Phase 2
Completed N=31
Safety and Efficacy of Donor T-lymphocytes Depleted ex Vivo of Host Alloreactive T-cells (ATIR) in Patients With a Hematologic Malignancy Who Received a Hematopoietic Stem Cell Transplantation From a Haploidentical Donor
Source: ClinicalTrials.gov NCT01794299 ↗Enrolled (actual)
31
Serious AEs
13.0%
Results posted
Jan 2021
Primary outcomePrimary: Transplant-related Mortality (TRM) — 13 Kaplan-Meier estimates (%)
Summary
The purpose of this study is to determine whether ATIR is safe and effective in reducing transplant-related mortality and improving overall survival, when infused in patients with a hematologic malignancy following a T-cell depleted stem cell graft from a related haploidentical donor.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Transplant-related Mortality (TRM) |
13 | — |
| SECONDARY Immune Reconstitution |
18; 13 | — |
| SECONDARY Relapse-related Mortality (RRM) |
5; 10; 25 | — |
| SECONDARY Overall Survival (OS) |
83; 61; 39 | — |
| SECONDARY Progression-free Survival (PFS) |
78; 61; 39 | — |
| SECONDARY Number of Participants With Viral, Fungal, and Bacterial Infections. |
17; 19; 13; 12 | — |
| SECONDARY Number of Participants With Graft Versus Host Disease (GVHD) |
2; 0; 3; 3; 4 | — |
Eligibility Criteria
Inclusion Criteria
- Any of the following hematologic malignancies: a) Acute myeloid leukemia (AML) in first remission with high-risk features or in second or higher remission b) Acute lymphoblastic leukemia (ALL) in first remission with high-risk features or in second or higher remission c) Myelodysplastic syndrome (MDS): transfusion-dependent, or intermediate or higher Revised International Prognostic Scoring System (IPSS-R) risk group
- Eligible for haploidentical stem cell transplantation according to the investigator
Exclusion Criteria
- Availability of a suitable matched related or unrelated donor following a donor search
- In second or higher remission with the previous remission having lasted less than 6 months
- Diffusing capacity for carbon monoxide (DLCO) 2.5 x upper limit of normal (ULN)(CTCAE grade 2)
- Bilirubin > 1.5 x ULN (CTCAE grade 2)
- Creatinine clearance < 50 mL/min (calculated or measured)
- Positive test for human immunodeficiency virus (HIV)
- Positive pregnancy test (women of childbearing age only)
- Prior allogeneic stem cell transplantation using stem cells from a matched sibling donor, a matched unrelated donor, a haploidentical donor, or a cord blood donor
- Prior autologous stem cell transplantation
- Stay at intensive care unit for more than 2 months in the preceding 12 months
- Estimated probability of surviving less than 3 months
- Known allergy to any of the components of ATIR (e.g., dimethyl sulfoxide)
- Any other condition which, in the opinion of the investigator, makes the patient ineligible for the study
Donor inclusion criteria
- Haploidentical family donor with 2 to 3 mismatches at the human leukocyte antigen (HLA)-A, -B and/or -DR loci of the unshared haplotype
- Male or female, age ≥ 16 and ≤ 75 years
- Eligible for donation according to the transplantation center
Donor exclusion criteria
- Positive viral test for HIV-1, HIV-2, hepatitis B virus (HBV), hepatitis C virus (HCV), Treponema pallidum, human T-lymphotropic virus (HTLV)-1*, HTLV-2*, or West Nile virus (WNV)* (if tested) (* at Canadian centers only)
- Positive pregnancy test or nursing (women of childbearing age only)
Data sourced from ClinicalTrials.gov (NCT01794299). 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.