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
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ClinicalTrials.gov Identifier: NCT02500550 |
Recruitment Status :
Completed
First Posted : July 16, 2015
Results First Posted : January 12, 2021
Last Update Posted : May 18, 2021
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Condition or disease | Intervention/treatment | Phase |
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Acute Myeloid Leukemia Acute Lymphoblastic Leukemia Myelodysplastic Syndrome | Biological: ATIR101 Procedure: Haploidentical hematopoietic stem cell transplantation (HSCT) Procedure: TBI regime Procedure: Non-TBI regime | Phase 2 |
Study CR-AIR-008 is an exploratory, open-label, multicenter study. After signing informed consent, patients will receive a hematopoietic stem cell transplantation (HSCT) from a related, haploidentical donor, followed by a first ATIR101 infusion at a dose of 2×10E6 viable T-cells/kg between 28 and 32 days after the HSCT. Patients will receive a second ATIR101 infusion at a dose of 2×10E6 viable T-cells/kg between 70 and 74 days after the HSCT. To evaluate safety of the second dose administration, the first 6 patients treated will be evaluated for the occurrence of dose limiting toxicity (DLT), defined as acute GvHD grade III/IV within 120 days post HSCT (or within 42 days after the second ATIR101 infusion in case of prior dose delays). If within the first 6 patients no DLT is observed, treatment of the remaining 9 patients will continue with two ATIR101 doses of 2×10E6 viable T cells/kg. If within the first 6 patients at least 2 patients show DLT, the second ATIR101 infusion will be adjusted to a dose of 1×10E6 viable T cells/kg. If in one of the next 3 patients treated at this lower dose again DLT is observed, the second ATIR101 infusion will be halted and the remaining patients will be given only a single dose of ATIR101.
All patients treated with ATIR101 will be followed up until 12 months after the HSCT. Assessments will be performed at weekly visits from the day of the first ATIR101 infusion (Week 4) until 6 weeks after the second ATIR101 infusion (Week 16), at monthly visits from 4 until 6 months after the HSCT, and every 3 months from 6 until 12 months after the HSCT.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 15 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Prevention |
Official Title: | An Exploratory, Open-label, Multicenter Study to Evaluate the Safety and Efficacy of a Two-dose Regimen of ATIR101, a T-lymphocyte Enriched Leukocyte Preparation Depleted ex Vivo of Host Alloreactive T-cells (Using Photodynamic Treatment), in Patients With a Hematologic Malignancy, Who Received a CD34-selected Hematopoietic Stem Cell Transplantation From a Haploidentical Donor |
Actual Study Start Date : | October 9, 2015 |
Actual Primary Completion Date : | July 2018 |
Actual Study Completion Date : | December 17, 2018 |

Arm | Intervention/treatment |
---|---|
Experimental: ATIR101 |
Biological: ATIR101
T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells (using photodynamic treatment). Two intravenous infusions with 2x10E6 viable T-cells/kg approximately 42 days apart (unless the second dose is reduced or halted for safety reasons). Procedure: Haploidentical hematopoietic stem cell transplantation (HSCT) CD34-selected HSCT from a haploidentical donor. In order to prepare the patient for the HSCT one of the following myeloablative conditioning regimens is recommended:
(See below for details) Procedure: TBI regime
Procedure: Non-TBI regime
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- Incidence of Acute Graft Versus Host Disease (GVHD) Grade III/IV [ Time Frame: 180 days post HSCT ]
- Incidence and Severity of Acute and Chronic GVHD [ Time Frame: Between 6 and 12 months after HSCT ]
- Percentage of Participants Who Achieved T-Cell Reconstitution at 6 and 12 Months Post HSCT [ Time Frame: 6 and 12 months post HSCT ]Defined as CD3+ in peripheral blood higher than 0.2×10E9/L at 6 and 12 months post HSCT.
- Viral, Fungal, and Bacterial Infections [ Time Frame: From 6 months to 1 year after HSCT ]Infection was defined as (1) a clinically apparent infectious disease with symptoms or (2) a viral reactivation. Severity was graded according to CTCAE vs. 4.0
- Transplant-related Mortality (TRM) [ Time Frame: 12 months post HSCT ]Defined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide)
- Relapse-related Mortality (RRM) [ Time Frame: 12 months post HSCT ]Defined as death due to disease relapse or disease progression
- Overall Survival (OS) [ Time Frame: 12 months post HSCT ]Defined as the time from HSCT until death from any cause
- Progression-free Survival (PFS) [ Time Frame: 12 months post HSCT ]Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first
- GVHD-free, Relapse-free Survival (GRFS) [ Time Frame: 12 months post HSCT ]Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first

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Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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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) < 50% predicted
- Left ventricular ejection fraction < 50% (evaluated by echocardiogram or MUGA)
- AST > 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)

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02500550
Belgium | |
Algemeen Ziekenhuis Sint-Jan | |
Brugge, Belgium, 8000 | |
Institut Jules Bordet | |
Brussels, Belgium, 1000 | |
Universitair Ziekenhuis Gasthuisberg | |
Leuven, Belgium, 3000 | |
Centre Hospitalier Universitaire de Liège | |
Liège, Belgium, 4000 | |
Canada, Ontario | |
Juravinski Hospital and Cancer Centre | |
Hamilton, Ontario, Canada, L8V 1C3 | |
Canada, Quebec | |
Maisonneuve-Rosemont Hospital | |
Montreal, Quebec, Canada, H1T 2M4 | |
Croatia | |
University Hospital Centre Zagreb | |
Zagreb, Croatia, 10000 | |
Germany | |
University Medical Center Mainz | |
Mainz, Germany, 55131 | |
Portugal | |
Hospital de Santa Maria, Clinica Universitaria Hematologia | |
Lisboa, Portugal, 1649-028 | |
United Kingdom | |
Heartlands Hospital | |
Birmingham, United Kingdom, B9 5SS | |
Hammersmith Hospital | |
London, United Kingdom, W12 ONN |
Principal Investigator: | Denis Claude Roy, Prof MD | Maisonneuve-Rosemont Hospital (Montreal, Canada) | |
Principal Investigator: | Stephan Mielke, Prof MD | Centre for Allogeneic Stem Cell Transplantation, Karolinska University Hospital (Stockholm, Sweden) |
Documents provided by Kiadis Pharma:
Responsible Party: | Kiadis Pharma |
ClinicalTrials.gov Identifier: | NCT02500550 |
Other Study ID Numbers: |
CR-AIR-008 |
First Posted: | July 16, 2015 Key Record Dates |
Results First Posted: | January 12, 2021 |
Last Update Posted: | May 18, 2021 |
Last Verified: | May 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Haploidentical stem cell transplantation Graft versus host disease Immune reconstitution Alloreactive T-cells |
Photodynamic treatment Hematologic malignancy Transplant-related mortality |
Leukemia Neoplasms Precursor Cell Lymphoblastic Leukemia-Lymphoma Hematologic Neoplasms Myelodysplastic Syndromes Neoplasms by Histologic Type Bone Marrow Diseases |
Hematologic Diseases Leukemia, Lymphoid Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Neoplasms by Site |