US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies
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|ClinicalTrials.gov Identifier: NCT04990323|
Recruitment Status : Recruiting
First Posted : August 4, 2021
Last Update Posted : January 13, 2022
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).
|Condition or disease||Intervention/treatment||Phase|
|High Risk Myeloid Malignancies Cord Blood Transplant||Biological: ECT-001-CB (UM171-Expanded Cord Blood Transplant)||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||December 1, 2021|
|Estimated Primary Completion Date :||April 1, 2023|
|Estimated Study Completion Date :||April 1, 2024|
Experimental: ECT-001-Expanded CB
Patients 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).
Biological: ECT-001-CB (UM171-Expanded Cord Blood Transplant)
Single UM171-Expanded CB transplant (CD34+: 2.5-50x10^5/kg, CD3+>1x10^6/kg)
- Adverse events of ECT-001-CB [ Time Frame: 100 days ]Incidence and severity of AEs according to the modified (for HSCT) CTCAE (v. 5.0)
- Relapse [ Time Frame: 1 year post-transplant ]Incidence of relapse will be measured from time of transplant
- Leukemia-free survival [ Time Frame: 1- and 2-year post-transplant ]LFS will be measured from time of transplant until disease relapse, death or last follow-up
- Non-Relapse Mortality [ Time Frame: 1 year post-transplant ]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
- GVHD [ Time Frame: 1- and 2-year post-transplant ]Incidence of acute and chronic GVHD will be measured by NIH criteria
- Grade 3 Infections [ Time Frame: 2-year post-transplant ]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
- Hematologic engraftment [ Time Frame: 42 and 100 days ]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)
- Pre-engraftment/engraftment syndrome [ Time Frame: 2-year post-transplant ]Incidence of pre-engraftment/engraftment syndrome requiring therapy
- Hospitalization events [ Time Frame: 100 days ]Duration of transplant admission and number of days in hospital in 1st 100 days, and last day of fever (>38°C) prior to engraftment
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04990323
|Contact: Jaap Jan Boelens, MD, PhDfirstname.lastname@example.org|
|Contact: Andromachi Scaradavou, MDemail@example.com|
|United States, New York|
|Memorial Sloan Kettering Cancer Center||Recruiting|
|New York, New York, United States, 10065|
|Contact: Andromachi Scaradavou, MD|
|Contact: Jaap J Boelens, MD|