NCT04709458

Brief Summary

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.

Trial Health

47
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Sep 2022

Typical duration for phase_1

Geographic Reach
2 countries

2 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 12, 2021

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 14, 2021

Completed
1.6 years until next milestone

Study Start

First participant enrolled

September 1, 2022

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 28, 2024

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 28, 2024

Completed
Last Updated

May 3, 2022

Status Verified

May 1, 2022

Enrollment Period

2 years

First QC Date

January 12, 2021

Last Update Submit

May 1, 2022

Conditions

MyelofibrosisAcute Myelogenous Leukemia

Keywords

MyelofibrosisLeukemiaAllogeneic stem cell transplantAcute Myelogenous LeukemiaMyeloproliferative disordersPrimary myelofibrosisBone Marrow CancerBone Marrow TransplantAcute Myeloid Leukemia

Outcome Measures

Primary Outcomes (2)

  • Incidence of adverse events according to NCI-CTCAE Version 5.0

    Adverse events from subject reporting or other assessments

    Two years

  • Transplant engraftment

    Assessment of transplant engraftment will include absolute neutrophil count, untransfused platelet count and donor chimerism

    1 year

Secondary Outcomes (8)

  • Immune reconstitution as measured by CD3+ cell count

    Up to Day 360

  • Immunoglobulin (IgA) levels

    Up to Day 360

  • Immunoglobulin (IgM) levels

    Up to Day 360

  • T-cell Engraftment

    Up to Day 360

  • Disease-free survival

    Two years

  • +3 more secondary outcomes

Study Arms (1)

TBX-2400 treatment

EXPERIMENTAL

Single intravenous infusion of TBX-2400

Biological: TBX-2400

Interventions

TBX-2400BIOLOGICAL

Hematopoietic stem cells transplantation

TBX-2400 treatment

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • For crAML: AML with ≥5% blasts (either by morphology or by multi-parameter flow cytometry \[MFC\]) in a marrow aspirate obtained within 21 days of enrollment in the trial, and following the administration of at least two prior courses of chemotherapy;
  • Subject undergoing allogeneic stem cell transplantation on the decision of transplanting physician;
  • Signed informed consent of donor and recipient;
  • Subjects of ≥ 18 years of age (no upper age limit);
  • Donor agrees to donate bone marrow-derived or mobilized peripheral blood stem cells;
  • Subjects with Eastern Cooperative Oncology Group (ECOG) Performance Status 0 to 2;
  • Adequate pulmonary function with Diffusing Capacity for Carbon Monoxide (DLCO) \> 50;
  • Women of childbearing potential (WOCBP) must agree to follow instructions for method(s) of contraception (e.g. sterilization, hormone implants, hormone injections, intrauterine devices, or vasectomized partner with combined use of condom and/or birth control pills) from screening until 6 months after TBX-2400 transplantation;
  • Able to adhere to all trial treatments and procedures.

You may not qualify if:

  • Previous stem cell transplantation;
  • For MF: Blasts \> 10% in a marrow aspirate obtained within 30 days of screening;
  • Renal function: serum creatinine \> 1.5 x Upper Limit of Normal (ULN);
  • Hepatic function: impaired synthetic function as indicated by a serum fibrinogen below the normal limit. Aspartate transaminase/alanine transaminase (AST/ALT) \> 3.0 x ULN. Bilirubin, \> 1.5 x ULN;
  • Cardiac function: ejection fraction \< 45% as determined by echocardiography;
  • Prior malignancy active within previous three years - except for locally curable cancers such as cutaneous basal or squamous cell cancer, superficial bladder cancer, carcinoma in situ of cervix or breast, or carcinoma in situ of the prostate;
  • Positive pregnancy test or breastfeeding for women of childbearing age;
  • Serologic evidence of chronic Hepatitis B virus infection or Hepatitis C exposure;
  • Known history of positive test for Human Immunodeficiency Virus (HIV) or known Acquired Immunodeficiency Syndrome (AIDS);
  • Hypersensitivity to any trial medication (including the preparative regimen, TBX-2400 treatment and any prophylaxis or other medication planned);
  • Presence of a serious or uncontrolled medical disorder that, in the opinion of the Investigator, may increase the risk associated with trial participation or trial drug administration, impair the ability of the participant to receive protocol therapy, or interfere with interpretation of trial results.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

University Hospital Centre Zagreb

Zagreb, 10000, Croatia

Location

Fondazione Policlinico Universitario Agostino Gemelli

Roma, 00168, Italy

Location

MeSH Terms

Conditions

Primary MyelofibrosisLeukemia, Myeloid, AcuteLeukemiaMyeloproliferative DisordersBone Marrow Neoplasms

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesLeukemia, MyeloidNeoplasms by Histologic TypeNeoplasmsHematologic NeoplasmsNeoplasms by Site

Study Officials

  • Andrea Bacigalupo, MD

    Unit for Hematology and BMT, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Vivienne Margolis, B.Sc

CONTACT

+972-4639634vmargolis@taigabiotech.com

Yosef Refaeli, Ph.D

CONTACT

+1-720-859-3547refaeli@taigabiotech.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 12, 2021

First Posted

January 14, 2021

Study Start

September 1, 2022

Primary Completion

August 28, 2024

Study Completion

October 28, 2024

Last Updated

May 3, 2022

Record last verified: 2022-05

Data Sharing

IPD Sharing
Will not share

Locations

IT(1)CR(1)