Safety and Early Efficacy Study of TBX-2400 in Patients With AML or Myelofibrosis
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|ClinicalTrials.gov Identifier: NCT04709458|
Recruitment Status : Not yet recruiting
First Posted : January 14, 2021
Last Update Posted : January 12, 2022
This is a study of allogeneic stem cell transplantation with TBX-2400 in adult subjects with Acute Myelogenous Leukemia (AML) or Myelofibrosis (MF).
The donor cells are exposed to a protein that has been shown in the laboratory to improve the ability of the donor cells to make blood and immune cells after transplant. Exposure of the donor cells to this protein does not modify the genes in the cells in any way.
This study has two goals. The first goal is to find out if transplant with TBX-2400 is safe. The second goal is to find out what effects TBX-2400 stem cells have on time to engraftment in adult subjects with AML or MF.
The study hypothesis is that TBX-2400 cells will shorten the time to immune reconstitution after transplant.
|Condition or disease||Intervention/treatment||Phase|
|Myelofibrosis Acute Myelogenous Leukemia||Biological: TBX-2400||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Study to Assess the Safety and Early Efficacy of TBX-2400 in Enhancing Engraftment in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant for the Treatment of Acute Myelogenous Leukemia or Myelofibrosis|
|Estimated Study Start Date :||February 1, 2022|
|Estimated Primary Completion Date :||August 28, 2024|
|Estimated Study Completion Date :||October 28, 2024|
Experimental: TBX-2400 treatment
Single intravenous infusion of TBX-2400
Hematopoietic stem cells transplantation
- Incidence of adverse events according to NCI-CTCAE Version 5.0 [ Time Frame: Two years ]Adverse events from subject reporting or other assessments
- Transplant engraftment [ Time Frame: 1 year ]Assessment of transplant engraftment will include absolute neutrophil count, untransfused platelet count and donor chimerism
- Immune reconstitution as measured by CD3+ cell count [ Time Frame: Up to Day 360 ]Immune reconstitution as measured by CD3+ cell count > 300/μL
- Immunoglobulin (IgA) levels [ Time Frame: Up to Day 360 ]
- Immunoglobulin (IgM) levels [ Time Frame: Up to Day 360 ]
- T-cell Engraftment [ Time Frame: Up to Day 360 ]CD45 RA versus RO at 3, 6, 9 and 12 months
- Disease-free survival [ Time Frame: Two years ]
- Incidence of secondary graft failure [ Time Frame: Two years ]
- Transplant-Related Mortality (TRM) [ Time Frame: 100 Days ]
- Quality of life using the World Health Organisation Five Wellbeing Index [ Time Frame: Up to day 360 ]QoL assessed at 3, 6, 9 and 12 months (World Health Organization [WHO] Five Wellbeing Index).
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): NCT04709458
|Contact: Vivienne Margolis, B.Scemail@example.com|
|Contact: Yosef Refaeli, Ph.Dfirstname.lastname@example.org|
|University Hospital Centre Zagreb|
|Zagreb, Croatia, 10000|
|Contact: Nadira Durakovic, MD, PhD +385-989611829 email@example.com|
|Principal Investigator: Nadira Durakovic, MD, PhD|
|Fondazione Policlinico Universitario Agostino Gemelli|
|Roma, Italy, 00168|
|Contact: Simona Sica, MD TBA Simona.firstname.lastname@example.org|
|Principal Investigator: Simona Sica, MD|
|Principal Investigator:||Andrea Bacigalupo, MD||Unit for Hematology and BMT, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy|