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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-000813-19 | EudraCT Number |
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Inability to close the study in a clinically relevant time frame
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| Name | Class |
|---|---|
| Horizon 2020 - European Commission | OTHER |
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The purpose of this first-in-man Phase I-IIa study is to evaluate the safety and antitumor activity of autologous CD44v6 CAR T-cells in patients with acute myeloid leukemia (AML) and multiple myeloma (MM).
The study is a seamless Phase I/IIa, open-label, multicenter clinical trial that combines Phase I dose escalation based on toxicity with Phase IIa dose expansion based on antitumor activity. Considering the "first in human" nature of this clinical study, the Bayesian Optimal Interval Design (BOIN) has been chosen to minimize any risks of exposure to the novel CD44v6 CAR T-cells during dose escalation. The study population is made up of patients with relapsed/refractory AML or MM expressing CD44v6.
The medicinal product under investigation (MLM-CAR44.1 T-cells) is patient specific as it is prepared starting from lymphocytes of the patient collected through lymphocyte apheresis. These autologous T-cells are expanded in vitro in large numbers and genetically modified to express the CAR CD44v6ΔNL gene and thus acquire antitumor functions. As a safety feature, the MLM-CAR44.1 T-cells are genetically modified to also express the HSV-TK Mut2 gene (suicide gene), which can be selectively activated in case of severe toxicity through the administration of ganciclovir (GCV), leading to the death of proliferating CAR T-cells.
The aim of this study is to assess the safety, antitumor activity and feasibility of CD44v6 CAR T cell immunotherapy in AML and MM.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MLM-CAR44.1 T-cells infusion | Experimental | PHASE I: i.v. single dose of MLM-CAR44.1 T-cells: 0.5 x 10E6/Kg or 1 x 10E6/Kg or 2 x10E6/Kg according to the BOIN design. PHASE IIa: i.v. single dose of MLM-CAR44.1 T-cells corresponding to the maximum tolerated dose (MTD). Phase I and IIa Pre-treatment: lymphodepleting chemotherapy with cyclophosphamide (500 mg/m2) and fludarabine (30 mg/m2) daily from day -5 to day -3 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MLM-CAR44.1 T-cells at day 0 Single intravenous infusion | Drug | Lymphodepleting chemotherapy with cyclophosphamide (500 mg/m2) and fludarabine (30 mg/m2) daily from day -5 to day -3. |
| Measure | Description | Time Frame |
|---|---|---|
| Phase I: Maximum Tolerated Dose (MTD) and the Recommended Phase IIa Dose of MLM-CAR44.1 T-cells in AML and MM | MTD established through BOIN design and the dose-limiting toxicities (DLTs) occurring following CAR T-cell infusion. | Within 30 days following CAR T-cell infusion, assessed as day 0 |
| Phase I: Overall Safety of Treatment With MLM-CAR44.1 T-cells | Safety will be evaluated by analyzing the type, frequency and severity of adverse events (AE) and by monitoring for systemic reactions (fever, tachycardia, nausea and vomiting, joint pain, skin rash). Overall, 3 study emergent serious adverse events (SAEs) were reported in patients treated with MLM-CAR44.1 T-cells. | For 30 days following CAR T-cell infusion, assessed as day 0. |
| Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 3 Months Post-infusion | The absence of replication competent retrovirus (RCR) in blood specimens: 3 months post-infusion will be monitored by DNA PCR for the Galv gene. RCR search was conducted centrally using a quantitative molecular test, (real time quantitative PCR, q-PCR analysis) determining the absence of RCR by DNA PCR for the Galv and gag-pol genes on genomic DNA from patient's peripheral blood lymphocytes. The objective of this q-PCR analysis is to exclude the presence of RCR originated by recombination with PG13 packaging cell sequences by detecting the Galv and gag-pol transcripts in the transduced cells. The absence of the Galv and gag-pol transcripts can exclude the presence of an RCR, while its presence is not sufficient to indicate the presence of an RCR, and in this case further analysis is required. | 3 months after infusion (assessed as day 0) |
| Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 6 Months Post-infusion | The absence of replication competent retrovirus (RCR) in blood specimens will be monitored by DNA PCR for the Galv gene. RCR search was conducted centrally using a quantitative molecular test, (real time quantitative PCR, q-PCR analysis) determining the absence of RCR by DNA PCR for the Galv and gag-pol genes on genomic DNA from patient's peripheral blood lymphocytes. The objective of this q-PCR analysis is to exclude the presence of RCR originated by recombination with PG13 packaging cell sequences by detecting the Galv and gag-pol transcripts in the transduced cells. The absence of the Galv and gag-pol transcripts can exclude the presence of an RCR, while its presence is not sufficient to indicate the presence of an RCR, and in this case further analysis is required |
| Measure | Description | Time Frame |
|---|---|---|
| Phase I: Hematologic Disease Response to MLM-CAR44.1 T-cells in AML | The hematologic disease response will be classified with the following response criteria: complete response (CR), incomplete response (CRi) and partial response (PR) according to ELN criteria. | 1 and 2 months following T-cell infusion, assessed as day 0 |
| Measure | Description | Time Frame |
|---|---|---|
| Exploratory Outcome of Phase IIa: Percentages of Patients With Minimal Residual Disease | AML: proportion of patients with a molecular complete response (CR); MM: proportion of patients with a molecular CR. | AML: 2 months after infusion, assessed as day 0. MM: 3 months after infusion, assessed as day 0 |
Inclusion Criteria:
Patients must meet all the following inclusion criteria to be eligible for the study.
Written informed consent before any study-related procedure.
Adults and children:
Confirmed diagnosis of AML or MM as follows:
Patients with relapse or refractory disease:
AML patients must be unlikely to benefit from cytotoxic chemotherapy as follows:
Patients with MM must have a relapse or refractory disease after at least 4 different prior treatments in 3 treatment lines, or 4 treatments in 2 treatment lines in case of early relapsing patients (relapse in less than 1.5 years). Treatments include:
Positive CD44v6 expression on tumor cells by flow cytometry.
Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
Life expectancy of at least 12 weeks.
Adequate organ function (hepatic, cardiac, pulmonary).
Recovery from toxicities of clinical consequence attributed to previous chemotherapy to CTCAE v5.0 Grade 1 (i.e., certain toxicities such as alopecia will not be considered in this category).
Ability to comply with study procedures, including hospitalization and protocol-specified acquisition of blood and/or bone marrow specimens.
Willing to be followed up long term, i.e. a 15-year follow up as required by health authorities for cell and gene therapy products.
Women of childbearing potential must test negative for pregnancy at enrolment and during the study.
Exclusion Criteria:
At screening: patients must meet none of the following exclusion criteria to be eligible for the study:
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| Name | Affiliation | Role |
|---|---|---|
| Fabio Ciceri, MD | IRCCS San Raffaele | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Department of Haematooncology, Fakultni Nemocnice | Ostrava | Czech Republic | Czechia | |||
| IRCCS San Raffaele |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33554732 | Derived | Stornaiuolo A, Valentinis B, Sirini C, Scavullo C, Asperti C, Zhou D, Martinez De La Torre Y, Corna S, Casucci M, Porcellini S, Traversari C. Characterization and Functional Analysis of CD44v6.CAR T Cells Endowed with a New Low-Affinity Nerve Growth Factor Receptor-Based Spacer. Hum Gene Ther. 2021 Jul;32(13-14):744-760. doi: 10.1089/hum.2020.216. Epub 2021 May 5. |
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The recruited patient population, due to the early termination of the clinical study, was made up only of patients affected by multiple myeloma (MM), whereas AML patients were not enrolled.
Eight patients, 6 males and 2 females were enrolled in 2 centers. Two patients received the investigational product (MLM-CAR44.1 T-cells), while 6 patients did not. Age ranged from 41 to 66 years, with a median of 56 years.
The study population originally planned is made up of: Adult patients 18 to 75 years old or 18 to 63 years old for CZ site, with relapsed/refractory AML or MM expressing CD44v6; Children 1 to 17 years old with AML, only in Phase IIa, except for CZ site. Up to the early termination of the study only adult patients 18 to 75 years old or 18 to 63 years old for CZ site, with relapsed/refractory MM expressing CD44v6 were recruited
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| ID | Title | Description |
|---|---|---|
| FG000 | MLM-CAR44.1 T-cells Infusion | Patients were scheduled to receive a single intravenous (iv) infusion of CD44v6.CAR-transduced autologous lymphocytes at day 0, after lymphodepleting chemotherapy with cyclophosphamide iv (500 mg/m2) and fludarabine iv (30 mg/m2) performed daily from day -5 to day -3 The dose of iv infused MLM-CAR44.1 T-cells is: PHASE I: 0.5x10E6/Kg or 1x10E6/Kg or 2x10E6/Kg according to the BOIN design. PHASE IIa: a dose of MLM-CAR44.1 T-cells corresponding to the maximum tolerated dose (MTD). |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Overall, 8 patients were enrolled in the study and underwent the screening phase to evaluate the compliance to the clinical inclusion / exclusion criteria as well as the CD44v6 expression by tumor cells (Table 10).
Of the 8 enrolled patients, 2 were treated with MLM-CAR44.1 T-cell at the first dose level. Five patients were screening failures and 1 patient dropped-out before lymphocyte apheresis for fulminant disease progression.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | MLM-CAR44.1 T-cells Infusion | Patients were scheduled to receive a single intravenous (iv) infusion of CD44v6.CAR-transduced autologous lymphocytes at day 0, after lymphodepleting chemotherapy with cyclophosphamide iv (500 mg/m2) and fludarabine iv (30 mg/m2) performed daily from day -5 to day -3 The dose of iv infused MLM-CAR44.1 T-cells is: PHASE I: 0.5x10E6/Kg or 1x10E6/Kg or 2x10E6/Kg according to the BOIN design. PHASE IIa: a dose of MLM-CAR44.1 T-cells corresponding to the maximum tolerated dose (MTD). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Phase I: Maximum Tolerated Dose (MTD) and the Recommended Phase IIa Dose of MLM-CAR44.1 T-cells in AML and MM | MTD established through BOIN design and the dose-limiting toxicities (DLTs) occurring following CAR T-cell infusion. | Data were not collected due to early end of trial | Posted | Within 30 days following CAR T-cell infusion, assessed as day 0 |
|
15 months
All SAEs and AEs are collected regardless of their relationship with the experimental product. Laboratory abnormalities that constitute an AE should be recorded on the Adverse Events page of the eCRF. Whenever possible, a diagnosis, rather than a symptom should be provided. Laboratory abnormalities that meet the criteria for Adverse Events should be followed until they have returned to normal or an adequate explanation of the abnormality is found.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | MLM-CAR44.1 T-cells Infusion | Patients were scheduled to receive a single intravenous (iv) infusion of CD44v6.CAR-transduced autologous lymphocytes at day 0, after lymphodepleting chemotherapy with cyclophosphamide iv (500 mg/m2) and fludarabine iv (30 mg/m2) performed daily from day -5 to day -3 The dose of iv infused MLM-CAR44.1 T-cells is: PHASE I: 0.5x10E6/Kg or 1x10E6/Kg or 2x10E6/Kg according to the BOIN design. PHASE IIa: a dose of MLM-CAR44.1 T-cells corresponding to the maximum tolerated dose (MTD). |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | Immune system disorders | Systematic Assessment |
The early termination of this study is due to the analysis of its feasibility, based on the low patient recruitment rate (due to the lower-than-expected proportion of myeloma and leukemia expressing the CD44v6). A further decrease in the recruitment occurred with the diffusion of the COVID-19 emergency and the availability of new drugs for the treatment of myeloma and leukemia. These reasons make impossible to foresee the conclusion of the study in a clinically relevant time frame.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Anna Stornaiuolo | AGC Biologics | 0221277440 | astornaiuolo@agcbio.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jan 8, 2020 | Oct 4, 2021 | Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D015470 | Leukemia, Myeloid, Acute |
| D009101 | Multiple Myeloma |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| D016219 | Immunotherapy, Adoptive |
| ID | Term |
|---|---|
| D019264 | Adoptive Transfer |
| D007116 | Immunization, Passive |
| D007114 | Immunization |
| D007167 | Immunotherapy |
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|
| 6 months after infusion (assessed as day 0) |
| Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 12 Months Post-infusion | The absence of replication competent retrovirus (RCR) in blood specimens will be monitored by DNA PCR for the Galv gene. | 12 months after infusion (assessed as day 0) |
| Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 24 Months Post-infusion | The absence of replication competent retrovirus (RCR) in blood specimens will be monitored by DNA PCR for the Galv gene. | 24 months after infusion (assessed as day 0) |
| Phase IIa: Hematological Disease Response to MLM-CAR44.1 T-cells in AML. | The hematologic disease response will be classified according to ELN criteria. | 2 months after MLM-CAR44.1 T-cell infusion, assessed as day 0 |
| Phase IIa: Hematological Disease Response to MLM-CAR44.1 T-cells in MM | The hematologic disease response will be classified according to IMWG criteria | 3 months after T-cell infusion, assessed as day 0 |
| Phase I: Hematologic Disease Response to MLM-CAR44.1 T-cells in MM |
Hematologic disease response evaluated at Day 28 following CART-cell infusion, as overall response rate (ORR), stringent complete response (sCR), CR, very good partial response (VGPR) and partial response (PR). |
| 1 and 3 months following T-cell infusion, assessed as day 0 |
| Phase I: The Levels of Circulating MLM-CAR44.1 T-cells in Blood Samples | The levels will be evaluated by flow cytometry and qPCR (in vivo pharmacokinetic profile). | At day 7, 14, 21, 28, 60, 90, 180 from infusion, assessed as day 0 |
| Phase I: The Percentage of Patients for Whom Activation of Suicide Gene Was Needed | Suicide gene activation and elimination of transduced cells will be established through administration of ganciclovir in case of cytokine-release syndrome (CRS) and other MLM-CAR44.1 T-cell related toxicities. | At day 7, 14, 21, 28, 60, 90, 180 from infusion, assessed as day 0 |
| Phase IIa: Hematologic Disease Response in AML | The hematologic disease response will be classified with the following response criteria: complete response (CR), incomplete response (CRi) and partial response (PR) according to ELN criteria. | 1, 2 and 6 months after MLM-CAR44.1 T-cell infusion, assessed as day 0. |
| Phase IIa: Hematologic Disease Response in MM | The hematologic disease response will be defined based on the overall response rate (ORR): stringent complete response (sCR), complete response (CR), very good partial response (VGPR), and partial response (PR) according to IMWG criteria | 1, 2 and 6 months after T-cell infusion, assessed as day 0 |
| Phase IIa: Overall Survival (OS) | Overall Survival (OS) is defined as the time from the date of MLM-CAR44.1 T-cell infusion to the date of last follow-up or death due to any cause, whichever occurs first. One patients out of the 2 treated was still alive at the date of the early study termination. One patient died after EURE-CART-1 cell infusion, at day 121, for disease progression. | At the date of the early study termination |
| Milan |
| Italy |
| IRCCS Ospedale Pediatrico Bambino Gesù | Roma | Italy |
| Participants |
|
| Age, Continuous | Median | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Confirmed diagnosis of MM | Count of Participants | Participants |
|
| Patients with relapse or refractory disease | Count of Participants | Participants |
|
|
| Primary | Phase I: Overall Safety of Treatment With MLM-CAR44.1 T-cells | Safety will be evaluated by analyzing the type, frequency and severity of adverse events (AE) and by monitoring for systemic reactions (fever, tachycardia, nausea and vomiting, joint pain, skin rash). Overall, 3 study emergent serious adverse events (SAEs) were reported in patients treated with MLM-CAR44.1 T-cells. | Posted | Number | Number of SAEs | For 30 days following CAR T-cell infusion, assessed as day 0. |
|
|
|
| Primary | Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 3 Months Post-infusion | The absence of replication competent retrovirus (RCR) in blood specimens: 3 months post-infusion will be monitored by DNA PCR for the Galv gene. RCR search was conducted centrally using a quantitative molecular test, (real time quantitative PCR, q-PCR analysis) determining the absence of RCR by DNA PCR for the Galv and gag-pol genes on genomic DNA from patient's peripheral blood lymphocytes. The objective of this q-PCR analysis is to exclude the presence of RCR originated by recombination with PG13 packaging cell sequences by detecting the Galv and gag-pol transcripts in the transduced cells. The absence of the Galv and gag-pol transcripts can exclude the presence of an RCR, while its presence is not sufficient to indicate the presence of an RCR, and in this case further analysis is required. | Posted | Count of Participants | Participants | 3 months after infusion (assessed as day 0) |
|
|
|
| Primary | Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 6 Months Post-infusion | The absence of replication competent retrovirus (RCR) in blood specimens will be monitored by DNA PCR for the Galv gene. RCR search was conducted centrally using a quantitative molecular test, (real time quantitative PCR, q-PCR analysis) determining the absence of RCR by DNA PCR for the Galv and gag-pol genes on genomic DNA from patient's peripheral blood lymphocytes. The objective of this q-PCR analysis is to exclude the presence of RCR originated by recombination with PG13 packaging cell sequences by detecting the Galv and gag-pol transcripts in the transduced cells. The absence of the Galv and gag-pol transcripts can exclude the presence of an RCR, while its presence is not sufficient to indicate the presence of an RCR, and in this case further analysis is required | Posted | Count of Participants | Participants | 6 months after infusion (assessed as day 0) |
|
|
|
| Primary | Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 12 Months Post-infusion | The absence of replication competent retrovirus (RCR) in blood specimens will be monitored by DNA PCR for the Galv gene. | Data were not collected due to early end of trial | Posted | 12 months after infusion (assessed as day 0) |
|
|
| Primary | Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 24 Months Post-infusion | The absence of replication competent retrovirus (RCR) in blood specimens will be monitored by DNA PCR for the Galv gene. | Data were not collected due to early end of trial | Posted | 24 months after infusion (assessed as day 0) |
|
|
| Primary | Phase IIa: Hematological Disease Response to MLM-CAR44.1 T-cells in AML. | The hematologic disease response will be classified according to ELN criteria. | Data were not collected because Phase IIa was not performed due to early end of trial | Posted | 2 months after MLM-CAR44.1 T-cell infusion, assessed as day 0 |
|
|
| Primary | Phase IIa: Hematological Disease Response to MLM-CAR44.1 T-cells in MM | The hematologic disease response will be classified according to IMWG criteria | Data were not collected because Phase IIa was not performed due to early end of trial | Posted | Count of Participants | Participants | 3 months after T-cell infusion, assessed as day 0 |
|
|
|
| Secondary | Phase I: Hematologic Disease Response to MLM-CAR44.1 T-cells in AML | The hematologic disease response will be classified with the following response criteria: complete response (CR), incomplete response (CRi) and partial response (PR) according to ELN criteria. | Data were not collected because no AML patients were enrolled | Posted | 1 and 2 months following T-cell infusion, assessed as day 0 |
|
|
| Secondary | Phase I: Hematologic Disease Response to MLM-CAR44.1 T-cells in MM | Hematologic disease response evaluated at Day 28 following CART-cell infusion, as overall response rate (ORR), stringent complete response (sCR), CR, very good partial response (VGPR) and partial response (PR). | Hematologic disease response of the two treated patients was evaluated at Day 28 following CART-cell infusion. Both patients showed a progressive disease (PD) and no sign of response. | Posted | Count of Participants | Participants | 1 and 3 months following T-cell infusion, assessed as day 0 |
|
|
|
| Secondary | Phase I: The Levels of Circulating MLM-CAR44.1 T-cells in Blood Samples | The levels will be evaluated by flow cytometry and qPCR (in vivo pharmacokinetic profile). | Posted | Count of Participants | Participants | At day 7, 14, 21, 28, 60, 90, 180 from infusion, assessed as day 0 |
|
|
|
| Secondary | Phase I: The Percentage of Patients for Whom Activation of Suicide Gene Was Needed | Suicide gene activation and elimination of transduced cells will be established through administration of ganciclovir in case of cytokine-release syndrome (CRS) and other MLM-CAR44.1 T-cell related toxicities. | Data were not collected because no MLM-CAR44.1 T-cell related toxicities occurred | Posted | At day 7, 14, 21, 28, 60, 90, 180 from infusion, assessed as day 0 |
|
|
| Secondary | Phase IIa: Hematologic Disease Response in AML | The hematologic disease response will be classified with the following response criteria: complete response (CR), incomplete response (CRi) and partial response (PR) according to ELN criteria. | Data were not collected because Phase IIa was not performed due to early end of trial | Posted | 1, 2 and 6 months after MLM-CAR44.1 T-cell infusion, assessed as day 0. |
|
|
| Secondary | Phase IIa: Hematologic Disease Response in MM | The hematologic disease response will be defined based on the overall response rate (ORR): stringent complete response (sCR), complete response (CR), very good partial response (VGPR), and partial response (PR) according to IMWG criteria | Data were not collected because Phase IIa was not performed due to early end of trial | Posted | 1, 2 and 6 months after T-cell infusion, assessed as day 0 |
|
|
| Secondary | Phase IIa: Overall Survival (OS) | Overall Survival (OS) is defined as the time from the date of MLM-CAR44.1 T-cell infusion to the date of last follow-up or death due to any cause, whichever occurs first. One patients out of the 2 treated was still alive at the date of the early study termination. One patient died after EURE-CART-1 cell infusion, at day 121, for disease progression. | Data were not collected because Phase IIa was not performed due to early end of trial | Posted | At the date of the early study termination |
|
|
| Other Pre-specified | Exploratory Outcome of Phase IIa: Percentages of Patients With Minimal Residual Disease | AML: proportion of patients with a molecular complete response (CR); MM: proportion of patients with a molecular CR. | Data were not collected because Phase IIa was not performed due to early end of trial | Posted | AML: 2 months after infusion, assessed as day 0. MM: 3 months after infusion, assessed as day 0 |
|
|
| 1 |
| 2 |
| 2 |
| 2 |
| 2 |
| 2 |
| Disease progression | General disorders | Systematic Assessment |
|
| Neutropenia | Blood and lymphatic system disorders | Systematic Assessment |
|
| Anemia | Blood and lymphatic system disorders | Systematic Assessment |
|
The Company will keep the information related to the trial in the strictest confidence and won't disclose such confidential information to third parties without the written consent of the Sponsor. It warrants that the obligation of confidentiality will be extended to the PI, his collaborators and to any other person who may become aware of confidential data. These obligations will remain in effect until the information is released into the public domain by the by the Sponsor.
| D006402 |
| Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D054219 | Neoplasms, Plasma Cell |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D010265 | Paraproteinemias |
| D001796 | Blood Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D056747 |
| Immunomodulation |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D007158 | Immunologic Techniques |
| D008919 | Investigative Techniques |