Acute Myelocytic LeukemiaPhase 1NCT03796390
Study Evaluating Safety and Efficacy of CAR-T Cells Targeting CD123 in Patients With Acute Myelocytic Leukemia
A Phase 1 study testing CD123 CAR-T cells in people with Acute Myelocytic Leukemia, run by Hebei Senlang Biotechnology Inc., Ltd. at 1 site in China.
Trial summary
This is an open, single-arm, phase I clinical study to evaluate efficacy and safety of chimeric antigen receptor T cell immunotherapy (CAR-T) targeting CD123 in the treatment of Acute Myelocytic Leukemia. A total of 15 patients are planned to be enrolled following up one year. Read more about this study
- Who is it for?
- Ages 2 to 65 years
- Conditions the study is for
- Acute Myelocytic Leukemia
- What is being tested?
- CD123 CAR-T cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- 26 Dec 2018to 6 Jun 2021
- 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 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects with acute myeloid leukemia who voluntarily signed informed consent and met the following criteria:
- Diagnosed as recurrent or refractory acute myeloid leukemia
- Tumor cells confirmed CD123 positive by Flow cytometry (FCM) or immunohistochemical detection, and CD123 positive rate >80%
- Age ≥ 2 years old, and <65 years old
- Estimated survival time is longer than 3 months from the date of signing the informed consent form
- KPS ≥ 80 points
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- Important organs function need to meet the following conditions:
- 1) EF>50%, and there is no obvious abnormality in ECG; 2) SpO2≥90%; 3)Cr≤2.5ULN; 4)ALT and AST≤4ULN, TBil≤50μmol/L 7. Subjects with a pregnancy plan must agree to take contraception before the enrollment study and after the study lasts for six months; if the subject is pregnant or suspects of pregnancy, the investigator should be notified immediately 8. Need to stop chemotherapy for at least 2 weeks before collecting the blood to manufacture CAR-T cells.
- 9. For allogeneic hematopoietic stem cell transplantation subjects, it is necessary to stop the immunosuppressant against GVHD for at least 2 weeks before collecting autologous blood preparation, and if the donor is preparing blood, it is of no influence; 10. If the subject has a history of central nervous system (CNS) leukemia, the tumor cells in the cerebrospinal fluid need to be cleared and the white blood cell count <5 10^6 / L ,then can proceed lymphodepletion 11. Subjects who participate in other studies must withdraw other studies for 2 weeks before they can be enrolled.
Who cannot join
- Combine other diseases not effectively controlled, including but not limited to persistent or poorly controlled infections, symptomatic congestive heart failure, unstable angina, arrhythmia, poorly controlled lung disease or mental illness
- There are other active malignant tumors
- Combined serious infection and can not be effectively controlled
- Active hepatitis (HBV DNA or hepatitis C virus ribonucleic acid [HCVRNA] detection positive)
- Human immunodeficiency virus (HIV) infection or syphilis infection
- Have a history of severe allergies in biological products (including antibiotics)
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- One month after discontinuation of immunosuppressants, allogeneic hematopoietic stem cell transplantation patients still have acute graft versus host response (GvHD)
- Female subjects are pregnant or lactating
- Systemic administration of glucocorticoids within one week prior to CAR-T treatment
- In the past, there was a prolonged QT interval or severe heart disease.
- Active autoimmune diseases requiring systemic immunosuppressive therapy
- The investigator believes that it may increase the risk of the subject or interfere with the study results.
- Exit criteria：
- The subjects request to withdraw from the study before CAR-T infusion
- The subjects seriously violate the protocol
- Before CAR-T infusion, the following indicators are still abnormal after treatment:
- 1) EF>50%, and there is no obvious abnormality in ECG 2) SpO2≥90% 3)Cr≤2.5ULN(the upper limit of normal ) 4) ALT and AST ≤ 4ULN, TBil ≤ 50μmol / L 4.Not enough T cells for manufacture standard CAR-T cells 5. Other serious adverse events occurred
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Hebei Yanda Ludaopei Hospital
Langfang, Hebei, 065000
Peihua Lu, PhD&MD, 008618611636172, peihua_lu@126.com
Peihua Lu, PhD&MD
About this study
This is an open, single-arm, phase I clinical study to evaluate efficacy and safety of chimeric antigen receptor T cell immunotherapy (CAR-T) targeting CD123 in the treatment of Acute Myelocytic Leukemia. A total of 15 patients are planned to be enrolled following up one year.
Read the detailed description
Chimeric antigen receptor (CAR)-modified T cells targeted against CD19 have demonstrated unprecedented successes in treating patients with hematopoietic and lymphoid malignancies. Besides CD19, many other molecules such as CD22, CD30,BCMA,CD123, etc. may be potential in developing the corresponding CAR-T cells to treat patients whose tumors expressing those markers. In this study, investigators will evaluate the safety and efficacy of CAR-T targeting CD123 in patients with Acute Myelocytic Leukemia. The primary goal is safety assessment including cytokine storm response and any other adverse effects. In addition, disease status after treatment will also be evaluated.
- Conditions
- Acute Myelocytic Leukemia
- Being tested
- CD123 CAR-T cells
- Keywords
- AML，CD123
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD123 CAR-T cellsBiological
Patients will be drawn 50-100 ml blood to obtain enough peripheral blood mononuclear cells (PBMC) for CAR-T manufacturing. The T cells will be purified from the PBMC, transduced with CAR lentiviral vector, expanded in vitro and then frozen for future administration. Chemotherapy will then be given. Following tumor burden reassessment, CD123 CAR-T cells will be infused.
| Study group | Role | What they receive |
|---|---|---|
| CD123 CAR-T cells | Experimental | Patients will be be treated with CD123 CAR-T cells Receives: Biological: CD123 CAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tumor load
Tumor load will be quantified with radiology, bone marrow and/or blood samples dependent on diagnosis.
Measured: Up to 12 months
Secondary outcomes (1)
CAR-T cell persistence
CAR-T cell persistence will be quantified with flow cytometry and qPCR； Percentage of CART cells in BM and copies of car per ug DNA
Measured: Up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2019
- Study start26 Dec 2018
- Primary completion6 Nov 2020 (estimated)
- Study completion6 Jun 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myelocytic 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
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hebei Senlang Biotechnology Inc., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hebei Yanda Ludaopei Hospital
- Responsible party
- Sponsor
- Investigators
- Peihua Lu, PhD&MD, Study chair, Hebei Yanda Ludaopei Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03796390
- Sponsor's protocol ID
- Daopei CD123CAR-T
- Status last verified
- Jan 2019
- First submitted
- 4 Jan 2019
- Last updated
- 11 Jan 2019
- 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 11 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study Evaluating Safety and Efficacy of CAR-T Cells Targeting CD123 in Patients With Acute Myelocytic Leukemia
- ClinicalTrials.gov ID
- NCT03796390
- Acronym / sponsor ID
- Daopei CD123CAR-T
- Conditions
- Acute Myelocytic Leukemia
- Keywords
- AML，CD123
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Hebei Senlang Biotechnology Inc., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Hebei Yanda Ludaopei Hospital
- Study start
- 26 Dec 2018 (actual)
- Primary completion
- 6 Nov 2020 (estimated)
- Study completion
- 6 Jun 2021 (estimated)
- First posted
- 8 Jan 2019 (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 an open, single-arm, phase I clinical study to evaluate efficacy and safety of chimeric antigen receptor T cell immunotherapy (CAR-T) targeting CD123 in the treatment of Acute Myelocytic Leukemia. A total of 15 patients are planned to be enrolled following up one year.
Detailed description
Chimeric antigen receptor (CAR)-modified T cells targeted against CD19 have demonstrated unprecedented successes in treating patients with hematopoietic and lymphoid malignancies. Besides CD19, many other molecules such as CD22, CD30,BCMA,CD123, etc. may be potential in developing the corresponding CAR-T cells to treat patients whose tumors expressing those markers. In this study, investigators will evaluate the safety and efficacy of CAR-T targeting CD123 in patients with Acute Myelocytic Leukemia. The primary goal is safety assessment including cytokine storm response and any other adverse effects. In addition, disease status after treatment will also be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD123 CAR-T cells | Experimental | Biological: CD123 CAR-T cellsPatients will be be treated with CD123 CAR-T cells |
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Subjects with acute myeloid leukemia who voluntarily signed informed consent and met the following criteria:
- 1) EF>50%, and there is no obvious abnormality in ECG; 2) SpO2≥90%; 3)Cr≤2.5ULN; 4)ALT and AST≤4ULN, TBil≤50μmol/L 7. Subjects with a pregnancy plan must agree to take contraception before the enrollment study and after the study lasts for six months; if the subject is pregnant or suspects of pregnancy, the investigator should be notified immediately 8. Need to stop chemotherapy for at least 2 weeks before collecting the blood to manufacture CAR-T cells.
- 9. For allogeneic hematopoietic stem cell transplantation subjects, it is necessary to stop the immunosuppressant against GVHD for at least 2 weeks before collecting autologous blood preparation, and if the donor is preparing blood, it is of no influence; 10. If the subject has a history of central nervous system (CNS) leukemia, the tumor cells in the cerebrospinal fluid need to be cleared and the white blood cell count <5 10^6 / L ,then can proceed lymphodepletion 11. Subjects who participate in other studies must withdraw other studies for 2 weeks before they can be enrolled.
Key exclusion criteria (17 listed)
- Combine other diseases not effectively controlled, including but not limited to persistent or poorly controlled infections, symptomatic congestive heart failure, unstable angina, arrhythmia, poorly controlled lung disease or mental illness
- There are other active malignant tumors
- Combined serious infection and can not be effectively controlled
- Active hepatitis (HBV DNA or hepatitis C virus ribonucleic acid [HCVRNA] detection positive)
- Human immunodeficiency virus (HIV) infection or syphilis infection
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 |
|---|---|---|
| CD123 CAR-T cellsPatients will be drawn 50-100 ml blood to obtain enough peripheral blood mononuclear cells (PBMC) for CAR-T manufacturing. The T cells will be purified from the PBMC, transduced with CAR lentiviral vector, expanded in vitro and then frozen for future administration. Chemotherapy will then be given. Following tumor burden reassessment, CD123 CAR-T cells will be infused. | Biological | CD123 CAR-T cells |
Primary outcome
Tumor load
Time frame: Up to 12 months
Tumor load will be quantified with radiology, bone marrow and/or blood samples dependent on diagnosis.
1 secondary outcome
CAR-T cell persistence
Time frame: Up to 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jan 2019.
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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, PhD&MD study chair
Hebei Yanda Ludaopei 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 CD123 CAR-T cells in people with Acute Myelocytic Leukemia, run by Hebei Senlang Biotechnology Inc., Ltd. 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 17 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 11 Jan 2019. 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 Peihua Lu, PhD&MD (008618611636172, peihua_lu@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tumor load (time frame: Up to 12 months). 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 11 Jan 2019.