Hematologic MalignancyPhase 1NCT06462365
Prevention of GvHD in Participants With Hematological Malignancies Undergoing Hematopoietic Stem Cell Transplant (HSCT)
A Phase 1 study testing TRX103 in people with Hematologic Malignancy and GvHD, run by Tr1X, Inc. at 5 sites in United States.
Trial summary
The purpose of this Phase 1, first in human open-label study is to assess the safety and tolerability of TRX-103 in patients with hematological malignancies undergoing HLA-mismatched related or unrelated hematopoietic stem cell transplantation (HSCT). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Malignancy, GvHD, GVHD,Acute
- What is being tested?
- TRX103
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 5 sitesUnited States
- Study dates
- 8 Apr 2024to 15 Apr 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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
- Subjects with one of the following hematologic malignancies: Acute Lymphoblastic Leukemia (B- or T-ALL), Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), or Chronic myelomonocytic leukemia (CMML)
- Males and Females Age ≥ 18 years.
- Weight of ≥ 35 Kg.
- Karnofsky performance status ≥ 70 %.
- Available mismatched related (haploidentical) or unrelated donors for peripheral blood stem cell (PBSC) donation.
- Subjects must otherwise fulfill institutional criteria for eligibility to undergo allogeneic stem cell transplantation.
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- Absence of uncontrolled bacterial, viral or fungal infection at time of enrollment.
- Have adequate organ function.
- Subjects > 65-year-old receiving MAC conditioning will only be eligible if they have a HSCT-comorbidity index score < 5.
- Subjects must be able to understand and sign informed consent and be willing and able to complete all specified procedures and visits.
Who cannot join
- Prior allogeneic bone marrow, peripheral blood, or cord blood HSCT.
- Any subject with a history of significant renal, hepatic, pulmonary, or cardiac dysfunction, or on treatment to support cardiac dysfunction.
- HIV positive.
- Positive hepatitis-B surface antigen. Subject may be included if they are HBV PCR negative.
- Positive hepatitis-C antibody with positive Recombinant Immunoblot Assay (RIBA) or PCR unless the subject has received curative anti-viral treatment and confirmed negative viral load by PCR.
- Received another investigational agent for treatment of disease understudy within 28 days (or 5 half-lives, whichever is shorter) of conditioning and/or have not recovered from treatment related toxicities.
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- Subjects with a previous history of Thrombotic Thrombocytopenic Purpura (TTP) or Hemolytic Uremic Syndrome (HUS) who are not good candidates for treatment with sirolimus.
- Subjects that are pregnant, breast feeding or aim to become pregnant during the study period. (Subjects must agree to use a highly effective method of contraception).
- Any serious illness, uncontrolled inter-current illness, psychiatric illness, active or uncontrolled infection, or other medical condition or history, including laboratory results.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
John Koreth, MD, 617-632-5138, John_Koreth@dfci.harvard.edu
- Recruiting
City of Hope
Duarte, California, 91010
(800)826-4673
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
Mark Juckett, MD, 612-676-4200, ccinfo@umn.edu
- Recruiting
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Miguel-Angel Perales, MD, 646-608-3757, peralesm@MSKCC.org
- Recruiting
Fred Hutchinson Cancer Center
Seattle, Washington, 98109
Filippo Milano, MD, 206-667-5925, fmilano@fredhutch.org
About this study
Official title: Phase I, First in Human, Open Label Study to Evaluate Safety and Tolerability of TRX103 Cells in Subjects With Hematological Malignancies Undergoing HLA-mismatched Related or Unrelated Hematopoietic Stem Cell Transplantation (HSCT)
The purpose of this Phase 1, first in human open-label study is to assess the safety and tolerability of TRX-103 in patients with hematological malignancies undergoing HLA-mismatched related or unrelated hematopoietic stem cell transplantation (HSCT). It is anticipated that up to 36 Subjects will be enrolled during a 18-24 month enrollment period. TRX-103 will be infused one time post HSCT.
- Conditions
- Hematologic Malignancy, GvHD, GVHD,Acute, GVHD, Chronic, Hematopoietic Stem Cell Transplant, Acute Lymphoblastic Leukemia, Adult B-Cell
- Being tested
- TRX103
- Keywords
- AML, CMML, Stem Cell Transplant, B-ALL, T-ALL, Acute GvHD, Graft versus Host Disease, Chronic GvHD, MDS, Cellular Therapy, Autoimmune, T regulatory cells
- Medical subject headings
- Hematologic Neoplasms, Bronchiolitis Obliterans Syndrome, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TRX103Biological
TRX103 infusion via central line.
| Study group | Role | What they receive |
|---|---|---|
| TRX-103 | Experimental | TRX-103 will be infused on time post hematopoietic stem cell transplant (HSCT). Receives: Biological: TRX103 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of TRX103 cell infusion through incidence of Adverse events.
Measured: Up to a year
Safety of TRX103 determined by stem cell engraftment and donor chimerism after HSCT measured by absolute neutrophil counts and percent donor chimerism.
Measured: Up to day 42
Safety of TRX103 determined by negative Replication Competent Lentivirus (RCL).
Measured: At 3-month, 6-month, and 1-year.
Secondary outcomes (4)
Incidence of Grade II-IV acute GvHD (aGvHD).
Measured: Day 0 to Day +100 day
Incidence of Grade III-IV acute GvHD (aGvHD).
Measured: Day 0 to Day +100 day
Incidence and severity of chronic GvHD (cGvHD)
Measured: Day +100 through Day +365
Overall survival at Day +365.
Measured: Up to a year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2024
- Study start8 Apr 2024
- Primary completion15 Apr 2026 (estimated)
- Study completion15 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematologic MalignancyStudies 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
- Prevention
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tr1X, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Maria Grazia Roncarolo, MD, Study director, Tr1X, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06462365
- Sponsor's protocol ID
- TRX103-01
- Status last verified
- Oct 2024
- First submitted
- 6 Jun 2024
- Last updated
- 15 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Uyeda MJ, Re A, Tang C, McLaughlin M, Humes D, Sexton S, Freeborn R, Villalba I, Nugent K, Adams J, Wei J, Breton M, Paliard X, Richter M, Roncarolo MG, Bjordahl R. Engineering allogeneic type 1 regulatory T cells: a scalable, off-the-shelf platform for restoring immune tolerance. Front Immunol. 2026 Jul 9;17:1848770. doi: 10.3389/fimmu.2026.1848770. eCollection 2026.
PubMed 42495646
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I, First in Human, Open Label Study to Evaluate Safety and Tolerability of TRX103 Cells in Subjects With Hematological Malignancies Undergoing HLA-mismatched Related or Unrelated Hematopoietic Stem Cell Transplantation (HSCT)
- ClinicalTrials.gov ID
- NCT06462365
- Acronym / sponsor ID
- TRX103-01
- Conditions
- Hematologic Malignancy, GvHD, GVHD,Acute, GVHD, Chronic, Hematopoietic Stem Cell Transplant, Acute Lymphoblastic Leukemia, Adult B-Cell
- Keywords
- AML, CMML, Stem Cell Transplant, B-ALL, T-ALL, Acute GvHD, Graft versus Host Disease, Chronic GvHD
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Tr1X, Inc.Industry (pharma, biotech, devices)
- Study start
- 8 Apr 2024 (actual)
- Primary completion
- 15 Apr 2026 (estimated)
- Study completion
- 15 Apr 2027 (estimated)
- First posted
- 17 Jun 2024 (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.
The purpose of this Phase 1, first in human open-label study is to assess the safety and tolerability of TRX-103 in patients with hematological malignancies undergoing HLA-mismatched related or unrelated hematopoietic stem cell transplantation (HSCT). It is anticipated that up to 36 Subjects will be enrolled during a 18-24 month enrollment period. TRX-103 will be infused one time post HSCT.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TRX-103 | Experimental | Biological: TRX103TRX-103 will be infused on time post hematopoietic stem cell transplant (HSCT). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Subjects with one of the following hematologic malignancies: Acute Lymphoblastic Leukemia (B- or T-ALL), Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), or Chronic myelomonocytic leukemia (CMML)
- Males and Females Age ≥ 18 years.
- Weight of ≥ 35 Kg.
- Karnofsky performance status ≥ 70 %.
- Available mismatched related (haploidentical) or unrelated donors for peripheral blood stem cell (PBSC) donation.
Key exclusion criteria (9 listed)
- Prior allogeneic bone marrow, peripheral blood, or cord blood HSCT.
- Any subject with a history of significant renal, hepatic, pulmonary, or cardiac dysfunction, or on treatment to support cardiac dysfunction.
- HIV positive.
- Positive hepatitis-B surface antigen. Subject may be included if they are HBV PCR negative.
- Positive hepatitis-C antibody with positive Recombinant Immunoblot Assay (RIBA) or PCR unless the subject has received curative anti-viral treatment and confirmed negative viral load by PCR.
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 |
|---|---|---|
| TRX103TRX103 infusion via central line. | Biological | TRX-103 |
Primary outcome
Safety and tolerability of TRX103 cell infusion through incidence of Adverse events.
Time frame: Up to a year
Safety of TRX103 determined by stem cell engraftment and donor chimerism after HSCT measured by absolute neutrophil counts and percent donor chimerism.
Time frame: Up to day 42
Safety of TRX103 determined by negative Replication Competent Lentivirus (RCL).
Time frame: At 3-month, 6-month, and 1-year.
4 secondary outcomes
Incidence of Grade II-IV acute GvHD (aGvHD).
Time frame: Day 0 to Day +100 day
Incidence of Grade III-IV acute GvHD (aGvHD).
Time frame: Day 0 to Day +100 day
Incidence and severity of chronic GvHD (cGvHD)
Time frame: Day +100 through Day +365
Overall survival at Day +365.
Time frame: Up to a year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2025.
- Derived from this study: PubMed 42495646Uyeda MJ, Re A, Tang C, McLaughlin M, Humes D, Sexton S, Freeborn R, Villalba I, Nugent K, Adams J, Wei J, Breton M, Paliard X, Richter M, Roncarolo MG, Bjordahl R. Engineering allogeneic type 1 regulatory T cells: a scalable, off-the-shelf platform for restoring immune tolerance. Front Immunol. 2026 Jul 9;17:1848770. doi: 10.3389/fimmu.2026.1848770. eCollection 2026.
- 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.
Maria Grazia Roncarolo, MD study director
Tr1X, Inc.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TRX103 in people with Hematologic Malignancy and GvHD, run by Tr1X, Inc. at 5 sites in United States.
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 9 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: looking for participants now, as of the sponsor's last update on 15 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Tr1X Clinical Trials (858-283-7879, Tr1xClinicalTrials@Tr1x.bio), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of TRX103 cell infusion through incidence of Adverse events. (time frame: Up to a year). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06462365, which also lists 1 linked publication.
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 15 Apr 2025; AzTrials last refreshed its copy on 8 Nov 2026.