| Primary | Incidence of Acute Graft Versus Host Disease (GVHD) Grade III/IV | | The primary study endpoint, grade III/IV acute GVHD within 180 days after HSCT, was met for two patients treated with two doses of ATIR101. Two patients developed grade III/IV acute GVHD within 180 days after HSCT treated with a single dose of ATIR101. | Posted | | Number | | participants | | 180 days post HSCT | | | | ID | Title | Description |
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| OG000 | ATIR101 | 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). 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:
- Total Body Irradiation (TBI) regime
- Non-TBI regime
(See below for details) TBI regime: • Fractionated TBI 200 cGy twice daily for 3 days on Day -10 to -8 (1200 cGy in 6 fractions)
- Fludarabine 30 mg/m2 IV once daily for 5 days on Day -7 to -3
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Anti-thymocyte globulin (ATG; Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
Non-TBI regime: • Fludarabine; 30 mg/m2 IV once daily for 5 days on Day -8 to -4
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Melphalan; 60 mg/m2 IV once daily for 2 days on Day -2 and -1
- ATG (Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
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| | | Title | Denominators | Categories |
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| Single dose of ATIR101, grade III/IV acute GVHD within 180 days after HSCT | | | | Two doses of ATIR101, Grade III/IV acute GVHD within 180 days after HSCT | |
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| Secondary | Incidence and Severity of Acute and Chronic GVHD | | 7 patients in the single dose and 4 patients in the double dose were analysed to make a total of 11 patients analysed. | Posted | | Number | | Participants | | Between 6 and 12 months after HSCT | | | | ID | Title | Description |
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| OG000 | ATIR101 | 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). 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:
- Total Body Irradiation (TBI) regime
- Non-TBI regime
(See below for details) TBI regime: • Fractionated TBI 200 cGy twice daily for 3 days on Day -10 to -8 (1200 cGy in 6 fractions)
- Fludarabine 30 mg/m2 IV once daily for 5 days on Day -7 to -3
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Anti-thymocyte globulin (ATG; Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
Non-TBI regime: • Fludarabine; 30 mg/m2 IV once daily for 5 days on Day -8 to -4
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Melphalan; 60 mg/m2 IV once daily for 2 days on Day -2 and -1
- ATG (Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
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| Secondary | Percentage of Participants Who Achieved T-Cell Reconstitution at 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. | Cumulative incidence estimates of T-cell reconstitution at 6 and 12 months post HSCT were analyzed for the single dose group (N=9) and double dose group (N=6). | Posted | | Number | 95% Confidence Interval | percentage of participants | | 6 and 12 months post HSCT | | | | ID | Title | Description |
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| OG000 | ATIR101 | 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). 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:
- Total Body Irradiation (TBI) regime
- Non-TBI regime
(See below for details) TBI regime: • Fractionated TBI 200 cGy twice daily for 3 days on Day -10 to -8 (1200 cGy in 6 fractions)
- Fludarabine 30 mg/m2 IV once daily for 5 days on Day -7 to -3
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Anti-thymocyte globulin (ATG; Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
Non-TBI regime: • Fludarabine; 30 mg/m2 IV once daily for 5 days on Day -8 to -4
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Melphalan; 60 mg/m2 IV once daily for 2 days on Day -2 and -1
- ATG (Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
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| Secondary | Viral, Fungal, and Bacterial Infections | 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 | | Posted | | Number | | Participants | | From 6 months to 1 year after HSCT | | | | ID | Title | Description |
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| OG000 | ATIR101 | 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). 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:
- Total Body Irradiation (TBI) regime
- Non-TBI regime
(See below for details) TBI regime: • Fractionated TBI 200 cGy twice daily for 3 days on Day -10 to -8 (1200 cGy in 6 fractions)
- Fludarabine 30 mg/m2 IV once daily for 5 days on Day -7 to -3
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Anti-thymocyte globulin (ATG; Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
Non-TBI regime: • Fludarabine; 30 mg/m2 IV once daily for 5 days on Day -8 to -4
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Melphalan; 60 mg/m2 IV once daily for 2 days on Day -2 and -1
- ATG (Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
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| Secondary | 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) | Nine participants received a single dose of ATIR101 and 6 participants received a double dose of ATIR101 | Posted | | Number | 95% Confidence Interval | percentage of participants that died | | 12 months post HSCT | | | | ID | Title | Description |
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| OG000 | ATIR101 | 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). 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:
- Total Body Irradiation (TBI) regime
- Non-TBI regime
(See below for details) TBI regime: • Fractionated TBI 200 cGy twice daily for 3 days on Day -10 to -8 (1200 cGy in 6 fractions)
- Fludarabine 30 mg/m2 IV once daily for 5 days on Day -7 to -3
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Anti-thymocyte globulin (ATG; Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
Non-TBI regime: • Fludarabine; 30 mg/m2 IV once daily for 5 days on Day -8 to -4
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Melphalan; 60 mg/m2 IV once daily for 2 days on Day -2 and -1
- ATG (Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
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| Secondary | Relapse-related Mortality (RRM) | Defined as death due to disease relapse or disease progression | Nine participants received a single dose of ATIR101 and 6 participants received a double dose of ATIR101 | Posted | | Number | 95% Confidence Interval | percentage of participants | | 12 months post HSCT | | | | ID | Title | Description |
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| OG000 | ATIR101 | 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). 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:
- Total Body Irradiation (TBI) regime
- Non-TBI regime
(See below for details) TBI regime: • Fractionated TBI 200 cGy twice daily for 3 days on Day -10 to -8 (1200 cGy in 6 fractions)
- Fludarabine 30 mg/m2 IV once daily for 5 days on Day -7 to -3
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Anti-thymocyte globulin (ATG; Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
Non-TBI regime: • Fludarabine; 30 mg/m2 IV once daily for 5 days on Day -8 to -4
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Melphalan; 60 mg/m2 IV once daily for 2 days on Day -2 and -1
- ATG (Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
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| Secondary | Overall Survival (OS) | Defined as the time from HSCT until death from any cause | Nine participants received a single dose of ATIR101 and 6 participants received a double dose of ATIR101 | Posted | | Number | 95% Confidence Interval | Percentage of participants | | 12 months post HSCT | | | | ID | Title | Description |
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| OG000 | ATIR101 | 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). 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:
- Total Body Irradiation (TBI) regime
- Non-TBI regime
(See below for details) TBI regime: • Fractionated TBI 200 cGy twice daily for 3 days on Day -10 to -8 (1200 cGy in 6 fractions)
- Fludarabine 30 mg/m2 IV once daily for 5 days on Day -7 to -3
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Anti-thymocyte globulin (ATG; Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
Non-TBI regime: • Fludarabine; 30 mg/m2 IV once daily for 5 days on Day -8 to -4
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Melphalan; 60 mg/m2 IV once daily for 2 days on Day -2 and -1
- ATG (Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
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| Secondary | Progression-free Survival (PFS) | Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first | Nine participants received a single dose of ATIR101 and 6 participants received a double dose of ATIR101 | Posted | | Number | 95% Confidence Interval | Percentage of participants | | 12 months post HSCT | | | | ID | Title | Description |
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| OG000 | ATIR101 | 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). 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:
- Total Body Irradiation (TBI) regime
- Non-TBI regime
(See below for details) TBI regime: • Fractionated TBI 200 cGy twice daily for 3 days on Day -10 to -8 (1200 cGy in 6 fractions)
- Fludarabine 30 mg/m2 IV once daily for 5 days on Day -7 to -3
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Anti-thymocyte globulin (ATG; Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
Non-TBI regime: • Fludarabine; 30 mg/m2 IV once daily for 5 days on Day -8 to -4
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Melphalan; 60 mg/m2 IV once daily for 2 days on Day -2 and -1
- ATG (Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
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| Secondary | GVHD-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 | Nine subjects received a single dose of ATIR101 and 6 subjects received a double dose of ATIR101. | Posted | | Number | 95% Confidence Interval | Percentage of participants | | 12 months post HSCT | | | | ID | Title | Description |
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| OG000 | ATIR101 | 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). 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:
- Total Body Irradiation (TBI) regime
- Non-TBI regime
(See below for details) TBI regime: • Fractionated TBI 200 cGy twice daily for 3 days on Day -10 to -8 (1200 cGy in 6 fractions)
- Fludarabine 30 mg/m2 IV once daily for 5 days on Day -7 to -3
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Anti-thymocyte globulin (ATG; Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
Non-TBI regime: • Fludarabine; 30 mg/m2 IV once daily for 5 days on Day -8 to -4
- Thiotepa; 5 mg/kg IV twice daily for 1 day on Day -7
- Melphalan; 60 mg/m2 IV once daily for 2 days on Day -2 and -1
- ATG (Thymoglobulin®); 2.5 mg/kg once daily for 4 days on Day -5 to -2, as a continuous IV infusion for 8 hours. During the course of ATG, patients will receive methylprednisolone 2 mg/kg/day IV.
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