Relapsed and/or Refractory Acute Myeloid LeukemiaPhase 1NCT05445765
Anti-CD33 CAR-T Cells for the Treatment of Relapsed/Refractory CD33+ Acute Myeloid Leukemia
A Phase 1 study testing anti-CD33 CAR T cells in people with Relapsed and/or Refractory Acute Myeloid Leukemia and High Risk Hematologic Malignancies, run by iCell Gene Therapeutics at 1 site in China.
Trial summary
This is a phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of anti-CD33 CAR-T cells in patients with relapsed and/or refractory, high risk hematologic malignancies. Read more about this study
- Who is it for?
- Ages 2 to 60 years
- Conditions the study is for
- Relapsed and/or Refractory Acute Myeloid Leukemia, High Risk Hematologic Malignancies
- What is being tested?
- anti-CD33 CAR T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2022to 30 Jun 2024
- Phase
- Phase 1
- 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 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Signed written informed consent; Patients volunteer to participate in the clinical trial;
- Diagnosis is mainly based on the World Health Organization (WHO) 2008;
- Complete remission cannot be achieved after induction therapy; recurrence occurs after completion remission; the burden of leukemic blasts in the peripheral blood or bone marrow is greater than 5%;
- Leukemic blast cells express CD33 (CD33 positive by flow cytometry or immunohistochemistry ≥70%);
- The expected survival period is greater than 12 weeks;
- ECOG score ≤2;
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- Age 2-60 years old;
- HGB≥70g/L (can be transfused);
- Total bilirubin does not exceed 3 times the upper limit of normal value, and AST and ALT do not exceed 5 times the upper limit of normal value.
Who cannot join
- Patients declining to consent for treatment
- Prior solid organ transplantation
- One of the following cardiac issues: atrial fibrillation; myocardial infarction within the past 12 months; prolonged QT syndrome or secondary QT prolongation; clinically significant pericardial effusion; cardiac insufficiency NYHA (New York Heart Association) III or IV;
- History of severe pulmonary dysfunction diseases;
- Severe infection or persistent infection cannot be effectively controlled;
- Severe autoimmune disease or congenital immunodeficiency;
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- Active hepatitis;
- Human immunodeficiency virus (HIV) infection;
- Clinically significant viral infections, or uncontrollable viral reactivation, including EBV (Epstein-Barr virus).
Trial locations and contacts
1 site in 1 country.
China1 site
Hebei Yanda Lu Daopei Hospital
Langfang, Hebei
Peihua Lu, MD, 011-86-18611636171, peihua_lu@126.com
About this study
This is a phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of anti-CD33 CAR-T cells in patients with relapsed and/or refractory, high risk hematologic malignancies.
Read the detailed description
AML is a rapidly progressing blood cancer and treated by high-dose multi-agent chemotherapy potentially followed by hematopoietic stem cell transplantation. Despite such intensive therapies, which are often associated with considerable toxicities and even death, about 60-70% of AML patients still relapse. Furthermore, the five-year survival rate from AML remains at a dismal 27%. AML is composed mostly of CD33+ leukemic blast cells. Therefore, CD33 is a potential good target by CAR T cells.
- Being tested
- anti-CD33 CAR T cells
- Keywords
- CD33, anti-CD33 CAR, leukemia, acute myeloid leukemia, hematologic malignancies
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute, Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-CD33 CAR T cellsBiological
Anti-CD33 CAR T cells are used to treat patients. Patient will be administered either fresh or thawed CAR T cells by IV injection after receiving lymphodepleting chemotherapy.
| Study group | Role | What they receive |
|---|---|---|
| anti-CD33 CAR T cells | Experimental | Dose escalation phase: anti-CD33 CAR T cells will be transduced with a lentiviral vector to express anti-CD33 CARs Receives: Biological: anti-CD33 CAR T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number and incidence of adverse events after anti-CD33 CAR infusion.
Determine the toxicity profile of anti-CD33 CAR T cell therapy including the number, incidence, and severity of symptoms such as cytokine release syndromes and neurotoxicity
Measured: 1 year, particularly the first 3 months after CAR infusion
Secondary outcomes (6)
The disease response to anti-CD33 CAR T cells
The disease response to anti-CD33 CAR T cells is evaluated by bone marrow biopsy and aspirate at 1, 2, 3, and 4 weeks. The proportion of subjects receiving anti-CD33 CAR T infusion to 1) morphological remission (blasts <5%): 2) flow cytometry analysis was blast negative, and 3) molecular biological remission (if applicable).
Measured: 4 weeks
Allogeneic hematopoietic stem cell transplantation (HCT)
Allogeneic hematopoietic stem cell transplantation (HCT) is performed after anti-CD33 CAR T treatment. The time after HCT engraftment [time range: 42 days after HCT ingraftemnt] is calculated from the day of HCT until the absolute neutrophil count (ANC) is greater than 500 / ul for three consecutive days.
Measured: 42 days after HCT ingraftment
HCT 100% chymerism time
HCT 100% chymerism time
Measured: 2 weeks after HCT
Overall survival
The time from the start of anti-CD33 CAR T injection to death is determined as the overall survival
Measured: 1 year after HCT
Progress-free survival
Progress-free survival is measured from the injection of anti-CD33 CAR T cells until the record of disease progression or death due to any reason, whichever comes first.
Measured: one year after HCT
Treatment-related mortality
Treatment-related mortality calculated from one year after HCT.
Measured: one year after HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2022
- Study start1 Jul 2022 (estimated)
- Primary completion30 Jun 2024 (estimated)
- Study completion30 Jun 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed and/or Refractory 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- iCell Gene Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- iCar Bio Therapeutics
- Responsible party
- Sponsor
- Investigators
- Peihua Lu, MD, Principal investigator, Hebei Yanda Lu Daopei Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05445765
- Sponsor's protocol ID
- ICG165-001
- Status last verified
- Jun 2022
- First submitted
- 30 Jun 2022
- Last updated
- 6 Jul 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Anti-CD33 CAR-T Cells for the Treatment of Relapsed/Refractory CD33+ Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT05445765
- Acronym / sponsor ID
- ICG165-001
- Keywords
- CD33, anti-CD33 CAR, leukemia, acute myeloid leukemia, hematologic malignancies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- iCell Gene TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- iCar Bio Therapeutics
- Study start
- 1 Jul 2022 (estimated)
- Primary completion
- 30 Jun 2024 (estimated)
- Study completion
- 30 Jun 2024 (estimated)
- First posted
- 6 Jul 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.
This is a phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of anti-CD33 CAR-T cells in patients with relapsed and/or refractory, high risk hematologic malignancies.
Detailed description
AML is a rapidly progressing blood cancer and treated by high-dose multi-agent chemotherapy potentially followed by hematopoietic stem cell transplantation. Despite such intensive therapies, which are often associated with considerable toxicities and even death, about 60-70% of AML patients still relapse. Furthermore, the five-year survival rate from AML remains at a dismal 27%. AML is composed mostly of CD33+ leukemic blast cells. Therefore, CD33 is a potential good target by CAR T cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| anti-CD33 CAR T cells | Experimental | Biological: anti-CD33 CAR T cellsDose escalation phase: anti-CD33 CAR T cells will be transduced with a lentiviral vector to express anti-CD33 CARs |
Who may qualify
- Ages
- 2 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Signed written informed consent; Patients volunteer to participate in the clinical trial;
- Diagnosis is mainly based on the World Health Organization (WHO) 2008;
- Complete remission cannot be achieved after induction therapy; recurrence occurs after completion remission; the burden of leukemic blasts in the peripheral blood or bone marrow is greater than 5%;
- Leukemic blast cells express CD33 (CD33 positive by flow cytometry or immunohistochemistry ≥70%);
- The expected survival period is greater than 12 weeks;
Key exclusion criteria (9 listed)
- Patients declining to consent for treatment
- Prior solid organ transplantation
- One of the following cardiac issues: atrial fibrillation; myocardial infarction within the past 12 months; prolonged QT syndrome or secondary QT prolongation; clinically significant pericardial effusion; cardiac insufficiency NYHA (New York Heart Association) III or IV;
- History of severe pulmonary dysfunction diseases;
- Severe infection or persistent infection cannot be effectively controlled;
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 |
|---|---|---|
| anti-CD33 CAR T cellsAnti-CD33 CAR T cells are used to treat patients. Patient will be administered either fresh or thawed CAR T cells by IV injection after receiving lymphodepleting chemotherapy. | Biological | anti-CD33 CAR T cells |
Primary outcome
The number and incidence of adverse events after anti-CD33 CAR infusion.
Time frame: 1 year, particularly the first 3 months after CAR infusion
Determine the toxicity profile of anti-CD33 CAR T cell therapy including the number, incidence, and severity of symptoms such as cytokine release syndromes and neurotoxicity
6 secondary outcomes
The disease response to anti-CD33 CAR T cells
Time frame: 4 weeks
Allogeneic hematopoietic stem cell transplantation (HCT)
Time frame: 42 days after HCT ingraftment
HCT 100% chymerism time
Time frame: 2 weeks after HCT
Overall survival
Time frame: 1 year after HCT
Progress-free survival
Time frame: one year after HCT
Treatment-related mortality
Time frame: one year after HCT
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2022.
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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.
Peihua Lu, MD principal investigator
Hebei Yanda Lu Daopei Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing anti-CD33 CAR T cells in people with Relapsed and/or Refractory Acute Myeloid Leukemia and High Risk Hematologic Malignancies, run by iCell Gene Therapeutics at 1 site in China.
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 9 inclusion and 9 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 6 Jul 2022. 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 China. 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 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.
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 Kevin Pinz, MS (6315386218, kevin.pinz@icellgene.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number and incidence of adverse events after anti-CD33 CAR infusion. (time frame: 1 year, particularly the first 3 months after CAR infusion). 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 6 Jul 2022.