Lymphoblastic LeukemiaPhase 1/2NCT05429905
Dual Anti-CD22/CD19 Chimeric Antigen Receptor-directed T Cells (CART2219.1) for Relapsed Refractory B-Lineage Leukaemia
A Phase 1/2 study testing Phase I and Phase II in people with Lymphoblastic Leukemia and Lymphoblastic Leukemia, Acute, Childhood, run by KK Women's and Children's Hospital at 1 site in Singapore.
Trial summary
The purpose of this study is to deliver dual-targeting CAR-T cell therapy (CART 2219.1) as a salvage treatment to patients with relapsed/refractory B-lineage leukaemia in place of stem cell transplant or irradiation. Read more about this study
- Who is it for?
- Ages 2 to 75 years
- Conditions the study is for
- Lymphoblastic Leukemia, Lymphoblastic Leukemia, Acute, Childhood, Lymphoblastic Leukemia in Children
- What is being tested?
- Phase I and Phase II
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteSingapore
- Study dates
- 18 Jul 2022to Dec 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 2 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- All Cohorts:
- Age 2 to 75 years
- Absolute blood CD3+ T cell count ≥100/μl
- ECOG performance score of ≤2 if >16 years old, or Lansky performance score of >50 if ≤16 years old at screening
- Patients and/or parents must give their written informed consent/assent.
- Patients have relapsed/refractory B-lineage acute lymphoblastic leukaemia, includes persistent minimal residual disease (MRD)
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- Cohort 1 (Phase I): Relapsed/Refractory B-ALL
- Patients must have relapsed/refractory (r/r) B-lineage ALL meeting one of the following disease-specific criteria:
- Patients with r/r ALL with >5% blasts in BM (M2 or M3) after at least one standard chemotherapy and one salvage regimen who are ineligible for allogeneic stem cell transplant (alloSCT) or have refractory disease activity (e.g. persistent MRD in bone marrow) precluding alloSCT, or
- Patients with Ph+ ALL if they are intolerant to tyrosine kinase inhibitor (TKI) therapy, or if they have r/r disease after treatment including TKI, or
- Patients who have relapsed post-allogeneic HSCT and are at least 100 days post-transplant, with no evidence of active GVHD, > 6 weeks post donor lymphocyte infusion (DLI) and no longer taking immunosuppressive agents for at least 30 days prior to enrolment.
- Patients who have relapsed after prior CAR-T therapy who are not eligible for stem cell transplant and have < 5% circulating CAR-T prior to apheresis
- Patients with refractory/relapsed combined extramedullary ALL are eligible. This includes patients with combined CNS-2 (<5 WBC/μl CSF, with blasts on cytospin) or CNS-3 (>5 WBC/ul CSF, with blasts on cytospin) disease and patients with combined testicular relapse.
- Cohort 2 (Phase II): B-ALL with persistent MRD (High Risk B-ALL)
- Patients must have persistent MRD >0.1% blasts after frontline induction chemotherapy or >0.01% blasts after consolidation therapy.
- Cohort 3 (Phase II): Relapsed/Refractory Extramedullary B-ALL
- Patients with testicular leukaemia confirmed on biopsy
- Patients with CNS-3 B-ALL or Leptomeningeal disease
- Patients with combined bone marrow and extramedullary relapse (as defined in Cohort 1) are eligible.
Who cannot join
- All Cohorts:
- Blasts are negative for both CD22 and CD19 on flow cytometry or immunohistochemistry, defined as < 10% of blasts staining positive for CD22 and CD19 respectively [IBFM 2016 Consensus Guidelines].
- Current autoimmune disease, or history of autoimmune disease with potential CNS involvement
- Active clinically significant CNS dysfunction (including but not limited to uncontrolled seizure disorders, cerebrovascular ischemia or hemorrhage, dementia, paralysis)
- History of an additional malignancy other than non-melanoma skin cancer or carcinoma in situ unless disease free for ≥3 years.
- Pulmonary function: Patients with pre-existing severe lung disease (FEV1 or FVC < 65%) or an oxygen requirement of >28% O2 supplementation or active pulmonary infiltrates on chest X-ray at the time scheduled for T cell infusion
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- Cardiac function: Fractional shortening <28% or left ventricular ejection fraction <50% by echocardiography
- Renal function: Creatinine clearance <50 mL/min/1.73 m2
- Liver function: Patients with a serum bilirubin >3 times upper limit of normal or an AST or ALT > 5 times upper limit of normal, unless due to leukemic liver infiltration in the estimation of the investigator
- Rapidly progressive disease that in the estimation of the investigator would compromise ability to complete study therapy
- Active Hepatitis B (HBsAg positive) or Hepatitis C (PCR positive), or known infection with human immunodeficiency virus (HIV)
- Pregnant or nursing (lactating) women
- In relation to prior therapy:
- Use of immunotherapy, cell-based or other investigational treatment within 30 days of CAR-T infusion
Trial locations and contacts
1 site in 1 country.
Singapore1 site, 1 recruiting
- Recruiting
KK Women's and Children's hospital
Singapore
Michaela Seng, +65 6394 1989, michaela.seng@singhealth.com.sg
Michaela Seng
About this study
The purpose of this study is to deliver dual-targeting CAR-T cell therapy (CART 2219.1) as a salvage treatment to patients with relapsed/refractory B-lineage leukaemia in place of stem cell transplant or irradiation.
- Conditions
- Lymphoblastic Leukemia, Lymphoblastic Leukemia, Acute, Childhood, Lymphoblastic Leukemia in Children, Lymphoblastic Leukemia, Acute Adult, CAR
- Being tested
- Phase I, Phase II
- Keywords
- CAR T-cell therapy, B-ALL, Relapsed/ Refractory
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Phase IBiological
- Dose level 0: CAR+CD3+ 0.4 x 10e6/kg (de-escalation dose)
- Dose level 1: CAR+CD3+ 1 x 10e6/kg (starting dose)
- Dose level 2: CAR+CD3+ 2.5 x 10e6/kg.
Dose-escalations for adults and paediatrics will be performed in 2 independent strata for determination of RP2D.
Phase IIBiological
RP2D will be determined in Phase I
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (Dose-escalation) | Experimental | Dose-finding and dose expansion cohort for intravenous autologous anti-CD22/CD19 CAR-T using a relapsed refractory B-ALL cohort. Receives: Biological: Phase I |
| Cohort 2 (High MRD) | Experimental | Patients with B-ALL with high MRD after induction therapy or after consolidation therapy in replacement of stem cell transplant Receives: Biological: Phase II |
| Cohort 3 (Extramedullary ALL) | Experimental | Patients with testicular or central nervous system B-ALL in replacement of radiation Receives: Biological: Phase II |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recommended Phase II Dose (Phase I, Cohort 1)
The RP2D will be the dose level at which < 1 DLT in 3 patients or < 2 DLT in 6 patients is observed in both the adult and paediatric dose escalation stratas.
Measured: 30 days
Number of patients with dose-limiting toxicities (Phase I, Cohort 1)
To be DLTs, adverse events must be suspected to be secondary to CAR-T cell infusion, occur during the first 30 days after infusion and meet at least one of the following criteria:
- Grade ≥ 3 non-hematologic toxicities, with the following exceptions:
- Laboratory abnormalities without associated symptomatology or clinical consequence that resolve in less than 7 days;
- Grade ≤ 2 cytokine release syndrome;
- Grade 3 cytokine release syndrome that improves to grade ≤ 2 within 3 days of intervention;
- Grade 3 neurotoxicity that resolves to grade 2 or less within 3 days.
- Laboratory abnormalities compatible with tumor lysis syndrome;
- Grade ≥ 4 hematologic toxicities that persist at a grade ≥ 3 for >21 days.
Cytokine release syndrome and immune-effector cell associated neurotoxicity syndrome (ICANS) are graded according to ASTCT consensus grading. Adverse events are graded according to CTCAE version 5.0.
Measured: 30 days
12-month Leukaemia Free Survival (Phase II, Cohort 2 and 3)
Survival with Marrow MRD <0.01% by flow cytometry
Measured: 12 months
Secondary outcomes (4)
Overall Survival Rate (OS)
Percentage of patients in the study who are alive 12 months after CAR-T infusion.
Measured: 12 months
Overall Response Rate (ORR)
Percentage of patients in the study who have a partial or complete response to the treatment within 3 months.
Measured: 3 months
Duration of Response (DOR)
Duration of complete remission (CR or CR with partial/incomplete haematological recovery)
Measured: Up to 24 months
Duration of CAR-T persistence
Duration of measurable CAR-T above detection limits in peripheral blood and/or marrow
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2022
- Study start18 Jul 2022
- Primary completionDec 2024 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lymphoblastic LeukemiaStudies of the same condition, open and under way.
- More trials run by KK Women's and Children's HospitalThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Sequential
- Model description
- Phase 1 run-in will be conducted to determine the recommended phase II dose(s) (RP2D) for adult and paediatric patients in independent dose escalation cohorts. Phase II will enrol patients in 2 concurrent cohorts at the R2PD.
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- KK Women's and Children's Hospital
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Michaela Seng, MD, Principal investigator, KK Women's and Children's Hospital, BMTCTWing Hang Leung, MD PhD, Study chair, KK Women's and Children's Hospital, BMTCT
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05429905
- Sponsor's protocol ID
- CART2219-1.0
- Status last verified
- Mar 2023
- First submitted
- 16 Dec 2021
- Last updated
- 9 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dual Anti-CD22/CD19 Chimeric Antigen Receptor-directed T Cells (CART2219.1) for Relapsed Refractory B-Lineage Leukaemia
- ClinicalTrials.gov ID
- NCT05429905
- Acronym / sponsor ID
- CART2219-1.0
- Conditions
- Lymphoblastic Leukemia, Lymphoblastic Leukemia, Acute, Childhood, Lymphoblastic Leukemia in Children, Lymphoblastic Leukemia, Acute Adult, CAR
- Keywords
- CAR T-cell therapy, B-ALL, Relapsed/ Refractory
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- KK Women's and Children's HospitalOther government
- Collaborators
- Singapore General Hospital, National University Hospital, Singapore
- Study start
- 18 Jul 2022 (actual)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 23 Jun 2022 (actual)
- Results
- Not posted in the source record
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 study is to deliver dual-targeting CAR-T cell therapy (CART 2219.1) as a salvage treatment to patients with relapsed/refractory B-lineage leukaemia in place of stem cell transplant or irradiation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Phase 1 run-in will be conducted to determine the recommended phase II dose(s) (RP2D) for adult and paediatric patients in independent dose escalation cohorts. Phase II will enrol patients in 2 concurrent cohorts at the R2PD.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (Dose-escalation) | Experimental | Biological: Phase IDose-finding and dose expansion cohort for intravenous autologous anti-CD22/CD19 CAR-T using a relapsed refractory B-ALL cohort. |
| Cohort 2 (High MRD) | Experimental | Biological: Phase IIPatients with B-ALL with high MRD after induction therapy or after consolidation therapy in replacement of stem cell transplant |
| Cohort 3 (Extramedullary ALL) | Experimental | Biological: Phase IIPatients with testicular or central nervous system B-ALL in replacement of radiation |
Who may qualify
- Ages
- 2 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (19 listed)
- Age 2 to 75 years
- Absolute blood CD3+ T cell count ≥100/μl
- ECOG performance score of ≤2 if >16 years old, or Lansky performance score of >50 if ≤16 years old at screening
- Patients and/or parents must give their written informed consent/assent.
- Patients have relapsed/refractory B-lineage acute lymphoblastic leukaemia, includes persistent minimal residual disease (MRD)
Key exclusion criteria (14 listed)
- Blasts are negative for both CD22 and CD19 on flow cytometry or immunohistochemistry, defined as < 10% of blasts staining positive for CD22 and CD19 respectively [IBFM 2016 Consensus Guidelines].
- Current autoimmune disease, or history of autoimmune disease with potential CNS involvement
- Active clinically significant CNS dysfunction (including but not limited to uncontrolled seizure disorders, cerebrovascular ischemia or hemorrhage, dementia, paralysis)
- History of an additional malignancy other than non-melanoma skin cancer or carcinoma in situ unless disease free for ≥3 years.
- Pulmonary function: Patients with pre-existing severe lung disease (FEV1 or FVC < 65%) or an oxygen requirement of >28% O2 supplementation or active pulmonary infiltrates on chest X-ray at the time scheduled for T cell infusion
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 |
|---|---|---|
| Phase I* Dose level 0: CAR+CD3+ 0.4 x 10e6/kg (de-escalation dose) * Dose level 1: CAR+CD3+ 1 x 10e6/kg (starting dose) * Dose level 2: CAR+CD3+ 2.5 x 10e6/kg. Dose-escalations for adults and paediatrics will be performed in 2 independent strata for determination of RP2D. | Biological | Cohort 1 (Dose-escalation) |
| Phase IIRP2D will be determined in Phase I | Biological | Cohort 2 (High MRD), Cohort 3 (Extramedullary ALL) |
Primary outcome
Recommended Phase II Dose (Phase I, Cohort 1)
Time frame: 30 days
The RP2D will be the dose level at which < 1 DLT in 3 patients or < 2 DLT in 6 patients is observed in both the adult and paediatric dose escalation stratas.
Number of patients with dose-limiting toxicities (Phase I, Cohort 1)
Time frame: 30 days
To be DLTs, adverse events must be suspected to be secondary to CAR-T cell infusion, occur during the first 30 days after infusion and meet at least one of the following criteria: 1. Grade ≥ 3 non-hematologic toxicities, with the following exceptions: * Laboratory abnormalities without associated symptomatology or clinical consequence that resolve in less than 7 days; * Grade ≤ 2 cytokine release syndrome; * Grade 3 cytokine release syndrome that improves to grade ≤ 2 within 3 days of intervention; * Grade 3 neurotoxicity that resolves to grade 2 or less within 3 days. * Laboratory abnormalities compatible with tumor lysis syndrome; 2. Grade ≥ 4 hematologic toxicities that persist at a grade ≥ 3 for >21 days. Cytokine release syndrome and immune-effector cell associated neurotoxicity syndrome (ICANS) are graded according to ASTCT consensus grading. Adverse events are graded according to CTCAE version 5.0.
12-month Leukaemia Free Survival (Phase II, Cohort 2 and 3)
Time frame: 12 months
Survival with Marrow MRD <0.01% by flow cytometry
4 secondary outcomes
Overall Survival Rate (OS)
Time frame: 12 months
Overall Response Rate (ORR)
Time frame: 3 months
Duration of Response (DOR)
Time frame: Up to 24 months
Duration of CAR-T persistence
Time frame: Up to 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2023.
- Individual participant dataSharing planned: Undecided
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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.
Michaela Seng, MD principal investigator
KK Women's and Children's Hospital, BMTCT
Wing Hang Leung, MD PhD study chair
KK Women's and Children's Hospital, BMTCT
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Phase I and Phase II in people with Lymphoblastic Leukemia and Lymphoblastic Leukemia, Acute, Childhood, run by KK Women's and Children's Hospital at 1 site in Singapore.
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 19 inclusion and 14 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: status not confirmed, as of the sponsor's last update on 9 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Singapore. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Michaela Seng, MD (+65 6394 1989, michaela.seng@singhealth.com.sg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recommended Phase II Dose (Phase I, Cohort 1) (time frame: 30 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Mar 2023.