Phase 2
Completed N=15
Safety and Efficacy of Two Doses of ATIR101, a T-lymphocyte Enriched Leukocyte Preparation Depleted of Host Alloreactive T-cells, in Patients With a Hematologic Malignancy Who Received a Hematopoietic Stem Cell Transplantation From a Haploidentical Donor
Source: ClinicalTrials.gov NCT02500550 ↗Enrolled (actual)
15
Serious AEs
46.7%
Results posted
Jan 2021
Primary outcomePrimary: Incidence of Acute Graft Versus Host Disease (GVHD) Grade III/IV — 2; 2 participants
Summary
The purpose of this study is to determine whether a repeat dose administration of ATIR101 is safe and effective when infused in patients with a hematologic malignancy following a T-cell depleted stem cell graft from a related haploidentical donor. All patients are planned to receive two ATIR101 doses of 2×10E6 viable T-cells/kg, unless the second dose is reduced or halted for safety reasons.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Acute Graft Versus Host Disease (GVHD) Grade III/IV |
2; 2 | — |
| SECONDARY Incidence and Severity of Acute and Chronic GVHD |
0; 0; 0; 2 | — |
| SECONDARY Percentage of Participants Who Achieved T-Cell Reconstitution at 6 and 12 Months Post HSCT |
44.4; 50.0; 77.8; 50.0 | — |
| SECONDARY Viral, Fungal, and Bacterial Infections |
10; 7; 3 | — |
| SECONDARY Transplant-related Mortality (TRM) |
22.2; 66.7 | — |
| SECONDARY Relapse-related Mortality (RRM) |
11.1; 0 | — |
| SECONDARY Overall Survival (OS) |
66.7; 33.3 | — |
| SECONDARY Progression-free Survival (PFS) |
55.6; 33.3 | — |
| SECONDARY GVHD-free, Relapse-free Survival (GRFS) |
55.6; 16.7 | — |
Eligibility Criteria
Inclusion Criteria
- Any of the following hematologic malignancies:
- Acute myeloid leukemia (AML) in first remission with high-risk features or in second or higher remission
- Acute lymphoblastic leukemia (ALL) in first remission with high-risk features or in second or higher remission
- Myelodysplastic syndrome (MDS): transfusion-dependent, or intermediate or higher IPSS-R risk group
- Karnofsky performance status ≥ 70%
- Eligible for haploidentical stem cell transplantation according to the investigator
- Male or female, age ≥ 18 years and ≤ 65 years
Exclusion Criteria
- Availability of a fully matched related or unrelated donor following a donor search
- Diffusing capacity for carbon monoxide (DLCO) 2.5 x ULN (CTCAE grade 2)
- Bilirubin > 1.5 x ULN (CTCAE grade 2)
- Creatinine clearance < 50 mL/min (calculated or measured)
- Positive HIV test
- Positive pregnancy test (women of childbearing age only)
- Prior allogeneic HSCT
- Estimated probability of surviving less than 3 months
- Known allergy to any of the components of ATIR101 (e.g., dimethyl sulfoxide)
- Known presence of HLA antibodies against the non-shared donor haplotype
- Any other condition which, in the opinion of the investigator, makes the patient ineligible for the study
Inclusion Criteria Donor:
- Haploidentical family donor with 2 to 3 mismatches at the HLA-A, -B and/or -DR loci of the unshared haplotype
- Male or female, age ≥ 16 and ≤ 75 years (If applicable, local legal requirements for donors under the age of 18 will be followed)
- Eligible for donations of human blood and blood components according to local requirements and regulations
- Eligible for donation according to the transplantation center
Exclusion Criteria Donor:
- Positive pregnancy test or nursing (women of childbearing age only)
- Positive viral test for HIV-1, HIV-2, HBV, HCV, Treponema pallidum, HTLV 1 (if tested), HTLV-2 (if tested), or WNV (if tested)
Data sourced from ClinicalTrials.gov (NCT02500550). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.