood Transplant) (BIOLOGICAL).
Who is sponsoring clinical trial NCT04990323?
This trial is sponsored by ExCellThera, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04990323 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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