Prevention of GvHD in Participants With Hematological Malignancies Undergoing Hematopoietic Stem Cell Transplant (HSCT)

April 10, 2025 updated by: Tr1X, Inc.

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

36

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Duarte, California, United States, 91010
        • Recruiting
        • City of Hope
        • Contact:
          • Phone Number: (800)826-4673
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota
        • Contact:
    • New York
      • New York, New York, United States, 10065
        • Recruiting
        • Memorial Sloan Kettering Cancer Center
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98109
        • Recruiting
        • Fred Hutchinson Cancer Center
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. 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)
  2. Males and Females Age ≥ 18 years.
  3. Weight of ≥ 35 Kg.
  4. Karnofsky performance status ≥ 70 %.
  5. Available mismatched related (haploidentical) or unrelated donors for peripheral blood stem cell (PBSC) donation.
  6. Subjects must otherwise fulfill institutional criteria for eligibility to undergo allogeneic stem cell transplantation.
  7. Absence of uncontrolled bacterial, viral or fungal infection at time of enrollment.
  8. Have adequate organ function.
  9. Subjects > 65-year-old receiving MAC conditioning will only be eligible if they have a HSCT-comorbidity index score < 5.
  10. Subjects must be able to understand and sign informed consent and be willing and able to complete all specified procedures and visits.

Exclusion Criteria:

  1. Prior allogeneic bone marrow, peripheral blood, or cord blood HSCT.
  2. Any subject with a history of significant renal, hepatic, pulmonary, or cardiac dysfunction, or on treatment to support cardiac dysfunction.
  3. HIV positive.
  4. Positive hepatitis-B surface antigen. Subject may be included if they are HBV PCR negative.
  5. 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.
  6. 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.
  7. Subjects with a previous history of Thrombotic Thrombocytopenic Purpura (TTP) or Hemolytic Uremic Syndrome (HUS) who are not good candidates for treatment with sirolimus.
  8. 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).
  9. Any serious illness, uncontrolled inter-current illness, psychiatric illness, active or uncontrolled infection, or other medical condition or history, including laboratory results.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: TRX-103
TRX-103 will be infused on time post hematopoietic stem cell transplant (HSCT).
Biological: TRX103
TRX103 infusion via central line.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety and tolerability of TRX103 cell infusion through incidence of Adverse events.
Time Frame: Up to a year
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
Up to day 42
Safety of TRX103 determined by negative Replication Competent Lentivirus (RCL).
Time Frame: At 3-month, 6-month, and 1-year.
At 3-month, 6-month, and 1-year.

Secondary Outcome Measures

Outcome Measure
Time Frame
Incidence of Grade II-IV acute GvHD (aGvHD).
Time Frame: Day 0 to Day +100 day
Day 0 to Day +100 day
Incidence of Grade III-IV acute GvHD (aGvHD).
Time Frame: Day 0 to Day +100 day
Day 0 to Day +100 day
Incidence and severity of chronic GvHD (cGvHD)
Time Frame: Day +100 through Day +365
Day +100 through Day +365
Overall survival at Day +365.
Time Frame: Up to a year
Up to a year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Tr1X, Inc.

Investigators

  • Study Director: Maria Grazia Roncarolo, MD, Tr1X, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 8, 2024

Primary Completion (Estimated)

April 15, 2026

Study Completion (Estimated)

April 15, 2027

Study Registration Dates

First Submitted

June 6, 2024

First Submitted That Met QC Criteria

June 11, 2024

First Posted (Actual)

June 17, 2024

Study Record Updates

Last Update Posted (Actual)

April 15, 2025

Last Update Submitted That Met QC Criteria

April 10, 2025

Last Verified

October 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TRX103-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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