POEMS SyndromeEarly Phase 1NCT05263817
A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
A Early Phase 1 study testing CD19/BCMA CAR T-cells in people with POEMS Syndrome and Amyloidosis, run by Zhejiang University at 1 site in China.
Trial summary
A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia
- What is being tested?
- CD19/BCMA CAR T-cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 1 siteChina
- Study dates
- 8 Oct 2021to 1 Oct 2024
- Phase
- Early 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1. Diagnosed with POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis, and the curative effect of conventional hormones, radiotherapy and chemotherapy, protease inhibitors is not good and (or) no effective treatment means.
- 2. After glucocorticoids, cyclophosphamide or methotrexate treatments there are still relapsed and refractory diseases, or clearly show intolerance/toxicity to these drugs.
- 3. Estimated survival time> 12 weeks; 4. Patients had a negative urine pregnancy test before the start of administration and agreed to take effective contraceptive measures during the test period until the last follow-up; 5. Patients or their legal guardians volunteer to participate in the study and sign the informed consent.
Who cannot join
- Subjects with any of the following exclusion criteria were not eligible for this trial:
- History of craniocerebral trauma, conscious disturbance, epilepsy,cerebrovascular ischemia, and cerebrovascular, hemorrhagic diseases;
- Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
- Pregnant (or lactating) women;
- Patients with severe active infections (excluding simple urinary tract infection and bacterial pharyngitis);
- Active infection of hepatitis B virus or hepatitis C virus;
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- Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
- Those who have used any gene therapy products before.
- The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
- Serum creatinine > 2.5mg/dl or ALT / AST > 3 times ULN or bilirubin > 2.0mg/dl;
- Those who suffer from other uncontrolled diseases are not suitable to join the study;
- HIV infection;
- Any situation that the researchers believe may increase the risk of patients or interfere with the test results.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The first affiliated hospital of medical college of zhejiang university
Hangzhou, Zhejiang, 310003
He Huang, PhD, 86-13605714822, hehuangyu@126.com
Yongxian Hu, PhD, +8615957162012, huyongxian2000@aliyun.com
About this study
Official title: A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
Read the detailed description
POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis and other diseases may only show local pathological damage or systemic lesions. If they are not diagnosed and treated in time or poorly controlled, they will progress as the course of the disease progresses. Risk of disability or even death.
- Conditions
- POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis
- Being tested
- CD19/BCMA CAR T-cells
- Keywords
- CD19 CAR-T, BCMA CAR-T, POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis
- Medical subject headings
- POEMS Syndrome, Amyloidosis, Anemia, Hemolytic, Autoimmune, Vasculitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD19/BCMA CAR T-cellsBiological
Also called CD19/BCMA CAR T-cells injection
Each subject receive CD19/BCMA CAR T-cells by intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| POEMS Syndrome | Experimental | Not described Receives: Biological: CD19/BCMA CAR T-cells |
| Amyloidosis | Experimental | Not described Receives: Biological: CD19/BCMA CAR T-cells |
| Autoimmune Hemolytic Anemia | Experimental | Not described Receives: Biological: CD19/BCMA CAR T-cells |
| Vasculitis | Experimental | Not described Receives: Biological: CD19/BCMA CAR T-cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose-limiting toxicity (DLT)
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Measured: Baseline up to 28 days after CD19/BCMA targeted CAR T-cells infusion
Incidence of treatment-emergent adverse events (TEAEs)
Incidence of treatment-emergent adverse events [Safety and Tolerability]
Measured: Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Secondary outcomes (5)
Titer of auto-antibody Titer of auto-antibody titer of auto-antibody
In peripheral blood and bone marrow
Measured: Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Overall response rate (ORR)
Proportion of subjects with complete or partial remission
Measured: Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Best overall response, BOR
Assessment of ORR at ≤3 month
Measured: At ≤3 month
Overall survival (OS)
The time from the cell reinfusion to death due to any cause
Measured: From CD19/BCMA CAR-T infusion to death，up to 2 years
Duration of remission, DOR
The time from the first assessment of remission or partial remission of the disease to the first assessment of disease progression or death from any cause
Measured: 2 years post CD19/BCMA CAR-T cells infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2022
- Study start8 Oct 2021
- Primary completion1 Oct 2024 (estimated)
- Study completion1 Oct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for POEMS SyndromeStudies of the same condition, open and under way.
- More trials run by Zhejiang 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Responsible party
- Principal investigator, He Huang, Zhejiang University
- Investigators
- He Huang, PhD, Principal investigator, First Affiliated Hospital of Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05263817
- Sponsor's protocol ID
- CD19/BCMA-005
- Status last verified
- Feb 2022
- First submitted
- 1 Dec 2021
- Last updated
- 3 Mar 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
- ClinicalTrials.gov ID
- NCT05263817
- Acronym / sponsor ID
- CD19/BCMA-005
- Conditions
- POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis
- Keywords
- CD19 CAR-T, BCMA CAR-T, POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Study start
- 8 Oct 2021 (actual)
- Primary completion
- 1 Oct 2024 (estimated)
- Study completion
- 1 Oct 2024 (estimated)
- First posted
- 3 Mar 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.
A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
Detailed description
POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis and other diseases may only show local pathological damage or systemic lesions. If they are not diagnosed and treated in time or poorly controlled, they will progress as the course of the disease progresses. Risk of disability or even death.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| POEMS Syndrome | Experimental | Biological: CD19/BCMA CAR T-cells |
| Amyloidosis | Experimental | Biological: CD19/BCMA CAR T-cells |
| Autoimmune Hemolytic Anemia | Experimental | Biological: CD19/BCMA CAR T-cells |
| Vasculitis | Experimental | Biological: CD19/BCMA CAR T-cells |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 1. Diagnosed with POEMS syndrome, amyloidosis, autoimmune hemolytic anemia, vasculitis, and the curative effect of conventional hormones, radiotherapy and chemotherapy, protease inhibitors is not good and (or) no effective treatment means.
- 2. After glucocorticoids, cyclophosphamide or methotrexate treatments there are still relapsed and refractory diseases, or clearly show intolerance/toxicity to these drugs.
- 3. Estimated survival time> 12 weeks; 4. Patients had a negative urine pregnancy test before the start of administration and agreed to take effective contraceptive measures during the test period until the last follow-up; 5. Patients or their legal guardians volunteer to participate in the study and sign the informed consent.
Key exclusion criteria (13 listed)
- Subjects with any of the following exclusion criteria were not eligible for this trial:
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 |
|---|---|---|
| CD19/BCMA CAR T-cellsAlso: CD19/BCMA CAR T-cells injectionEach subject receive CD19/BCMA CAR T-cells by intravenous infusion | Biological | Amyloidosis, Autoimmune Hemolytic Anemia, POEMS Syndrome, Vasculitis |
Primary outcome
Dose-limiting toxicity (DLT)
Time frame: Baseline up to 28 days after CD19/BCMA targeted CAR T-cells infusion
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Incidence of treatment-emergent adverse events [Safety and Tolerability]
5 secondary outcomes
Titer of auto-antibody Titer of auto-antibody titer of auto-antibody
Time frame: Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Overall response rate (ORR)
Time frame: Up to 2 years after CD19/BCMA targeted CAR T-cells infusion
Best overall response, BOR
Time frame: At ≤3 month
Overall survival (OS)
Time frame: From CD19/BCMA CAR-T infusion to death，up to 2 years
Duration of remission, DOR
Time frame: 2 years post CD19/BCMA CAR-T cells infusion
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 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.
He Huang, PhD principal investigator
First Affiliated Hospital of Zhejiang University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CD19/BCMA CAR T-cells in people with POEMS Syndrome and Amyloidosis, run by Zhejiang 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 3 inclusion and 13 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 3 Mar 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 He Huang, PhD (+8613605714822, hehuangyu@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose-limiting toxicity (DLT) (time frame: Baseline up to 28 days after CD19/BCMA targeted CAR T-cells infusion). 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 3 Mar 2022.