Preventing GvHD in patients with blood cancers undergoing stem cell transplants

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)

This study is testing a new treatment called TRX-103 to see if it can help prevent graft-versus-host disease in people with blood cancers who are getting stem cell transplants from mismatched donors.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Tr1X, Inc. (Industry-sponsored)
• Locations: 5 sites (Duarte, California and 4 other locations)
• Trial ID: NCT06462365 on ClinicalTrials.gov

What this trial studies

This Phase 1 open-label study aims to evaluate the safety and tolerability of TRX-103 in patients with hematological malignancies who are undergoing hematopoietic stem cell transplantation (HSCT) from HLA-mismatched donors. Up to 36 participants will be enrolled over an 18-24 month period, receiving a single infusion of TRX-103 post-transplant. The study focuses on patients with conditions such as Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia. The primary goal is to prevent graft-versus-host disease (GvHD), a common complication following HSCT.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with specific hematological malignancies who are eligible for HSCT and have available mismatched donors.

Not a fit: Patients who have previously undergone allogeneic stem cell transplantation or those with uncontrolled infections may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could significantly reduce the incidence of GvHD in patients undergoing stem cell transplants, improving their overall outcomes.

How similar studies have performed: While there have been studies addressing GvHD prevention, this approach with TRX-103 is novel and has not been previously tested in humans.

Eligibility criteria

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.

Where this trial is running

Duarte, California and 4 other locations

• City of Hope — Duarte, California, United States (Recruiting)
• Dana-Farber Cancer Institute — Boston, Massachusetts, United States (Recruiting)
• University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
• Memorial Sloan Kettering Cancer Center — New York, New York, United States (Recruiting)
• Fred Hutchinson Cancer Center — Seattle, Washington, United States (Recruiting)

Study contacts

• Study coordinator: Tr1X Clinical Trials

Conditions Hematologic Malignancy; GvHD; GVHD,Acute; GVHD, Chronic; Hematopoietic Stem Cell Transplant; Acute Lymphoblastic Leukemia, Adult B-Cell; Acute Lymphoblastic Leukemia, Adult T-Cell; Acute Myeloid Leukemia in Remission