MyelofibrosisPhase 1NCT04709458
Safety and Early Efficacy Study of TBX-2400 in Patients With AML or Myelofibrosis
A Phase 1 study testing TBX-2400 in people with Myelofibrosis and Acute Myelogenous Leukemia, run by Taiga Biotechnologies, Inc. at 2 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis, Acute Myelogenous Leukemia
- What is being tested?
- TBX-2400
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 1 Sep 2022to 28 Oct 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
- For MF: primary MF or MF that has progressed from a myeloproliferative disease. Dynamic International Prognostic Scoring System (DIPSS)-plus to be utilized to support the inclusion of MF subjects at screening;
- 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;
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- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
Croatia1 site
University Hospital Centre Zagreb
Zagreb, 10000
Nadira Durakovic, MD, PhD, +385-989611829, nadira.durakovic@mef.hr
Nadira Durakovic, MD, PhD
Italy1 site
Fondazione Policlinico Universitario Agostino Gemelli
Roma, 00168
Simona Sica, MD, TBA, Simona.sica@unicatt.it
Simona Sica, MD
About this study
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
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.
Read more
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.
- Conditions
- Myelofibrosis, Acute Myelogenous Leukemia
- Being tested
- TBX-2400
- Keywords
- Myelofibrosis, Leukemia, Allogeneic stem cell transplant, Acute Myelogenous Leukemia, Myeloproliferative disorders, Primary myelofibrosis, Bone Marrow Cancer, Bone Marrow Transplant, Acute Myeloid Leukemia
- Medical subject headings
- Primary Myelofibrosis, Leukemia, Myeloid, Acute, Leukemia, Myeloproliferative Disorders, Bone Marrow Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TBX-2400Biological
Hematopoietic stem cells transplantation
| Study group | Role | What they receive |
|---|---|---|
| TBX-2400 treatment | Experimental | Single intravenous infusion of TBX-2400 Receives: Biological: TBX-2400 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events according to NCI-CTCAE Version 5.0
Adverse events from subject reporting or other assessments
Measured: Two years
Transplant engraftment
Assessment of transplant engraftment will include absolute neutrophil count, untransfused platelet count and donor chimerism
Measured: 1 year
Secondary outcomes (8)
Immune reconstitution as measured by CD3+ cell count
Immune reconstitution as measured by CD3+ cell count > 300/μL
Measured: Up to Day 360
Immunoglobulin (IgA) levels
Measured: Up to Day 360
Immunoglobulin (IgM) levels
Measured: Up to Day 360
T-cell Engraftment
CD45 RA versus RO at 3, 6, 9 and 12 months
Measured: Up to Day 360
Disease-free survival
Measured: Two years
Incidence of secondary graft failure
Measured: Two years
Transplant-Related Mortality (TRM)
Measured: 100 Days
Quality of life using the World Health Organisation Five Wellbeing Index
QoL assessed at 3, 6, 9 and 12 months (World Health Organization [WHO] Five Wellbeing Index).
Measured: Up to day 360
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2021
- Study start1 Sep 2022 (estimated)
- Primary completion28 Aug 2024 (estimated)
- Study completion28 Oct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyelofibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Taiga Biotechnologies, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Andrea Bacigalupo, MD, Principal investigator, Unit for Hematology and BMT, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04709458
- Sponsor's protocol ID
- TBX-2400-001
- Status last verified
- May 2022
- First submitted
- 12 Jan 2021
- Last updated
- 3 May 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04709458
- Acronym / sponsor ID
- TBX-2400-001
- Conditions
- Myelofibrosis, Acute Myelogenous Leukemia
- Keywords
- Myelofibrosis, Leukemia, Allogeneic stem cell transplant, Acute Myelogenous Leukemia, Myeloproliferative disorders, Primary myelofibrosis, Bone Marrow Cancer, Bone Marrow Transplant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Taiga Biotechnologies, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2022 (estimated)
- Primary completion
- 28 Aug 2024 (estimated)
- Study completion
- 28 Oct 2024 (estimated)
- First posted
- 14 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TBX-2400 treatment | Experimental | Biological: TBX-2400Single intravenous infusion of TBX-2400 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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;
- For MF: primary MF or MF that has progressed from a myeloproliferative disease. Dynamic International Prognostic Scoring System (DIPSS)-plus to be utilized to support the inclusion of MF subjects at screening;
- 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);
Key exclusion criteria (11 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TBX-2400Hematopoietic stem cells transplantation | Biological | TBX-2400 treatment |
Primary outcome
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
8 secondary outcomes
Immune reconstitution as measured by CD3+ cell count
Time frame: Up to Day 360
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
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrea Bacigalupo, MD principal investigator
Unit for Hematology and BMT, Fondazione Policlinico Universitario Agostino Gemelli, Rome, Italy
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TBX-2400 in people with Myelofibrosis and Acute Myelogenous Leukemia, run by Taiga Biotechnologies, Inc. at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Croatia, Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Vivienne Margolis, B.Sc (+972-4639634, vmargolis@taigabiotech.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events according to NCI-CTCAE Version 5.0 (time frame: Two years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 May 2022.