Multiple Myeloma, Newly DiagnosedPhase 2ASCTvsCAR-TNCT06963866
Comparing ASCT Followed by Anti-BCMA CAR-T vs. ASCT Alone in NDMM Patients Eligible for ASCT
A Phase 2 study testing autologous stem cell transplantation and CAR-T in people with Multiple Myeloma, Newly Diagnosed, run by Xuzhou Medical University at 1 site in China.
Trial summary
This is a prospective study comparing autologous hematopoietic stem cell transplantation followed by anti-BCMA CAR-T to autologous hematopoietic stem cell transplantation alone in the treatment of newly diagnosed multiple myeloma patients. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Multiple Myeloma, Newly Diagnosed
- What is being tested?
- autologous stem cell transplantation and CAR-T
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- 1 May 2025to 30 Apr 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age between 18 and 70 years (inclusive);
- Estimated life expectancy of more than 12 weeks;
- Diagnosis of multiple myeloma confirmed by physical examination, pathological evaluation, laboratory tests, and imaging studies;
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels less than 3 times the upper limit of normal (ULN);
- Karnofsky Performance Status (KPS) score > 50%.
- Eligible for ASCT.
Who cannot join
- Pregnant or lactating women, or women planning to become pregnant within the next six months;
- Transduction efficiency of targeted lymphocytes <10%, or expansion fold <5× under CD3/CD28 co-stimulation, as determined by feasibility screening;
- History of severe allergies or hypersensitivity, especially to interleukin-2 (IL-2);
- Significant dysfunction of vital organs including the heart, lungs, or brain;
- Any other condition that, in the investigator's judgment, makes the patient unsuitable for participation in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The Affiliated Hospital oh Xuzhou Medical University
Xuzhou, Jiangsu, 221006
Kailin Xu, +8615162166166, lihmd@163.com
Xu
About this study
Official title: A Multicenter, Open-Label, Randomized, Controlled Study of Autologous Stem Cell Transplantation Followed by Anti-BCMA CAR-T Therapy Versus ASCT Alone in Transplant-Eligible Patients With Newly Diagnosed Multiple Myeloma
This is a prospective study comparing autologous hematopoietic stem cell transplantation followed by anti-BCMA CAR-T to autologous hematopoietic stem cell transplantation alone in the treatment of newly diagnosed multiple myeloma patients.
Read the detailed description
- Pre-screening Evaluation. The patient's medical history will be reviewed to assess eligibility based on inclusion criteria. The treatment process and potential risks will be explained, and informed consent will be obtained. Peripheral blood samples will then be collected to test for HIV and to assess the feasibility of CAR-T cell manufacturing. The CAR-T production unit will evaluate the in vitro expansion capacity and transduction efficiency of the patient's T cells within approximately 1-2 weeks to determine whether the patient's peripheral blood is suitable for large-scale CAR-T cell production.
- Patient Enrollment and Baseline Data Collection. 2.1 Patients who pass the pre-screening will undergo disease assessment and a series of routine evaluations to confirm eligibility for CAR-T cell therapy or autologous hematopoietic stem cell transplantation (ASCT) based on inclusion and exclusion criteria. 2.2 Required Baseline Tests (within 4 weeks prior to treatment): Complete medical history and physical examination; Electrocardiogram and cardiac function evaluation (MUGA scan or echocardiography); Pulse oximetry to assess lung function; Serum electrolytes and biochemistry; Peripheral blood flow cytometry for quantification of T cells and CD4:CD8 ratio HIV, hepatitis B (HBsAb, HBsAg, HBeAb, HBeAg, HBcAb), and hepatitis C (HCV Ab) screening; Human anti-mouse antibody (HAMA) testing, along with collection of blood and bone marrow samples for DNA extraction and biobanking for future analysis related to tumorigenesis.
- Autologous Stem Cell Collection and CAR-T Cell Manufacturing. 3.1 Autologous Hematopoietic Stem Cell Collection and Cryopreservation Granulocyte-colony stimulating factor (G-CSF) at 300 μg twice daily for 4 days will be administered. Once peripheral blood CD34+ cells reach the collection threshold, apheresis will be performed with a total blood volume of approximately 6 liters to collect autologous hematopoietic stem cells. The minimum cell dose should be ≥2.0 × 10^6CD34+ cells/kg. DMSO will be added to the collection bag to a final concentration of 20%. The stem cell product will be stored in cryogenic bags, placed in freezing containers, and stored at -80°C. 3.2 Anti-BCMA CAR-T Cell Manufacturing Apheresis will also be conducted to collect peripheral blood mononuclear cells (PBMCs) for CAR-T cell manufacturing, using approximately 6 liters of blood. Lymphocytes not used for production will be cryopreserved for future research or regulatory review.
- Conditioning Chemotherapy. High-dose melphalan will be used as conditioning therapy beginning 5 days prior to ASCT. The dose ranges from 140-200 mg/m². The purposes of chemotherapy are: To reduce tumor burden; To deplete endogenous lymphocytes and facilitate CAR-T cell expansion; To modulate the tumor immune microenvironment. If absolute neutrophil count (ANC) drops below 1.0 × 10^9/L post-chemotherapy, granulocyte colony-stimulating factor (G-CSF) will be administered until ANC exceeds 1.5 × 10^9/L. Antibiotics may be used prophylactically if neutropenia occurs.
- Post-conditioning Evaluation. Tumor status will be reassessed after conditioning to evaluate baseline disease burden. This includes physical examination, laboratory tests, bone marrow MRD (minimal residual disease) evaluation, and assessment of chemotherapy-related toxicity.
- Autologous Stem Cell Infusion. Autologous stem cells (minimum 2.0 × 10^6 CD34+ cells/kg) will be reinfused 2 days after conditioning ends. The cryopreserved product will be thawed at 37°C in a water bath and infused intravenously immediately upon full thaw. Vital signs will be closely monitored before, during, and after infusion at 15-minute intervals until the patient stabilizes.
- Anti-BCMA CAR-T Cell Infusion. CAR-T cells will be infused approximately 3 days after autologous stem cell infusion. The thawed CAR-T product should be infused within 10-15 minutes, with a minimum dose of 2.0 × 10^6 cells/kg. Vital signs will be closely monitored before, during, and after infusion at 15-minute intervals until patient stabilization. Acetaminophen 325-650 mg will be administered orally 30-60 minutes before infusion to reduce infusion-related reactions.
- Conditions
- Multiple Myeloma, Newly Diagnosed
- Being tested
- autologous stem cell transplantation, CAR-T
- Keywords
- Multiple Myeloma, Eligible for ASCT, ASCT, CAR-T
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
autologous stem cell transplantationBiological
Patients in this arm will receive autologous hematopoietic stem cell transplantation (ASCT). Prior to ASCT, the patients underwent 3-4 cycles of induction chemotherapy.
CAR-TBiological
The T cells are genetically modified to express a chimeric antigen receptor targeting BCMA and are infused 3 days after ASCT at a target dose of ≥2.0×10^6 cells/kg.
| Study group | Role | What they receive |
|---|---|---|
| ASCT + CAR-T | Experimental | ASCT followed by BCMA CAR-T Receives: Biological: autologous stem cell transplantation; Biological: CAR-T |
| ASCT alone | Active comparator | ASCT alone, without CAR-T infusion Receives: Biological: autologous stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression-Free Survival (PFS)
Progression-Free Survival (PFS) is defined as the time from the date of CAR-T cell infusion to the date of disease progression or death from any cause, whichever occurs first. Disease progression will be determined based on the International Myeloma Working Group (IMWG) criteria. Patients who have not progressed or died will be censored at the date of last follow-up.
Measured: Up to 36 months after CAR-T infusion
Overall Survival (OS)
Overall Survival (OS) is defined as the time from the date of CAR-T cell infusion to death from any cause. Patients who are alive at the time of analysis will be censored at their last known date of follow-up.
Measured: Up to 36 months after CAR-T infusion
Secondary outcomes (3)
Objective Response Rate (ORR)
Objective Response Rate (ORR) is defined as the proportion of patients achieving a response of partial response (PR) or better, including PR, very good partial response (VGPR), complete response (CR), and stringent complete response (sCR), according to the International Myeloma Working Group (IMWG) criteria. The assessment will be based on laboratory parameters, imaging, and bone marrow evaluation, as applicable.
Measured: Up to 36 months after CAR-T infusion
MRD
The proportion and immunophenotype of plasma cells in bone marrow were analyzed using flow cytometry, with a sensitivity of 10^-5.
Measured: Up to 36 months after CAR-T infusion
Adverse Events (AE)
Focus on adverse events related to ASCT and CAR-T, including CRS, ICANS, coagulation disorders, infections, and other complications.
Measured: Up to 36 months after CAR-T infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2025
- Study start1 May 2025 (estimated)
- Primary completion30 Apr 2028 (estimated)
- Study completion30 Apr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple Myeloma, Newly DiagnosedStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xuzhou Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kai Lin Xu，MD, Xuzhou Medical University
- Investigators
- Kailin Xu, Study chair, The Affiliated Hospital oh Xuzhou Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06963866
- Sponsor's protocol ID
- XYFY2025-KL064-01
- Other IDs
- XYFY2025-KL064-01
- Acronym
- ASCTvsCAR-T
- Status last verified
- Apr 2025
- First submitted
- 30 Apr 2025
- Last updated
- 9 May 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label, Randomized, Controlled Study of Autologous Stem Cell Transplantation Followed by Anti-BCMA CAR-T Therapy Versus ASCT Alone in Transplant-Eligible Patients With Newly Diagnosed Multiple Myeloma
- ClinicalTrials.gov ID
- NCT06963866
- Acronym / sponsor ID
- ASCTvsCAR-T / XYFY2025-KL064-01
- Conditions
- Multiple Myeloma, Newly Diagnosed
- Keywords
- Multiple Myeloma, Eligible for ASCT, ASCT, CAR-T
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Xuzhou Medical UniversityUniversity, hospital or other
- Study start
- 1 May 2025 (estimated)
- Primary completion
- 30 Apr 2028 (estimated)
- Study completion
- 30 Apr 2028 (estimated)
- First posted
- 9 May 2025 (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 prospective study comparing autologous hematopoietic stem cell transplantation followed by anti-BCMA CAR-T to autologous hematopoietic stem cell transplantation alone in the treatment of newly diagnosed multiple myeloma patients.
Detailed description
- Pre-screening Evaluation. The patient's medical history will be reviewed to assess eligibility based on inclusion criteria. The treatment process and potential risks will be explained, and informed consent will be obtained. Peripheral blood samples will then be collected to test for HIV and to assess the feasibility of CAR-T cell manufacturing. The CAR-T production unit will evaluate the in vitro expansion capacity and transduction efficiency of the patient's T cells within approximately 1-2 weeks to determine whether the patient's peripheral blood is suitable for large-scale CAR-T cell production.
- Patient Enrollment and Baseline Data Collection. 2.1 Patients who pass the pre-screening will undergo disease assessment and a series of routine evaluations to confirm eligibility for CAR-T cell therapy or autologous hematopoietic stem cell transplantation (ASCT) based on inclusion and exclusion criteria. 2.2 Required Baseline Tests (within 4 weeks prior to treatment): Complete medical history and physical examination; Electrocardiogram and cardiac function evaluation (MUGA scan or echocardiography); Pulse oximetry to assess lung function; Serum electrolytes and biochemistry; Peripheral blood flow cytometry for quantification of T cells and CD4:CD8 ratio HIV, hepatitis B (HBsAb, HBsAg, HBeAb, HBeAg, HBcAb), and hepatitis C (HCV Ab) screening; Human anti-mouse antibody (HAMA) testing, along with collection of blood and bone marrow samples for DNA extraction and biobanking for future analysis related to tumorigenesis.
- Autologous Stem Cell Collection and CAR-T Cell Manufacturing. 3.1 Autologous Hematopoietic Stem Cell Collection and Cryopreservation Granulocyte-colony stimulating factor (G-CSF) at 300 μg twice daily for 4 days will be administered. Once peripheral blood CD34+ cells reach the collection threshold, apheresis will be performed with a total blood volume of approximately 6 liters to collect autologous hematopoietic stem cells. The minimum cell dose should be ≥2.0 × 10^6CD34+ cells/kg. DMSO will be added to the collection bag to a final concentration of 20%. The stem cell product will be stored in cryogenic bags, placed in freezing containers, and stored at -80°C. 3.2 Anti-BCMA CAR-T Cell Manufacturing Apheresis will also be conducted to collect peripheral blood mononuclear cells (PBMCs) for CAR-T cell manufacturing, using approximately 6 liters of blood. Lymphocytes not used for production will be cryopreserved for future research or regulatory review.
- Conditioning Chemotherapy. High-dose melphalan will be used as conditioning therapy beginning 5 days prior to ASCT. The dose ranges from 140-200 mg/m². The purposes of chemotherapy are: To reduce tumor burden; To deplete endogenous lymphocytes and facilitate CAR-T cell expansion; To modulate the tumor immune microenvironment. If absolute neutrophil count (ANC) drops below 1.0 × 10^9/L post-chemotherapy, granulocyte colony-stimulating factor (G-CSF) will be administered until ANC exceeds 1.5 × 10^9/L. Antibiotics may be used prophylactically if neutropenia occurs.
- Post-conditioning Evaluation. Tumor status will be reassessed after conditioning to evaluate baseline disease burden. This includes physical examination, laboratory tests, bone marrow MRD (minimal residual disease) evaluation, and assessment of chemotherapy-related toxicity.
- Autologous Stem Cell Infusion. Autologous stem cells (minimum 2.0 × 10^6 CD34+ cells/kg) will be reinfused 2 days after conditioning ends. The cryopreserved product will be thawed at 37°C in a water bath and infused intravenously immediately upon full thaw. Vital signs will be closely monitored before, during, and after infusion at 15-minute intervals until the patient stabilizes.
- Anti-BCMA CAR-T Cell Infusion. CAR-T cells will be infused approximately 3 days after autologous stem cell infusion. The thawed CAR-T product should be infused within 10-15 minutes, with a minimum dose of 2.0 × 10^6 cells/kg. Vital signs will be closely monitored before, during, and after infusion at 15-minute intervals until patient stabilization. Acetaminophen 325-650 mg will be administered orally 30-60 minutes before infusion to reduce infusion-related reactions.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ASCT + CAR-T | Experimental | Biological: autologous stem cell transplantation, Biological: CAR-TASCT followed by BCMA CAR-T |
| ASCT alone | Active comparator | Biological: autologous stem cell transplantationASCT alone, without CAR-T infusion |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age between 18 and 70 years (inclusive);
- Estimated life expectancy of more than 12 weeks;
- Diagnosis of multiple myeloma confirmed by physical examination, pathological evaluation, laboratory tests, and imaging studies;
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels less than 3 times the upper limit of normal (ULN);
- Karnofsky Performance Status (KPS) score > 50%.
Key exclusion criteria (5 listed)
- Pregnant or lactating women, or women planning to become pregnant within the next six months;
- Transduction efficiency of targeted lymphocytes <10%, or expansion fold <5× under CD3/CD28 co-stimulation, as determined by feasibility screening;
- History of severe allergies or hypersensitivity, especially to interleukin-2 (IL-2);
- Significant dysfunction of vital organs including the heart, lungs, or brain;
- Any other condition that, in the investigator's judgment, makes the patient unsuitable for participation in this study.
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 |
|---|---|---|
| autologous stem cell transplantationPatients in this arm will receive autologous hematopoietic stem cell transplantation (ASCT). Prior to ASCT, the patients underwent 3-4 cycles of induction chemotherapy. | Biological | ASCT + CAR-T, ASCT alone |
| CAR-TThe T cells are genetically modified to express a chimeric antigen receptor targeting BCMA and are infused 3 days after ASCT at a target dose of ≥2.0×10^6 cells/kg. | Biological | ASCT + CAR-T |
Primary outcome
Progression-Free Survival (PFS)
Time frame: Up to 36 months after CAR-T infusion
Progression-Free Survival (PFS) is defined as the time from the date of CAR-T cell infusion to the date of disease progression or death from any cause, whichever occurs first. Disease progression will be determined based on the International Myeloma Working Group (IMWG) criteria. Patients who have not progressed or died will be censored at the date of last follow-up.
Overall Survival (OS)
Time frame: Up to 36 months after CAR-T infusion
Overall Survival (OS) is defined as the time from the date of CAR-T cell infusion to death from any cause. Patients who are alive at the time of analysis will be censored at their last known date of follow-up.
3 secondary outcomes
Objective Response Rate (ORR)
Time frame: Up to 36 months after CAR-T infusion
MRD
Time frame: Up to 36 months after CAR-T infusion
Adverse Events (AE)
Time frame: Up to 36 months after CAR-T infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2025.
- Individual participant dataSharing planned: No
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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.
Kailin Xu study chair
The Affiliated Hospital oh Xuzhou Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing autologous stem cell transplantation and CAR-T in people with Multiple Myeloma, Newly Diagnosed, run by Xuzhou Medical 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 6 inclusion and 5 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: opening soon, as of the sponsor's last update on 9 May 2025. 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 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kailin Xu (15162166166, lihmd@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression-Free Survival (PFS) (time frame: Up to 36 months after CAR-T 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 9 May 2025; AzTrials last refreshed its copy on 7 Nov 2026.