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

NCT06462365 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: TRX103-01
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 5

Brief 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). 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; Acute Lymphoblastic Leukemia, Adult T-Cell; Acute Myeloid Leukemia in Remission; Myelodysplastic Syndromes; Chronic Myelomonocytic Leukemia, in Remission; Cancer Remission

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

Outcome Measures

Primary Outcomes (3)

• Safety and tolerability of TRX103 cell infusion through incidence of Adverse events.

  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.

  Up to day 42

• Safety of TRX103 determined by negative Replication Competent Lentivirus (RCL).

  At 3-month, 6-month, and 1-year.

Secondary Outcomes (4)

• Incidence of Grade II-IV acute GvHD (aGvHD).

  Day 0 to Day +100 day

• Incidence of Grade III-IV acute GvHD (aGvHD).

  Day 0 to Day +100 day

• Incidence and severity of chronic GvHD (cGvHD)

  Day +100 through Day +365

• Overall survival at Day +365.

  Up to a year

Study Arms (1)

TRX-103

EXPERIMENTAL

TRX-103 will be infused on time post hematopoietic stem cell transplant (HSCT).

Biological: TRX103

Interventions

TRX103 BIOLOGICAL

TRX103 infusion via central line.

TRX-103

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Tr1X, Inc.: lead

Study Sites (5)

City of Hope

Duarte, California, 91010, United States

RECRUITING

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

RECRUITING

Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States

RECRUITING

MeSH Terms

Conditions

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

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Immune System Diseases; Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Bone Marrow Diseases; Leukemia, Myeloid; Myelodysplastic-Myeloproliferative Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Maria Grazia Roncarolo, MD

  Tr1X, Inc.

  STUDY DIRECTOR

Central Study Contacts

Tr1X Clinical Trials

CONTACT

858-283-7879; Tr1xClinicalTrials@Tr1x.bio

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2024
• First Posted: June 17, 2024
• Study Start: April 8, 2024
• Primary Completion: April 15, 2026
• Study Completion (Estimated): April 15, 2027
• Last Updated: April 15, 2025

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)