Safety and Efficacy of ATIR101 as Adjunctive Treatment to Blood Stem Cell Transplantation From a Haploidentical Family Donor Compared to Post-transplant Cyclophosphamide in Patients With Blood Cancer



Status:Recruiting
Conditions:Cancer, Blood Cancer, Blood Cancer, Blood Cancer, Blood Cancer
Therapuetic Areas:Oncology
Healthy:No
Age Range:18 - 70
Updated:3/13/2019
Start Date:November 29, 2017
End Date:February 2021
Contact:Andrew Sandler, MD
Email:clinicaltrials@kiadis.com
Phone:+31 20 2405250

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A Phase III, Multicenter, Randomized Controlled Study to Compare Safety and Efficacy of a Haploidentical HSCT and Adjunctive Treatment With ATIR101, a T-lymphocyte Enriched Leukocyte Preparation Depleted ex Vivo of Host Alloreactive T-cells, Versus a Haploidentical HSCT With Post-transplant Cyclophosphamide in Patients With a Hematologic Malignancy

The primary objective of this study is to compare safety and efficacy of a haploidentical
T-cell depleted HSCT and adjunctive treatment with ATIR101 versus a haploidentical T cell
replete HSCT with post-transplant administration of high dose cyclophosphamide (PTCy) in
patients with a hematologic malignancy. An additional objective of the study is to compare
the effect of the two treatments on quality of life.

Study CR-AIR-009 is a Phase III randomized controlled multicenter open-label study comparing
two parallel groups. After signing informed consent, a total of 250 patients will be
randomized in a 1:1 fashion to receive either a T-cell depleted hematopoietic stem cell
transplantation (HSCT; CD34 selection) from a related, haploidentical donor, followed by
ATIR101 infusion, or a T-cell replete HSCT, followed by a high dose of post-transplant
cyclophosphamide (PTCy).

Randomization will use minimization to balance treatment groups with respect to underlying
disease (AML, ALL, or MDS), Disease Risk Index (DRI; intermediate risk, high risk, or very
high risk) and center. A stochastic treatment allocation procedure will be used so that the
treatment assignment is random for all patients entered in the study.

Patients randomized in the ATIR101 group will receive a single ATIR101 dose of 2×10E6 viable
T-cells/kg between 28 and 32 days after the HSCT. Patients randomized in the PTCy group will
receive cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT. All patients will be
followed up for at least 24 months post HSCT.

Inclusion Criteria:

- Any of the following hematologic malignancies:

- Acute myeloid leukemia (AML) in first cytomorphological remission (with < 5%
blasts in the bone marrow) with Disease Risk Index (DRI) intermediate or above,
or in second or higher cytomorphological remission (with < 5% blasts in the bone
marrow)

- Acute lymphoblastic leukemia (ALL) in first or higher remission (with < 5% blasts
in the bone marrow)

- Myelodysplastic syndrome (MDS): transfusion-dependent (requiring at least one
transfusion per month), or intermediate or higher Revised International
Prognostic Scoring System (IPSS-R) risk group

- Clinical justification of allogeneic stem cell transplantation where a suitable HLA
matched sibling or unrelated donor is unavailable in a timely manner

- Availability of a related haploidentical donor with one fully shared haplotype and 2
to 4 mismatches at the HLA-A, -B, -C, and -DRB1 loci of the unshared haplotype, as
determined by high resolution human leukocyte antigen (HLA)-typing

- Karnofsky Performance Status (KPS) ≥ 70%

- Male or female, age ≥ 18 years and ≤ 70 years. Patients aged ≥ 65 years must have a
Sorror score ≤ 3

- Patient weight ≥ 25 kg and ≤ 130 kg

- Availability of a donor aged ≥ 16 years and ≤ 75 years who is eligible according to
local requirements and regulations. Donors aged < 16 years are allowed if they are the
only option for an HSCT, if they are permitted by local regulations, and if the
IRB/IEC approves participation in the study.

- For females of childbearing potential who are sexually active and males who have
sexual contact with a female of childbearing potential: willingness to use of reliable
methods of contraception (oral contraceptives, intrauterine device, hormone implants,
contraceptive injection or abstinence) during study participation

- Given written informed consent (patient and donor)

Exclusion Criteria:

- Diagnosis of chronic myelomonocytic leukemia (CMML)

- Availability of a suitable HLA-matched sibling or unrelated donor in a donor search

- Prior allogeneic hematopoietic stem cell transplantation

- Diffusing capacity for carbon monoxide (hemoglobin corrected DLCO) < 50% predicted

- Left ventricular ejection fraction < 45% (evaluated by echocardiogram or MUGA scan)

- Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 2.5 × upper
limit of normal (CTCAE grade 2)

- Creatinine clearance < 50 ml/min (calculated or measured)

- Positive pregnancy test or breastfeeding of patient or donor (women of childbearing
age only)

- Estimated probability of surviving less than 3 months

- Known allergy to any of the components of ATIR101 (e.g., dimethyl sulfoxide)

- Known hypersensitivity to cyclophosphamide or any of its metabolites

- Any contraindication for GVHD prophylaxis with mycophenolate mofetil, cyclosporine A,
or tacrolimus

- Known presence of HLA antibodies against the non-shared donor haplotype

- Positive viral test of the patient or donor for human immunodeficiency virus (HIV)-1,
HIV-2, hepatitis B virus (HBV), hepatitis C virus (HCV), Treponema pallidum, human
T-lymphotropic virus (HTLV)-1 (if tested), HTLV-2 (if tested), West Nile virus (WNV;
if tested), or Zika virus (if tested)

- Any other condition that, in the opinion of the investigator, makes the patient or
donor ineligible for the study
We found this trial at
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
Principal Investigator: Yi-Bin Chen, MD
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201 Dowman Dr
Atlanta, Georgia 30303
(404) 727-6123
Principal Investigator: Edmund K. Waller, Prof MD
Emory University Emory University, recognized internationally for its outstanding liberal artscolleges, graduate and professional schools,...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: Brandon Hayes-Lattin, MD
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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291 Campus Dr
Stanford, California 94305
(650) 725-3900
Principal Investigator: Laura Johnston, MD
Stanford University School of Medicine Vast in both its physical scale and its impact on...
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500 S State St
Ann Arbor, Michigan 48109
(734) 764-1817
Principal Investigator: Monalisa Ghosh, MD
University of Michigan The University of Michigan was founded in 1817 as one of the...
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Antwerp,
Principal Investigator: Wilfried Schroyens, MD
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1500 East Duarte Road
Duarte, California 91010
626-256-HOPE (4673)
Principal Investigator: Ryotaro Nakamura, Prof MD
City of Hope National Medical Center City of Hope is dedicated to making a difference...
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Los Angeles, California 90095
Principal Investigator: Hyung C. Suh, Prof MD
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New York, New York 10021
Principal Investigator: Sebastian A. Mayer, Ass Prof MD
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630 W 168th St
New York, New York
212-305-2862
Principal Investigator: Ran Reshef, Prof MD
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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