A Phase 3 interventional study of ATIR101 and Cyclophosphamide in Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome, sponsored by Kiadis Pharma. Terminated at 42 sites in 13 countries. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2022-05-24.
Sponsored by Kiadis Pharma · Phase 3, Interventional, and Prevention
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.
3,035 studies on the registry are indexed under Leukemia, Myeloid, Acute; 746 are open to participants now.
This study's enrollment of 63 is above the median of 40 across 2,561 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →Kiadis Pharma is the lead sponsor of 10 studies on the registry; none are open to participants now.
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Any of the following hematologic malignancies:
Exclusion Criteria:
T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT
Biological: ATIR101 · Procedure: T-cell depleted HSCT from a related, haploidentical donor
T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT
Drug: Cyclophosphamide · Procedure: T-cell replete HSCT from a related, haploidentical donor
ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion)
High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion)
T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Graft-versus-host Disease-free, Relapse-free Survival (GRFS)
Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.
Time frame: 24 months post-HSCT
Overall Survival (OS)
OS is defined as the time from HSCT until death from any cause. Kaplan-Meier estimates (percentage of participants) of OS were calculated at 24 months post HSCT.
Time frame: 24 months post-HSCT
Progression-free Survival (PFS)
Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
Time frame: 24 months post-HSCT
Relapse-related Mortality (RRM)
Time from randomization to death due to disease relapse or disease progression
Time frame: Through study completion, at least two years post HSCT
Transplant-related Mortality (TRM)
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). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
Time frame: 24 months post-HSCT
Immune Reconstitution
Time to CD3+ \> 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)
Time frame: Through study completion, at least two years post HSCT
Cumulative Incidence of Grade II-IV and Grade III-IV Acute Graft-versus-host-disease (GVHD)
Time frame: Through study completion, at least two years post HSCT
Cumulative Incidence of Moderate/Severe Chronic GVHD
Time frame: Through study completion, at least two years post HSCT
Cumulative Incidence of Chronic GVHD Requiring Systemic Immunosuppressive Treatment
Time frame: Through study completion, at least two years post HSCT
Duration of GVHD Episodes
Time frame: Through study completion, at least two years post HSCT
Cumulative Incidence of NCI CTCAE Grade 2-5 and Grade 3-5 Infections
Viral, fungal, and bacterial infections
Time frame: Until 2 years after the HSCT
Cumulative Incidence of NCI CTCAE Grade 3-5 Adverse Events
Viral, fungal, and bacterial infections
Time frame: Until 2 years after the HSCT
FACT-BMT Total Score (Change From Screening)
Quality of life: Foundation for the Accreditation of Cellular Therapy - Bone Marrow Transplantation questionnaire (FACT-BMT)
Time frame: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)
SF-36 Total Score (Change From Screening)
Quality of life: Short Form 36-item health survey (SF-36)
Time frame: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)
MDASI Total Score (Change From Screening)
Quality of life: MD Anderson Symptom Inventory (MDASI)
Time frame: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)
EQ-5D-5L (Change From Screening)
Quality of life: EQ-5D-5L
Time frame: Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)
| Milestone | ATIR101 | PTCy |
|---|---|---|
| Started | 32 | 31 |
| Completed | 8 | 22 |
| Not completed | 24 | 9 |
| Withdrew: Screen failures | 7 | 2 |
| Withdrew: Discontinued before hsct | 8 | 2 |
| Withdrew: Death | 7 | 5 |
| Withdrew: Completed 2 year follow-up | 1 | 0 |
| Withdrew: End of study after hsct but before treatment | 1 | 0 |
| Milestone | ATIR101 | PTCy |
|---|---|---|
| Started | 8 | 22 |
| Completed | 5 | 19 |
| Not completed | 3 | 3 |
| Withdrew: Death | 1 | 1 |
| Withdrew: Withdrawal by subject | 1 | 1 |
| Withdrew: Lost to follow-up | 1 | 1 |
Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.
| Percentage of participants | ATIR101 | PTCy |
|---|---|---|
| Graft-versus-host Disease-free, Relapse-free Survival (GRFS) | 25 (8 to 47) | 62 (41 to 78) |
OS is defined as the time from HSCT until death from any cause. Kaplan-Meier estimates (percentage of participants) of OS were calculated at 24 months post HSCT.
| percentage of participants | ATIR101 | PTCy |
|---|---|---|
| Overall Survival (OS) | 49 (24 to 71) | 77 (55 to 89) |
Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
| percentage of participants | ATIR101 | PTCy |
|---|---|---|
| Progression-free Survival (PFS) | 44 (20 to 66) | 73 (52 to 86) |
Time from randomization to death due to disease relapse or disease progression
No measurements were reported for this outcome.
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). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
| percentage of participants | ATIR101 | PTCy |
|---|---|---|
| Transplant-related Mortality (TRM) | 44 (19 to 66) | 15 (5 to 31) |
Time to CD3+ \> 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
No measurements were reported for this outcome.
Viral, fungal, and bacterial infections
No measurements were reported for this outcome.
Viral, fungal, and bacterial infections
No measurements were reported for this outcome.
Quality of life: Foundation for the Accreditation of Cellular Therapy - Bone Marrow Transplantation questionnaire (FACT-BMT)
No measurements were reported for this outcome.
Quality of life: Short Form 36-item health survey (SF-36)
No measurements were reported for this outcome.
Quality of life: MD Anderson Symptom Inventory (MDASI)
No measurements were reported for this outcome.
Quality of life: EQ-5D-5L
No measurements were reported for this outcome.
Collected over Long term safety follow-up, 24 months.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| ATIR101 | 8/16 (50%) | 13/16 (81.3%) | 0/16 (0%) |
| PTCy | 6/27 (22.2%) | 19/27 (70.4%) | 0/27 (0%) |
| Event | ATIR101 | PTCy |
|---|---|---|
| Infections and infestationsInfections and infestations | 10/16 | 12/27 |
| Blood and lymphatic system disordersBlood and lymphatic system disorders | 5/16 | 5/27 |
| General disorders and administration site conditionsGeneral disorders | 4/16 | 6/27 |
| Nervous system disordersNervous system disorders | 3/16 | 0/27 |
| Cardiac disordersCardiac disorders | 2/16 | 5/27 |
| Respiratory, thoracic and mediastinal disordersRespiratory, thoracic and mediastinal disorders | 2/16 | 1/27 |
| Injury, poisoning and procedural complicationsInjury, poisoning and procedural complications | 2/16 | 0/27 |
| Metabolism and nutrition disordersMetabolism and nutrition disorders | 0/16 | 3/27 |
| InvestigationsInvestigations | 0/16 | 2/27 |
| Gastrointestinal disordersGastrointestinal disorders | 0/16 | 1/27 |
| Age, Continuous(years) | ATIR101 | PTCy | Total |
|---|---|---|---|
| Mean | 36.2 ± 9.63 | 32.8 ± 13.20 | 34.5 ± 11.42 |
| Sex: Female, Male(Participants) | ATIR101 | PTCy | Total |
|---|---|---|---|
| Female | 10 | 20 | 30 |
| Male | 6 | 7 | 13 |
| Ethnicity (NIH/OMB)(Participants) | ATIR101 | PTCy | Total |
|---|---|---|---|
| Hispanic or Latino | 3 | 1 | 4 |
| Not Hispanic or Latino | 12 | 22 | 34 |
| Unknown or Not Reported | 1 | 4 | 5 |
| Race (NIH/OMB)(Participants) | ATIR101 | PTCy | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 1 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 1 | 1 |
| White | 14 | 23 | 37 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 2 | 2 | 4 |
| Hematologic malignancy(Participants) | ATIR101 | PTCy | Total |
|---|---|---|---|
| Acute lymphatic leukemia (ALL) | 4 | 2 | 6 |
| Acute myeloid leukemia (AML) | 9 | 18 | 27 |
| Myelodysplastic syndrome (MDS) | 3 | 7 | 10 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: No
Ran from Nov 2017 to primary completion in Nov 2021, 47 months.
Results were first posted in May 2022, 6 months after primary completion.
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