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NCT02999854: HATCY

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

Terminated Phase 3 Results posted Last updated 24 May 2022
What this trial tests

Phase 3 trial testing ATIR101 in Acute Myeloid Leukemia in 63 participants. Terminated before completion.

Timeline
29 November 2017
Primary endpoint
9 November 2021
17 December 2021

Quick facts

Lead sponsorKiadis Pharma
PhasePhase 3
StatusTerminated
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingnone
Primary purposeprevention
Enrollment63
Start date29 November 2017
Primary completion9 November 2021
Estimated completion17 December 2021
Sites42 locations across France, Italy, Netherlands, Belgium, Sweden, United Kingdom, Germany, Israel

Drugs / interventions tested

Conditions studied

Sponsor

Kiadis Pharma — full company profile →

Who can join

Adults 18 to 70, any sex, with Acute Myeloid Leukemia or Acute Lymphoblastic Leukemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Graft-versus-host Disease-free, Relapse-free Survival (GRFS) Primary · 24 months post-HSCT

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.

GroupValue95% CI
ATIR101258 – 47
PTCy6241 – 78
Overall Survival (OS) Secondary · 24 months post-HSCT

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.

GroupValue95% CI
ATIR1014924 – 71
PTCy7755 – 89
Progression-free Survival (PFS) Secondary · 24 months post-HSCT

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.

GroupValue95% CI
ATIR1014420 – 66
PTCy7352 – 86
Transplant-related Mortality (TRM) Secondary · 24 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). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.

GroupValue95% CI
ATIR1014419 – 66
PTCy155 – 31

Adverse events — posted to ClinicalTrials.gov

Time frame: Long term safety follow-up, 24 months.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

ATIR101
Serious: 13/16 (81%)
Deaths: 8/16
PTCy
Serious: 19/27 (70%)
Deaths: 6/27

Serious adverse events (10 terms)

ReactionSystemATIR101PTCy
Infections and infestationsInfections and infestations
General disorders and administration site conditionsGeneral disorders
Blood and lymphatic system disordersBlood and lymphatic system disorders
Cardiac disordersCardiac disorders
Metabolism and nutrition disordersMetabolism and nutrition disorders
Nervous system disordersNervous system disorders
Respiratory, thoracic and mediastinal disordersRespiratory, thoracic and mediastinal disorders
Injury, poisoning and procedural complicationsInjury, poisoning and procedural complications
InvestigationsInvestigations
Gastrointestinal disordersGastrointestinal disorders

Most-reported serious reactions: Infections and infestations, General disorders and administration site conditions, Blood and lymphatic system disorders, Cardiac disorders, Metabolism and nutrition disorders, Nervous system disorders, Respiratory, thoracic and mediastinal disorders, Injury, poisoning and procedural complications.

Data from ClinicalTrials.gov NCT02999854 adverse events section.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Cell therapies in the clinic.
    Wang LL, Janes ME, Kumbhojkar N, Kapate N, et al · · 2021 · cited 99× · PMID 34027097 · DOI 10.1002/btm2.10214
  2. Immune Reconstitution After Allogeneic Haematopoietic Cell Transplantation: From Observational Studies to Targeted Interventions.
    Yanir A, Schulz A, Lawitschka A, Nierkens S, et al · · 2021 · cited 35× · PMID 35087775 · DOI 10.3389/fped.2021.786017
  3. ATIR101 administered after T-cell-depleted haploidentical HSCT reduces NRM and improves overall survival in acute leukemia.
    Roy DC, Walker I, Maertens J, Lewalle P, et al · · 2020 · cited 21× · PMID 32047237 · DOI 10.1038/s41375-020-0733-0
  4. Technology forecast: advanced therapies in late clinical research, EMA approval or clinical application via hospital exemption.
    Eder C, Wild C. · · 2019 · cited 19× · PMID 31069029 · DOI 10.1080/20016689.2019.1600939
  5. Concise Review: Boosting T-Cell Reconstitution Following Allogeneic Transplantation-Current Concepts and Future Perspectives.
    Simons L, Cavazzana M, André I. · · 2019 · cited 17× · PMID 30887712 · DOI 10.1002/sctm.18-0248
  6. Allodepleted T-cell immunotherapy after haploidentical haematopoietic stem cell transplantation without severe acute graft-versus-host disease (GVHD) in the absence of GVHD prophylaxis.
    Roy DC, Lachance S, Cohen S, Delisle JS, et al · · 2019 · cited 15× · PMID 31135970 · DOI 10.1111/bjh.15970
  7. T-cell depleted haploidentical hematopoietic cell transplantation for pediatric malignancy.
    Takahashi T, Prockop SE. · · 2022 · cited 9× · PMID 36313879 · DOI 10.3389/fped.2022.987220
  8. Proceedings From the Fourth Haploidentical Stem Cell Transplantation Symposium (HAPLO2016), San Diego, California, December 1, 2016.
    Al Malki MM, Jones R, Ma Q, Lee D, et al · · 2018 · cited 9× · PMID 29339270 · DOI 10.1016/j.bbmt.2018.01.008

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