Extramedullary Multiple MyelomaPhase 1NCT05201118
A Study of a Fully Human BCMA-targeting CAR (CT103A) Combined With Selinexor in Patients With Relapsed/Refractory Extramedullary Multiple Myeloma
A Phase 1 study testing Selinexor and CT103A in people with Extramedullary Multiple Myeloma, run by Chunrui Li at 1 site in China.
Trial summary
This study is a single-center, open Phase I study, to observe the effectiveness and safety of CT103A combined with different doses of Selinexor in patients with relapsed/refractory extramedullary multiple myeloma, and the pharmacokinetics of Selinexor and CT103A Kinetic and pharmacodynamic characteristics. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Extramedullary Multiple Myeloma
- What is being tested?
- Selinexor and CT103A
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2022to 31 Dec 2023
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects must satisfy all the following criteria to be enrolled in the study:
- age ≥18 years old, male or female.
- Subjects with diagnosed relapsed or refractory extramedullary multiple myeloma according to IMWG criteria and have had at least 3 prior lines of therapy
- Evidence of cell membrane BCMA expression, as determined by a validated immunohistochemistry (IHC) or flow cytometry of tumor tissue(e.g., bone marrow biopsies, or plasmacytoma).
- Subjects with extramedullary myeloma require extramedullary lesions with a maximum diameter of ≥2cm
- ECOG score is ≤ 2
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- Estimated life expectancy ≥ 12 weeks.
- Subjects should have adequate organ function:
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- Absolute neutrophil count (ANC) ≥1×10^9 /L; absolute lymphocyte count (ALC) ≥0.3×10^9 /L; platelets ≥50×10^9 /L; hemoglobin ≥60 g/L.
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5×upper limit of normal (ULN); total serum bilirubin ≤ 1.5×ULN.
- Creatinine clearance rate (CrCl) calculated according to Cockcroft-Gault formula ≥ 40 ml/min.
- Fibrinogen ≥ 1.0 g/L; activated partial thromboplastin time (APTT) ≤ 1.5×ULN, prothrombin time (PT) ≤1.5×ULN.
- SpO2 > 91%.
- Left ventricular ejection fraction (LVEF) ≥ 50%. 8. The subject and his/her spouse agree to use an effective contraceptive tool or medication (excluding safety period contraception) from the date of the subject's informed consent to one year post CAR T cell infusion.
- 9. Subject must sign the informed consent form approved by the ethics board in person before starting any screening procedure.
Who cannot join
- The presence of any of the following will exclude a subject from enrollment:
- Subjects who are known to be resistant to Selinexor;
- Subjects who need to use immunosuppressive agents for a long time due to graft-versus-host disease (GVHD) or autoimmune diseases.
- Subjects have received any anti-cancer treatment as follows: monoclonal antibody for treating multiple myeloma within 21 days before leukapheresis, or cytotoxic therapy or proteasome inhibitors within 14 days before leukapheresis, or immunomodulatory agents within 7 days before leukapheresis, or anti-tumor treatments other than those listed above within 30 days before leukapheresis.
- Subjects who were receiving a used therapeutic dose of corticosteroid treatment (defined as prednisone or equivalent > 20mg) within 7 days prior to screening, except for physiological alternatives, inhalation, or topical use.
- Subjects with hypertension that cannot be controlled by medication
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- Subjects with serious heart disease: including but not limited to unstable angina, myocardial infarction (within 6 months prior to screening), congestive heart failure (NYHA classification ≥III), and severe arrhythmias.
- Subjects with systemic diseases that the investigator determined to be unstable include, but are not limited to, severe liver and kidney or metabolic diseases requiring medical treatment.
- Subjects with second malignancies in addition to MM within the past 5 years before the screening, exceptions to this criterion: successfully treated cervical carcinoma in situ and non-metastatic basal or squamous cell skin carcinoma, local prostate cancer after radical surgery, and ductal carcinoma in situ of the breast after radical surgery.
- Subjects with a history of organ transplantation.
- Subjects have received major surgery within 2 weeks prior to leukapheresis or plan to receive surgery during the study or within 2 weeks after the study treatment (excluding local anesthesia)
- Subjects participated in another interventional clinical study within 1 month before signing the informed consent (ICF).
- Subjects with any uncontrolled active infection needed to receive systemic therapy within 7 days before leukapheresis.
- Positive for any of the following tests:
- Hepatitis B virus (HBV) surface antigen (HBsAg) or hepatitis B core antibody-positive and detectable HBV DNA in peripheral blood
- Hepatitis C virus (HCV) antibody and hepatitis C virus RNA in peripheral blood
- Human immunodeficiency virus (HIV) antibody
- Cytomegalovirus (CMV) DNA
- Treponema Pallidum antibody
- Pregnant or lactating women.
- Subjects with mental illness or consciousness disorder or disease of the central nervous system
- Other conditions that researchers consider inappropriate for enrollment.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, 430000
Chunrui Li, +86 13647233185, cunrui5650@126.com
About this study
Official title: Phase I Clinical Study of a Fully Human BCMA-targeting CAR (CT103A) Combined With Selinexor in the Treatment of Patients With Relapsed/Refractory Extramedullary Multiple Myeloma
This study is a single-center, open Phase I study, to observe the effectiveness and safety of CT103A combined with different doses of Selinexor in patients with relapsed/refractory extramedullary multiple myeloma, and the pharmacokinetics of Selinexor and CT103A Kinetic and pharmacodynamic characteristics.
Read the detailed description
In this study, two dose groups of 20 mg/week and 40 mg/week will be set for Selinexor, and the dose of CT103A is 1.0×106 cells/Kg. Subjects in all dose groups will firstly receive a single dose infusion of CT103A, at least 1 month post infusion and platelet recovery to ≥50×109/L. Then subjects began to take Selinexor once a week for one year. Each dose group level will include 8-10 subjects, and a total of 16-20 subjects are expected to be enrolled.
- Conditions
- Extramedullary Multiple Myeloma
- Being tested
- Selinexor, CT103A
- Keywords
- CT103A, Selinexor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelinexorDrug
Selinexor, 20 mg/tablet, is a first-in-class, oral Selective-Inhibitor-of-Nuclear-Export (SINE) compound that impedes XPO-1which is a major nuclear export protein of macromolecular cargo frequently overexpressed in MM.
CT103ADrug
CT103A consists of autologous T lymphocytes transduced with anti-BCMA CAR lentiviral vector that containing a unique CAR structure with a fully human single-chain variable fragment (scFv).
| Study group | Role | What they receive |
|---|---|---|
| CT103A combined with Selinexor | Experimental | All subjects will be assigned to two Selinexor dose groups of 20 mg/week and 40 mg/week after receiving a single dose infusion of CT103A. Receives: Drug: Selinexor; Drug: CT103A |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression-free survival (PFS)
The time from the start of CT103A treatment for the subjects to the first disease progression or death for any reason.
Measured: 1 year post CT103A infusion
Objective response rate (ORR)
The percentage of subjects who achieved sCR、CR、VGPR、PR.
Measured: 1 year post CT103A infusion
Duration of response (DOR) after administration
DOR will be calculated among responders (with a PR or better response) from the date of initial documentation of a response (PR or better) to the date of first documented evidence of progressive disease, as defined in the IMWG criteria
Measured: 1 year post CT103A infusion
Secondary outcomes (13)
Overall survival (OS)
OS is measured from the date of the initial infusion of CT103A to the date of the participant's death.
Measured: 1 year post CT103A infusion
Minimal Residual Disease (MRD) efficacy evaluation
MRD evaluation according to IMWG, including the proportion of subjects who achieved MRD negative and the duration of MRD negative.
Measured: 1 year post CT103A infusion
Type and incidence of adverse events (AEs) and serious adverse events (SAEs) by dose group
Calculate type and incidence of adverse events (AE), serious adverse event (SAE), including those happened after lymphodepletion and after infusion, those related to study drug and lymphodepletion, or those that led to withdrawal from the study. They will also be aggregated by systematic organ classification (SOC), preferred term (PT), and severity
Measured: 1 year post CT103A infusion
Pharmacokinetics - Cmax of CT103A
The maximum transgene level at Cmax fo CT103A
Measured: 1 year post CT103A infusion
Pharmacokinetics - Tmax of CT103A
The maximum transgene level at Tmax fo CT103A
Measured: 1 year post CT103A infusion
Pharmacokinetics - AUC0-28days of CT103A
Area under the curve of CT103A cells from time zero to Day 28 of CT103A
Measured: 1 year post CT103A infusion
Pharmacokinetics - AUC0-90days of CT103A
Area under the curve of CT103A cells from time zero to Day 90 of CT103A
Measured: 1 year post CT103A infusion
Pharmacokinetics of Selinexor
The changes of concentration of Selinexor in peripheral blood will be assessed.
Measured: 1 year post CT103A infusion
PD endpoints
The concentration levels of CAR-T-related serum cytokines such as Ferritin and IL-6
Measured: 1 year post CT103A infusion
Health-related quality of life assessment
HRQoL will be assessed by the European Organization for Cancer Research and Treatment Quality of Life Questionnaire (EORTC-QLQ-C30)
Measured: 1 year post CT103A infusion
Appraisal of life quality
Appraisal of life quality of the subjects will be assessed by the Quality of Life Multiple Myeloma Module Questionnaire (QLQ-MY20)
Measured: 1 year post CAR-T cell infusion
Evaluation of lymphocyte subsets
Lymphocyte subsets will be assessed by FACS
Measured: 1 year post CAR-T cell infusion
Concentration of immunoglobulins
The levels of Immunoglobulins in peripheral blood will be assessed to monitor changes at each time point
Measured: 1 year post CAR-T cell infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2022
- Study start1 Jan 2022
- Primary completion31 Dec 2022 (estimated)
- Study completion31 Dec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Extramedullary Multiple MyelomaStudies 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
- Model description
- The efficacy and safety, as well as the pharmacokinetic and pharmacodynamic characteristics of CT103A combined with different doses of Selinexor will be assessed in patients with relapsed/refractory extramedullary multiple myeloma. In this study, CT103A will be infused at the dose of 1.0×10\^6 cells/Kg, while Selinexor was set as two dosage groups of 20 mg/week and 40 mg/week. Subjects in all dose groups received a single infusion of CT103A, and at least 1 month after the infusion of CT103A, Selinexor was administered orally once a week for one year when platelet count recovered to ≥50×10\^9 /L. 8 to 10 subjects were enrolled at each dose group level, with a total of 16 to 20 subjects expected to be enrolled.
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chunrui Li
- Sponsor type
- University, hospital or other
- Collaborators
- Nanjing IASO Biotechnology Co., Ltd.
- Responsible party
- Sponsor-investigator, Chunrui Li, Tongji Hospital
- Investigators
- Chunrui Li, Principal investigator, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05201118
- Sponsor's protocol ID
- CAR CT103A SELINEXOR001
- Status last verified
- May 2022
- First submitted
- 5 Jan 2022
- Last updated
- 24 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Keam SJ. Equecabtagene Autoleucel: First Approval. Mol Diagn Ther. 2023 Nov;27(6):781-787. doi: 10.1007/s40291-023-00673-y. Epub 2023 Sep 2.
PubMed 37658205
Study at a glance
From the registry record, last updated by the sponsor on 24 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Clinical Study of a Fully Human BCMA-targeting CAR (CT103A) Combined With Selinexor in the Treatment of Patients With Relapsed/Refractory Extramedullary Multiple Myeloma
- ClinicalTrials.gov ID
- NCT05201118
- Acronym / sponsor ID
- CAR CT103A SELINEXOR001
- Conditions
- Extramedullary Multiple Myeloma
- Keywords
- CT103A, Selinexor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Chunrui LiUniversity, hospital or other
- Collaborators
- Nanjing IASO Biotechnology Co., Ltd.
- Study start
- 1 Jan 2022 (actual)
- Primary completion
- 31 Dec 2022 (estimated)
- Study completion
- 31 Dec 2023 (estimated)
- First posted
- 21 Jan 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 study is a single-center, open Phase I study, to observe the effectiveness and safety of CT103A combined with different doses of Selinexor in patients with relapsed/refractory extramedullary multiple myeloma, and the pharmacokinetics of Selinexor and CT103A Kinetic and pharmacodynamic characteristics.
Detailed description
In this study, two dose groups of 20 mg/week and 40 mg/week will be set for Selinexor, and the dose of CT103A is 1.0×106 cells/Kg. Subjects in all dose groups will firstly receive a single dose infusion of CT103A, at least 1 month post infusion and platelet recovery to ≥50×109/L. Then subjects began to take Selinexor once a week for one year. Each dose group level will include 8-10 subjects, and a total of 16-20 subjects are expected to be enrolled.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The efficacy and safety, as well as the pharmacokinetic and pharmacodynamic characteristics of CT103A combined with different doses of Selinexor will be assessed in patients with relapsed/refractory extramedullary multiple myeloma. In this study, CT103A will be infused at the dose of 1.0×10^6 cells/Kg, while Selinexor was set as two dosage groups of 20 mg/week and 40 mg/week. Subjects in all dose groups received a single infusion of CT103A, and at least 1 month after the infusion of CT103A, Selinexor was administered orally once a week for one year when platelet count recovered to ≥50×10^9 /L. 8 to 10 subjects were enrolled at each dose group level, with a total of 16 to 20 subjects expected to be enrolled.
| Arm | Type | Receives |
|---|---|---|
| CT103A combined with Selinexor | Experimental | Drug: Selinexor, Drug: CT103AAll subjects will be assigned to two Selinexor dose groups of 20 mg/week and 40 mg/week after receiving a single dose infusion of CT103A. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Subjects must satisfy all the following criteria to be enrolled in the study:
- <!-- -->
- 9. Subject must sign the informed consent form approved by the ethics board in person before starting any screening procedure.
Key exclusion criteria (22 listed)
- The presence of any of the following will exclude a subject from enrollment:
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 |
|---|---|---|
| SelinexorSelinexor, 20 mg/tablet, is a first-in-class, oral Selective-Inhibitor-of-Nuclear-Export (SINE) compound that impedes XPO-1which is a major nuclear export protein of macromolecular cargo frequently overexpressed in MM. | Drug | CT103A combined with Selinexor |
| CT103ACT103A consists of autologous T lymphocytes transduced with anti-BCMA CAR lentiviral vector that containing a unique CAR structure with a fully human single-chain variable fragment (scFv). | Drug | CT103A combined with Selinexor |
Primary outcome
Progression-free survival (PFS)
Time frame: 1 year post CT103A infusion
The time from the start of CT103A treatment for the subjects to the first disease progression or death for any reason.
Objective response rate (ORR)
Time frame: 1 year post CT103A infusion
The percentage of subjects who achieved sCR、CR、VGPR、PR.
Duration of response (DOR) after administration
Time frame: 1 year post CT103A infusion
DOR will be calculated among responders (with a PR or better response) from the date of initial documentation of a response (PR or better) to the date of first documented evidence of progressive disease, as defined in the IMWG criteria
13 secondary outcomes
Overall survival (OS)
Time frame: 1 year post CT103A infusion
Minimal Residual Disease (MRD) efficacy evaluation
Time frame: 1 year post CT103A infusion
Type and incidence of adverse events (AEs) and serious adverse events (SAEs) by dose group
Time frame: 1 year post CT103A infusion
Pharmacokinetics - Cmax of CT103A
Time frame: 1 year post CT103A infusion
Pharmacokinetics - Tmax of CT103A
Time frame: 1 year post CT103A infusion
Pharmacokinetics - AUC0-28days of CT103A
Time frame: 1 year post CT103A infusion
Pharmacokinetics - AUC0-90days of CT103A
Time frame: 1 year post CT103A infusion
Pharmacokinetics of Selinexor
Time frame: 1 year post CT103A infusion
PD endpoints
Time frame: 1 year post CT103A infusion
Health-related quality of life assessment
Time frame: 1 year post CT103A infusion
Appraisal of life quality
Time frame: 1 year post CAR-T cell infusion
Evaluation of lymphocyte subsets
Time frame: 1 year post CAR-T cell infusion
Concentration of immunoglobulins
Time frame: 1 year post CAR-T cell infusion
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 May 2022.
- Derived from this study: PubMed 37658205Keam SJ. Equecabtagene Autoleucel: First Approval. Mol Diagn Ther. 2023 Nov;27(6):781-787. doi: 10.1007/s40291-023-00673-y. Epub 2023 Sep 2.
- 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. This study is not recruiting at the moment.
Chunrui Li principal investigator
Tongji 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 Selinexor and CT103A in people with Extramedullary Multiple Myeloma, run by Chunrui Li 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 16 inclusion and 22 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 24 May 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 Chunrui Li (86-13647233185, cunrui5650@126.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: 1 year post CT103A infusion). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05201118, which also lists 1 linked publication.
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 24 May 2022; AzTrials last refreshed its copy on 6 Nov 2026.