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Use of T-allo10 in Hematopoietic Stem Cell Transplantation (HSCT) for Blood Disorders
Phase 1 trial testing T-allo10 in AML - Acute Myeloid Leukemia in 5 participants. Terminated before completion.
| Lead sponsor | Roncarolo, Maria Grazia, MD |
|---|---|
| Phase | Phase 1 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | sequential |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 5 |
| Start date | 30 August 2017 |
| Primary completion | 11 November 2021 |
| Estimated completion | 11 November 2021 |
| Sites | 1 location across United States |
Roncarolo, Maria Grazia, MD — full company profile →
Adults 3 to 45, any sex, with AML - Acute Myeloid Leukemia or MDS (Myelodysplastic Syndrome). Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of participants experiencing TEAEs. Assessments of TEAE will include laboratory abnormalities, changes in vital signs, and changes in physical examination related to the infusion of T-allo10 cells in order to assess the tolerability of T-allo10.
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 1 | |
| Cohort 2 | 0 |
Number of participants experiencing TEAEs related to infusion, by severity graded according to the CTCAE grading system, from grade 1 (least severe) to grade 5 (death). Assessments of TEAE will include laboratory abnormalities, changes in vital signs, and changes in physical examination following infusion of T-allo10 cells in order to assess the safety of T-allo10.
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 1 | |
| Cohort 2 | 0 |
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 0 | |
| Cohort 2 | 0 |
Stem cell engraftment is evaluated by clinical laboratory studies including absolute neutrophil count above 500/mm3 for three consecutive days, hematopoiesis at bone marrow examination, with cellularity \>5 % and donor chimerism \>90% by short tandem repeat (STR) analysis for the presence of donor cells, and minimal residual disease (MRD) assay \< 0.1%.
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 3 | |
| Cohort 2 | 1 |
Feasibility defined by the rate of successful manufacture of the T-allo10 product to satisfy the targeted dose level and meet the required release specifications. Number of products meeting specifications out of total products manufactured is reported.
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 3 | |
| Cohort 2 | 0 |
The number of patients who experienced grade III and IV acute GvHD at Day +100 following infusion of Tallo10 cells, assessed using the Modified Keystone scale administered by an independent evaluator on study visits through Day +100
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 2 | |
| Cohort 2 | 0 |
The number of participants who experienced chronic GvHD and severity will be assessed by an independent evaluator. Outcome is reported as the highest level of chronic GVHD reported.
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 2 | |
| Cohort 2 | 0 |
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 1 | |
| Cohort 2 | 1 |
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 0 | |
| Cohort 2 | 0 |
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 3 | |
| Cohort 2 | 1 |
Immune reconstitution will be evaluated by clinical laboratory studies of CD3+ T cells, assessed by the number of days to reach \>200/microliter CD3+ T cells.
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 22 | 21 – 28 |
| Cohort 2 | 56 | 56 – 56 |
Disease free survival is defined as the absence of minimal residual disease in the bone marrow. The investigators will use bone marrow aspirate examination, minimal residual disease (MRD) assay, and donor chimerism by STR analysis to evaluate disease free survival.
| Group | Value | 95% CI |
|---|---|---|
| Cohort 1 | 3 | |
| Cohort 2 | 1 |
Time frame: Adverse events were recorded up to 1 year post-transplant.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Cohort 1 | Cohort 2 |
|---|---|---|---|
| Fever | General disorders | — | — |
| Acute kidney injury | Renal and urinary disorders | — | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — | — |
| Duodenal hematoma | Vascular disorders | — | — |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | — | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Neutrophil count decreased | Investigations | — | — |
| Sepsis | Infections and infestations | — | — |
| Viremia: RSV | Infections and infestations | — | — |
| Reaction | System | Cohort 1 | Cohort 2 |
|---|---|---|---|
| Alanine aminotransferase increased (ALT) | Investigations | — | — |
| Aspartate aminotransferase increased (AST) | Investigations | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | — | — |
| Hypertriglyceridemia | Metabolism and nutrition disorders | — | — |
| Hypokalemia | Metabolism and nutrition disorders | — | — |
| Mucositis Oral | Gastrointestinal disorders | — | — |
| Neutrophil count decreased | Investigations | — | — |
| Platelet count decreased | Investigations | — | — |
| Sinus tachycardia | Cardiac disorders | — | — |
| Viremia: CMV | Infections and infestations | — | — |
| Viremia: HHV6 | Infections and infestations | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — |
| Anorexia | Metabolism and nutrition disorders | — | — |
| Cholesterol high | Investigations | — | — |
| Diarrhea | Gastrointestinal disorders | — | — |
| Enterocolitis infectious (C.difficile) | Infections and infestations | — | — |
| Fatigue | General disorders | — | — |
| Fever | General disorders | — | — |
| GVHD: Chronic skin | Immune system disorders | — | — |
| GVHD: Acute skin | Immune system disorders | — | — |
| Headache | Nervous system disorders | — | — |
| High Blood Urea Nitrogen (BUN) | Metabolism and nutrition disorders | — | — |
| Hypoalbuminemia | Metabolism and nutrition disorders | — | — |
| Hypocalcemia | Metabolism and nutrition disorders | — | — |
| Hypomagnesemia | Metabolism and nutrition disorders | — | — |
| Hypophosphatemia | Metabolism and nutrition disorders | — | — |
| Hypotension | Vascular disorders | — | — |
| Increased LDH | Investigations | — | — |
| Insomnia | Psychiatric disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Oral pain | Gastrointestinal disorders | — | — |
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | — | — |
| Sinus bradycardia | Cardiac disorders | — | — |
| Skin redness | Skin and subcutaneous tissue disorders | — | — |
| Viremia: Adenovirus | Infections and infestations | — | — |
| Viremia: EBV | Infections and infestations | — | — |
| Vitamin D deficiency | Investigations | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Weight loss | Investigations | — | — |
Most-reported serious reactions: Fever, Acute kidney injury, Cough, Duodenal hematoma, Dyspnea, Febrile neutropenia, Nausea, Neutrophil count decreased.
Data from ClinicalTrials.gov NCT03198234 adverse events section.
A significant number of patients with hematologic malignancies need a hematopoietic stem cell transplant (HSCT) to be cured. Only about 50% of these patients have a fully matched donor, the remaining patients will require an HSCT from a mismatched related or unrelated donor. Almost 60% of these mismatched donor HSCTs will result in graft-versus-host disease (GvHD), which can cause significant morbidity and increased non-relapse mortality. GvHD is caused by the donor effector T cells present in the HSC graft that recognize and react against the mismatched patient's tissues. Researchers and physicians at Lucile Packard Children's Hospital, Stanford are working to prevent GvHD after HSCT with a new clinical trial. The objective of this clinical program is to develop a cell therapy to prevent GvHD and induce graft tolerance in patients receiving mismatched unmanipulated donor HSCT. The cell therapy consists of a cell preparation from the same donor of the HSCT (T-allo10) containing T regulatory type 1 (Tr1) cells able to suppress allogenic (host-specific) responses, thus decreasing the incidence of GvHD. This is the first trial of its kind in pediatric patients and is only available at Lucile Packard Children's Hospital, Stanford. The purpose of this phase 1 study is to determine the safety and tolerability of a cell therapy, T-allo10, to prevent GvHD in patients receiving mismatched related or mismatched unrelated unmanipulated donor HSCT for hematologic malignancies.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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