Platelet Transfusion RefractorinessPhase 1/2NCT04846439
Sequential Infusion of CD19 and BCMA CAR-T Cells to Improve PTR in Patients With AL
A Phase 1/2 study testing CAR-T infusion in people with Platelet Transfusion Refractoriness and Acute Leukemia in Remission, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
Alloimmune-mediated platelet transfusion refractoriness(PTR) was usually caused by repeated blood transfusions and pregnancy and accounts for about 20-25% of PTR patients. Patients with acute leukemia need repeated platelet infusion in myelosuppression period after chemotherapy, and PTR incidence is more higher.PTR… Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Platelet Transfusion Refractoriness, Acute Leukemia in Remission
- What is being tested?
- CAR-T infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 29 Apr 2021to 31 Mar 2024
- 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 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Ages 16-65 years inclusive.
- Ability to comprehend the investigational nature of the study and provide informed consent.
- Expected survival time ≥ 3 months (according to investigator's judgement)
- Acute leukemia in complete remission diagnosed with alloimmune-mediated PTR, characterized by all of the following:
- Lack of adequate post-transfusion platelet count increment, defined by CCI <7500/μl at 10-60 min, and CCI <5000/μl at 18-24 hrs (in those who had a CCI at 10-60 min greater than or equal to 5000/μl) after at least 2 consecutive transfusions.
- Presence of anti-HLA class A and/or B antibody.
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- Left ventricular ejection fractions ≥ 0.5 by echocardiography.
- Creatinine < 1.6 mg/dL.
- Aspartate aminotransferase/aspartate aminotransferase < 3x upper limit of normal.
- Total bilirubin <2.0 mg/dL.
- karnofsky performance status ≥ 60.
Who cannot join
- PTR induced by other reasons(eg:DIC,fever,infection and splenomegaly)
- Uncontrolled active infection.
- Active hepatitis B or hepatitis C infection
- Patients with HIV or syphilis infection
- Patients are pregnant or lactating
- Patients has a history of allo-HSCT
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- Alloimmune-mediated PTR responsive to treatment with plasma exchange
- Alloimmune-mediated PTR responsive to treatment with rituximab or IVIG
- Grade III/IV cardiovascular disability according to the New York Heart Association Classification
- Patients with other contraindications considered unsuitable for participation in this study (according to investigator's judgement)
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
xiaowen tang, Ph.D, (0086)51267781856, xwtang1020@163.com
About this study
Official title: Sequential Infusion of CD19 and BCMA Chimeric Antigen Receptor T Cells to Improve Alloimmune-mediated Platelet Transfusion Refractoriness in Patients With Acute Leukemia in Complete Remission
Alloimmune-mediated platelet transfusion refractoriness(PTR) was usually caused by repeated blood transfusions and pregnancy and accounts for about 20-25% of PTR patients. Patients with acute leukemia need repeated platelet infusion in myelosuppression period after chemotherapy, and PTR incidence is more higher.PTR was associated with adverse events,including longer hospital stays,severe hemorrhages and an increased risk of early deaths and may have a negative impact on the success of HSCT. The current management of patients with PTR includes specific transfusion strategies, IVIG, rituximab,thrombopoietin-receptor agonists(TPO-RA) ,bortezomib or splenectomy,have been largely unsatisfactory. As we know, HLA antibodies are mainly secreted by the plasma cells. Researchers want to see if sequential infusion of CD19 and BCMA CAR-T cells can clear the B cells and plasma cells, can help increase platelet levels and reduce bleeding in patients with platelet transfusion refractoriness. To see if sequential infusion can increases platelet levels more after a transfusion. To see if it reduces the chance of bleeding. Adults 16-65 years old who diagnosed with acute leukemia in CR and alloimmune platelet transfusion refractoriness.
Read the detailed description
The patients will receive infusion of CAR T-cells targeting CD19 and BCMA to confirm the safety and efficacy of CD19 and BCMA CAR T-Cells Sequential infusion in acute leukemia with alloimmune-mediated platelet transfusion refractoriness.
- Being tested
- CAR-T infusion
- Keywords
- CAR-T, alloimmune, platelet transfusion refractoriness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAR-T infusionBiological
Sequential infusion of CD19 and BCMA autologous chimeric antigen receptor T cells, the infusion dose was determined according to the body weight of the subject and the effective content of cell preparation.
| Study group | Role | What they receive |
|---|---|---|
| CAR-T infusion | Experimental | CD19 and BCMA CAR-T cells were infused into complete remission acute leukemia patients with PTR sequentially, with（1.0-2.0）×10e7/kg respectively. Each patient was followed up for 1 years. Receives: Biological: CAR-T infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects With Sustained Platelet Transfusion Responsiveness
To evaluate the safety and efficacy of sequential infusion of CD19 and BCMA CAR-T cells to improve PTR, estimate by platelet increment, defined as Corrected Count Increment (CCI) >7500/μL at 10-60 min together with CCI>5000/μL at 18-24 hrs post platelet transfusion in patients with platelet transfusion refractoriness.
Measured: 12 months
Adverse events after sequential infusion of CAR-T
Adverse events are evaluated with CTCAE V5.0.
Measured: 12 months
Secondary outcomes (3)
B lymphocytes/plasma cell clearance
To investigate the possible mechanisms of sequential infusion in alloimmune-mediated PTR
Measured: 12 months
Amplification,distribution and persistence of CAR T-cells in vivo
To evaluate the persistence of CAR-T cells in vivo
Measured: 12 months
Alloimmune antibodies(include HLA and HPA) in PB after sequential transfusion
To evaluate the clearance of alloimmune antibodies.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2021
- Study start29 Apr 2021
- Primary completion31 Mar 2023 (estimated)
- Study completion31 Mar 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Platelet Transfusion RefractorinessStudies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow UniversityThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- The Second People's Hospital of Huai'an, The First Affiliated Hospital with Nanjing Medical University, The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School, Shanghai Unicar-Therapy Bio-medicine Technology Co.,Ltd
- Responsible party
- Sponsor
- Investigators
- Haiping Dai, Ph.D, Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04846439
- Sponsor's protocol ID
- 2021062
- Status last verified
- Apr 2021
- First submitted
- 13 Apr 2021
- Last updated
- 27 Oct 2021
- 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 27 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sequential Infusion of CD19 and BCMA Chimeric Antigen Receptor T Cells to Improve Alloimmune-mediated Platelet Transfusion Refractoriness in Patients With Acute Leukemia in Complete Remission
- ClinicalTrials.gov ID
- NCT04846439
- Acronym / sponsor ID
- 2021062
- Keywords
- CAR-T, alloimmune, platelet transfusion refractoriness
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Collaborators
- The Second People's Hospital of Huai'an, The First Affiliated Hospital with Nanjing Medical University, The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School, Shanghai Unicar-Therapy Bio-medicine Technology Co.,Ltd
- Study start
- 29 Apr 2021 (actual)
- Primary completion
- 31 Mar 2023 (estimated)
- Study completion
- 31 Mar 2024 (estimated)
- First posted
- 15 Apr 2021 (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.
Alloimmune-mediated platelet transfusion refractoriness(PTR) was usually caused by repeated blood transfusions and pregnancy and accounts for about 20-25% of PTR patients. Patients with acute leukemia need repeated platelet infusion in myelosuppression period after chemotherapy, and PTR incidence is more higher.PTR was associated with adverse events,including longer hospital stays,severe hemorrhages and an increased risk of early deaths and may have a negative impact on the success of HSCT. The current management of patients with PTR includes specific transfusion strategies, IVIG, rituximab,thrombopoietin-receptor agonists(TPO-RA) ,bortezomib or splenectomy,have been largely unsatisfactory. As we know, HLA antibodies are mainly secreted by the plasma cells. Researchers want to see if sequential infusion of CD19 and BCMA CAR-T cells can clear the B cells and plasma cells, can help increase platelet levels and reduce bleeding in patients with platelet transfusion refractoriness. To see if sequential infusion can increases platelet levels more after a transfusion. To see if it reduces the chance of bleeding. Adults 16-65 years old who diagnosed with acute leukemia in CR and alloimmune platelet transfusion refractoriness.
Detailed description
The patients will receive infusion of CAR T-cells targeting CD19 and BCMA to confirm the safety and efficacy of CD19 and BCMA CAR T-Cells Sequential infusion in acute leukemia with alloimmune-mediated platelet transfusion refractoriness.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CAR-T infusion | Experimental | Biological: CAR-T infusionCD19 and BCMA CAR-T cells were infused into complete remission acute leukemia patients with PTR sequentially, with（1.0-2.0）×10e7/kg respectively. Each patient was followed up for 1 years. |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Ages 16-65 years inclusive.
- Ability to comprehend the investigational nature of the study and provide informed consent.
- Expected survival time ≥ 3 months (according to investigator's judgement)
- Acute leukemia in complete remission diagnosed with alloimmune-mediated PTR, characterized by all of the following:
- Lack of adequate post-transfusion platelet count increment, defined by CCI <7500/μl at 10-60 min, and CCI <5000/μl at 18-24 hrs (in those who had a CCI at 10-60 min greater than or equal to 5000/μl) after at least 2 consecutive transfusions.
Key exclusion criteria (10 listed)
- PTR induced by other reasons(eg:DIC,fever,infection and splenomegaly)
- Uncontrolled active infection.
- Active hepatitis B or hepatitis C infection
- Patients with HIV or syphilis infection
- Patients are pregnant or lactating
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 |
|---|---|---|
| CAR-T infusionSequential infusion of CD19 and BCMA autologous chimeric antigen receptor T cells, the infusion dose was determined according to the body weight of the subject and the effective content of cell preparation. | Biological | CAR-T infusion |
Primary outcome
Number of Subjects With Sustained Platelet Transfusion Responsiveness
Time frame: 12 months
To evaluate the safety and efficacy of sequential infusion of CD19 and BCMA CAR-T cells to improve PTR, estimate by platelet increment, defined as Corrected Count Increment (CCI) >7500/μL at 10-60 min together with CCI>5000/μL at 18-24 hrs post platelet transfusion in patients with platelet transfusion refractoriness.
Adverse events after sequential infusion of CAR-T
Time frame: 12 months
Adverse events are evaluated with CTCAE V5.0.
3 secondary outcomes
B lymphocytes/plasma cell clearance
Time frame: 12 months
Amplification,distribution and persistence of CAR T-cells in vivo
Time frame: 12 months
Alloimmune antibodies(include HLA and HPA) in PB after sequential transfusion
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2021.
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Acalabrutinib Maleate and Bortezomib for Patients With HLA Antibodies
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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.
Haiping Dai, Ph.D principal investigator
The First Affiliated Hospital of Soochow University
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 CAR-T infusion in people with Platelet Transfusion Refractoriness and Acute Leukemia in Remission, run by The First Affiliated Hospital of Soochow University 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 11 inclusion and 10 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 27 Oct 2021. 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 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 Xiaowen Tang, Ph.D ((0086)51267781856, xwtang1020@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Subjects With Sustained Platelet Transfusion Responsiveness (time frame: 12 months). The study lists 2 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 27 Oct 2021.