Acute Myeloid LeukemiaPhase 1/2NCT04097301
Study of CAR T-cell Therapy in Acute Myeloid Leukemia and Multiple Myeloma
A Phase 1/2 study testing MLM-CAR44.1 T-cells at day 0 Single intravenous infusion in people with Acute Myeloid Leukemia and Multiple Myeloma, run by AGC Biologics S.p.A. at 3 sites in 2 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 1 year to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia, Multiple Myeloma
- What is being tested?
- MLM-CAR44.1 T-cells at day 0 Single intravenous infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 27 Aug 2019to 18 Jun 2021
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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:
- Adults 18 to 75 (65) years old with AML or MM.
- Children 1 to 17 years old with AML, only in Phase IIa.
- Confirmed diagnosis of AML or MM as follows:
Show all 27Show fewer
- AML: Primary or secondary AML (any subtype except acute promyelocytic leukemia) according to World Health Organization (WHO) classification.
- MM with measurable disease as defined by the International Myeloma Working Group (IMWG).
- Patients with relapse or refractory disease:
- AML patients must be unlikely to benefit from cytotoxic chemotherapy as follows:
- Leukemia refractory to at least 2 induction attempts.
- Leukemia in relapse within 1 year following complete response (CR) after at least 2 induction attempts.
- High-risk leukemia in adults according to 2017 European LeukemiaNet (ELN) in first relapse after a hypomethylating agent or a cycle containing cytarabine at a dose ≥ 1g/sqm a day (e.g. FLAG-IDA), except for FLT3-mutated AML.
- High-risk leukemia in children as defined by the Italian Association of Pediatric Hematology and Oncology (AIEOP).
- 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:
- Proteasome inhibitor
- High-dose alkylating agent if patients less than 70 years old
- Immunomodulatory drug (IMID)
- A monoclonal antibody (i.e. anti CD38 monoclonal antibody)
- 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.
Who cannot join
- At screening: patients must meet none of the following exclusion criteria to be eligible for the study:
- History of or candidate for allogeneic stem cell transplantation.
- Cardiovascular, pulmonary, renal, and hepatic functions that in the judgment of the investigator are insufficient for the patient to undergo investigational CAR T-cell therapy.
- Any history of or suspected current autoimmune disorders (apart from vitiligo, resolved childhood atopic dermatitis, Graves' disease clinically controlled).
- History of rheumatologic disorders requiring specific treatment at any time in the patient's medical history.
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
Italy2 sites
IRCCS San Raffaele
Milan
IRCCS Ospedale Pediatrico Bambino Gesù
Roma
Czechia1 site
Department of Haematooncology, Fakultni Nemocnice
Ostrava, Czech Republic
About this study
Official title: A Phase I-IIa Trial to Assess the Safety and Antitumor Activity of Autologous CD44v6 CAR T-cells in Acute Myeloid Leukemia and Multiple Myeloma Expressing CD44v6
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).
Read the detailed description
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.
- Conditions
- Acute Myeloid Leukemia, Multiple Myeloma
- Being tested
- MLM-CAR44.1 T-cells at day 0 Single intravenous infusion
- Medical subject headings
- Leukemia, Myeloid, Acute, Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MLM-CAR44.1 T-cells at day 0 Single intravenous infusionDrug
Also called CAR-T cells
Lymphodepleting chemotherapy with cyclophosphamide (500 mg/m2) and fludarabine (30 mg/m2) daily from day -5 to day -3.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: MLM-CAR44.1 T-cells at day 0 Single intravenous infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 3 months after T-cell infusion, assessed as day 0
Secondary outcomes (7)
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.
Measured: 1 and 2 months following 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).
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: At the date of the early study termination
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2019
- Study start27 Aug 2019
- Primary completion18 Jun 2021
- Study completion18 Jun 2021
Why it stopped: Inability to close the study in a clinically relevant time frame
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- AGC Biologics S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Horizon 2020 - European Commission
- Responsible party
- Sponsor
- Investigators
- Fabio Ciceri, MD, Principal investigator, IRCCS San Raffaele
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04097301
- Sponsor's protocol ID
- EURE-CART-1
- Other IDs
- 2018-000813-19, 2018
- Status last verified
- Dec 2021
- First submitted
- 5 Aug 2019
- Last updated
- 19 Jan 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 33554732
Study at a glance
From the registry record, last updated by the sponsor on 19 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I-IIa Trial to Assess the Safety and Antitumor Activity of Autologous CD44v6 CAR T-cells in Acute Myeloid Leukemia and Multiple Myeloma Expressing CD44v6
- ClinicalTrials.gov ID
- NCT04097301
- Acronym / sponsor ID
- EURE-CART-1
- Conditions
- Acute Myeloid Leukemia, Multiple Myeloma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Inability to close the study in a clinically relevant time frame
- Enrolment
- 8 participants (actual)
- Lead sponsor
- AGC Biologics S.p.A.Industry (pharma, biotech, devices)
- Collaborators
- Horizon 2020 - European Commission
- Study start
- 27 Aug 2019 (actual)
- Primary completion
- 18 Jun 2021 (actual)
- Study completion
- 18 Jun 2021 (actual)
- First posted
- 20 Sep 2019 (actual)
- Results
- Posted 19 Jan 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MLM-CAR44.1 T-cells infusion | Experimental | Drug: MLM-CAR44.1 T-cells at day 0 Single intravenous infusionPHASE 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 |
Who may qualify
- Ages
- 1 year to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (27 listed)
- 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:
Key exclusion criteria (5 listed)
- At screening: patients must meet none of the following exclusion criteria to be eligible for the study:
- History of or candidate for allogeneic stem cell transplantation.
- Cardiovascular, pulmonary, renal, and hepatic functions that in the judgment of the investigator are insufficient for the patient to undergo investigational CAR T-cell therapy.
- Any history of or suspected current autoimmune disorders (apart from vitiligo, resolved childhood atopic dermatitis, Graves' disease clinically controlled).
- History of rheumatologic disorders requiring specific treatment at any time in the patient's medical history.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MLM-CAR44.1 T-cells at day 0 Single intravenous infusionAlso: CAR-T cellsLymphodepleting chemotherapy with cyclophosphamide (500 mg/m2) and fludarabine (30 mg/m2) daily from day -5 to day -3. | Drug | MLM-CAR44.1 T-cells infusion |
Primary outcome
Phase I: Maximum Tolerated Dose (MTD) and the Recommended Phase IIa Dose of MLM-CAR44.1 T-cells in AML and MM
Time frame: Within 30 days following CAR T-cell infusion, assessed as day 0
MTD established through BOIN design and the dose-limiting toxicities (DLTs) occurring following CAR T-cell infusion.
Phase I: Overall Safety of Treatment With MLM-CAR44.1 T-cells
Time frame: For 30 days following CAR T-cell infusion, assessed as day 0.
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.
Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 3 Months Post-infusion
Time frame: 3 months after infusion (assessed as day 0)
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.
Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 6 Months Post-infusion
Time frame: 6 months after infusion (assessed as day 0)
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
Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 12 Months Post-infusion
Time frame: 12 months after infusion (assessed as day 0)
The absence of replication competent retrovirus (RCR) in blood specimens will be monitored by DNA PCR for the Galv gene.
Phase I: Absence of Replication Competent Retrovirus (RCR) in Blood Specimens: 24 Months Post-infusion
Time frame: 24 months after infusion (assessed as day 0)
The absence of replication competent retrovirus (RCR) in blood specimens will be monitored by DNA PCR for the Galv gene.
Phase IIa: Hematological Disease Response to MLM-CAR44.1 T-cells in AML.
Time frame: 2 months after MLM-CAR44.1 T-cell infusion, assessed as day 0
The hematologic disease response will be classified according to ELN criteria.
Phase IIa: Hematological Disease Response to MLM-CAR44.1 T-cells in MM
Time frame: 3 months after T-cell infusion, assessed as day 0
The hematologic disease response will be classified according to IMWG criteria
7 secondary outcomes
Phase I: Hematologic Disease Response to MLM-CAR44.1 T-cells in AML
Time frame: 1 and 2 months following T-cell infusion, assessed as day 0
Phase I: Hematologic Disease Response to MLM-CAR44.1 T-cells in MM
Time frame: 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
Time frame: 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
Time frame: At day 7, 14, 21, 28, 60, 90, 180 from infusion, assessed as day 0
Phase IIa: Hematologic Disease Response in AML
Time frame: 1, 2 and 6 months after MLM-CAR44.1 T-cell infusion, assessed as day 0.
Phase IIa: Hematologic Disease Response in MM
Time frame: 1, 2 and 6 months after T-cell infusion, assessed as day 0
Phase IIa: Overall Survival (OS)
Time frame: At the date of the early study termination
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33554732Stornaiuolo 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Fabio Ciceri, MD principal investigator
IRCCS San Raffaele
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing MLM-CAR44.1 T-cells at day 0 Single intravenous infusion in people with Acute Myeloid Leukemia and Multiple Myeloma, run by AGC Biologics S.p.A. at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 27 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 19 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Italy, Czechia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Jan 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase I: Maximum Tolerated Dose (MTD) and the Recommended Phase IIa Dose of MLM-CAR44.1 T-cells in AML and MM (time frame: Within 30 days following CAR T-cell infusion, assessed as day 0). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04097301, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jan 2022.