GT729 universal cell injection for refractory or relapsed chronic graft-versus-host disease

A Clinical Study Evaluating the Safety and Efficacy of GT729 Universal Cell Injection in the Treatment of Refractory or Relapsed Chronic Graft-versus-host Disease (cGVHD)

This trial will try GT729 universal cell injections in adults whose chronic graft-versus-host disease has not responded to at least two prior systemic treatments after an allogeneic stem cell transplant.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Grit Biotechnology (Industry-sponsored)
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT07253259 on ClinicalTrials.gov

What this trial studies

This is an early Phase 1 interventional trial testing an off-the-shelf 'universal' cell product, GT729, given by injection to adults with active refractory or relapsed chronic graft-versus-host disease (cGVHD) after allogeneic hematopoietic stem cell transplantation. The primary focus is safety with collection of initial signals of clinical activity; patients must have failed at least two prior systemic therapies. Treatment and follow-up occur at the Institute of Hematology & Blood Diseases Hospital in Tianjin, China, with screening laboratory cutoffs required for blood counts and liver tests. Investigators will monitor adverse events, laboratory values, and cGVHD response measures over the study period.

Who should consider this trial

Good fit: Adults aged 18 to 65 who underwent allogeneic stem cell transplant, have active refractory or relapsed cGVHD requiring systemic therapy, and have failed at least two prior systemic treatments with adequate blood counts and liver tests are ideal candidates.

Not a fit: Patients outside the 18–65 age range, those with controlled cGVHD on standard therapy, or those with inadequate blood counts or significant liver dysfunction are unlikely to benefit from this experimental intervention.

Why it matters

Potential benefit: If successful, GT729 could provide a new off-the-shelf cellular option to reduce symptoms of cGVHD in patients who have not responded to standard treatments.

How similar studies have performed: Cellular therapies such as mesenchymal stromal cells have produced mixed results in cGVHD, and engineered 'universal' off-the-shelf cell injections like GT729 remain a novel, early-stage approach.

Eligibility criteria

Inclusion Criteria:

* Participants or their legal representatives voluntarily sign a written informed consent form, are willing and able to comply with the procedures of this study.
* Aged 18 to 65 years old (inclusive) when signing the informed consent, regardless of gender.
* Participants must meet the following criteria:

  1. The subjects are allogeneic hematopoietic stem cell transplantation (alloHSCT) recipients with active chronic graft-versus-host disease (active cGVHD) requiring systemic immunosuppressive therapy.
  2. The subjects are patients with refractory or relapsed active cGVHD after receiving at least two lines of systemic treatment.
* The laboratory test results during the screening period must meet the following criteria (except for indicators related to the study disease):

  1. Neutrophil count ≥ 1.0×10⁹/L;
  2. Hemoglobin ≥ 80g/L; Platelet count ≥ 30×10⁹/L;
  3. Alanine transaminase ≤ 3×upper limit of normal (ULN); Aspartate transaminase ≤ 3×ULN; Total bilirubin (TBIL) \< 2×ULN;
  4. Creatinine clearance rate ≥ 30 mL/min.
* Women of childbearing age must:

At the time of screening, as confirmed by the investigator, the result of the serum β-human chorionic gonadotropin (β-hCG) pregnancy test is negative.

Exclusion Criteria:

* Evidence of recurrence of underlying malignant tumors or post-transplant lymphoproliferative disorder (PTLD) at the time of screening
* Having a history of severe hypersensitivity or allergies
* Suffering from the following heart diseases:

  1. New York Heart Association (NYHA) Class III or IV congestive heart failure;
  2. A myocardial infarction occurred or coronary artery bypass surgery was performed within 6 months before the screening period.
* Participants with clinically significant bleeding symptoms or a clear bleeding tendency within the 6 months prior to screening;
* Participants with severe underlying medical conditions at the time of screening;
* Participants who have undergone major surgery within 8 weeks prior to screening or are scheduled to undergo surgery during the study period;
* History of organ transplantation;
* According to the investigator's judgment, there are circumstances that would prevent the participant from completing the entire trial, confuse the trial results, or make participation in the trial not in the best interest of the participant.

Where this trial is running

Tianjin, Tianjin Municipality

• Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Study coordinator: Erlie Jiang

Conditions cGVHD