E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with a hematologic malignancy (AML, ALL, or MDS) who are eligible for a haploidentical HSCT |
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E.1.1.1 | Medical condition in easily understood language |
Patients with blood cancer who are eligible for a stem cell transplantation from a related haploidentical donor |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000845 |
E.1.2 | Term | Acute lymphoblastic leukemia |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10059044 |
E.1.2 | Term | Allogeneic peripheral hematopoietic stem cell transplant |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10027703 |
E.1.2 | Term | Mismatched donor bone marrow transplantation therapy |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10000880 |
E.1.2 | Term | Acute myeloid leukaemia |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028533 |
E.1.2 | Term | Myelodysplastic syndrome |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To study the safety and efficacy of ATIR at a dose of 2x10E6 viable T-cells/kg body weight in patients with a hematologic malignancy who received a haploidentical hematopoietic stem cell transplant (HSCT). |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• 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 • Eligible for haploidentical stem cell transplantation according to the investigator • Male or female, age ≥ 18 years and ≤ 65 years |
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E.4 | Principal 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) < 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 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 |
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E.5 End points |
E.5.1 | Primary end point(s) |
Transplant-related mortality (TRM) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
• Incidence and severity of acute and chronic graft versus host disease (GvHD) • Immune reconstitution • Incidence and severity of viral, fungal, and bacterial infections • TRM, relapse-related mortality (RRM), overall survival (OS), and progression-free survival (PFS)
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to 12-24 months post HSCT |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 6 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Belgium |
Canada |
Germany |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The end of the study is defined as the date at which the last data point from the last patient is received. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |