Immune ThrombocytopeniaPhase 2NCT06199089
A Randomized, Double-blind, Placebo-controlled, and Multi-center Clinical Study of CM313 in the Treatment of Immune Thrombocytopenia
A Phase 2 study testing CM313 Injection in people with Immune Thrombocytopenia and Treatment, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
To evaluate the efficacy and safety of CM313 in the treatment of immune thrombocytopenia in patients who have failed glucocorticoid therapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia, Treatment
- What is being tested?
- CM313 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 45 participants
- Where
- 1 siteChina
- Study dates
- 16 Jan 2024to Dec 2024
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 years, male or female.
- Before enrollment, the subjects have been clinically diagnosed with primary immune thrombocytopenia for no less than three months according to the American Society of Hematology guidelines 2011 Evidence-Based Practice Guideline (Neunert et al. 2011) or the International Consensus Report for the Investigation and Management of Primary Immune Thrombocytopenia (Provan et al. 2010), as applicable locally.
- Patients have failed glucocorticoid therapy (either due to inefficacy, efficacy could not be maintained, or relapse). Patients were required to have a response history (PLT≥50×10^9/L) to standard first-line treatment of ITP (glucocorticoid and/or intravenous immunoglobulin).
- Subjects with a platelet count of <30×10^9/L within the 24 hours prior to the first dose of the study drug; The mean platelet count of at least two separate assessments (at least 1 week apart) <30×10^9/L during the screening visit, and no platelet count > 35×10^9/L.
- ECOG performance status score of ≤2.
- Enrollment of subjects receiving maintenance therapy with a stable dosage is permitted, including glucocorticoids (≤0.5 mg/kg of prednisone or equivalent) or TPO receptor agonists. However, at the time of enrollment, subjects are restricted to using only one concomitant medication with a stable dose, and the concomitant medication must have been stable for a minimum of 4 weeks prior to the initial infusion of the study drug.
- For fertile female patients, a negative pregnancy test result is required. Fertile female and male patients must use effective contraception separately during the study and for 4 or 6 months after the cessation of study drug treatment.
- Subjects comprehensively understand and can adhere to the study protocol requirements and willingly signed the informed consent form.
Who cannot join
- Subjects with a known allergy to anti-CD38 monoclonal antibodies or excipients, or those who have previously received anti-CD38 monoclonal antibodies with ineffective therapeutic outcomes.
- Subjects who are diagnosed with autoimmune hemolytic anemia or various secondary thrombocytopenic disorders.
- Subjects with history of any thrombotic or embolic events or extensive and severe bleeding, such as hemoptysis, major upper gastrointestinal bleeding, intracranial hemorrhage, or the presence of sepsis or other irregular bleeding within the 12 months preceding the initiation of the first dose of study drug.
- Subjects who have participated in any other investigational drug studies (including vaccine studies) or been exposed to other investigational drugs within the first 4 weeks or 5 half-lives (whichever was longer) prior to the first dose of study drug.
- Subjects who have used anticoagulants or any agents with antiplatelet effects, such as aspirin, within 3 weeks prior to the first dose of study drug.
- Subjects who have received emergency treatment for ITP (e.g., methylprednisolone, platelet transfusion, intravenous immunoglobulin infusion, or thrombopoietin receptor agonist therapy) within 2 weeks prior to the first dose of study drug.
Show all 24Show fewer
- Subjects who have been treated with medications including azathioprine, danazol, dapsone, cyclosporine A, tacrolimus, and sirolimus within 4 weeks prior to the first dose of study drug. Subjects who have receive anti-CD20 monoclonal antibodies such as rituximab, or medications including cyclophosphamide and vindesine within 6 months prior to the first dose of study drug.
- Subjects who have undergone splenectomy within 6 months prior to the first dose of study drug.
- Subjects who have received live vaccines within 4 weeks prior to the first dose of study drug, or plan to receive any live vaccines during the course of the study.
- Subjects who are diagnosed with Myelodysplastic syndromes (MDS); Subjects with a with a history of malignancy within the 5 years prior to screening (excluding completely cured in situ cervical cancer and non-metastatic skin squamous cell carcinoma or basal cell carcinoma).
- Subjects who have undergone allogeneic stem cell transplantation or organ transplantation.
- Subjects with a clinically significant medical history, as perceived by investigators, that will pose risks to subjects' safety during the study or potentially affect the safety or efficacy analyses, includes major clinical histories such as circulatory system abnormalities, endocrine system abnormalities, nervous system diseases, blood system diseases, immune system diseases, mental diseases and metabolic abnormalities and so on. e.g., subjects with acute myocardial infarction, unstable angina pectoris, or severe arrhythmias (multifocal ventricular premature contractions, ventricular tachycardia, or ventricular fibrillation) within the 6 months before screening ; New York Heart Association (NYHA) class III-IV heart failure; subjects who were known to have had moderate or severe persistent asthma or chronic obstructive pulmonary disease within the 5 years prior to screening, or whose condition was currently poorly controlled;
- Subjects with a history of severe recurrent or chronic infections, or acute infections requiring systemic treatment with antibiotics, antiviral drugs, antiparasitic drugs, anti-amoebic drugs, or antifungal drugs within 4 weeks prior to the first dose and during the screening period, or superficial skin infections requiring systemic treatment within one week prior to the first dose of study drug. Notably, after the resolution of the infection, the subject may be re-screened.
- Subjects with a history of known or suspected immunosuppression, including invasive opportunistic infections such as histoplasmosis, listeriosis, coccidioidomycosis, pneumocystis pneumonia, and aspergillosis, even if the infection has resolved; or unusually frequent, recurrent, or prolonged infections (as judged by the investigator).
- Significant laboratory abnormalities during screening included:
- Alanine aminotransferase or aspartate aminotransferase greater than three times the upper limit of normal (ULN).
- Total bilirubin greater than 1.5 times the ULN (note: subjects diagnosed with Gilbert syndrome based on medical records should not be excluded based on this criterion).
- absolute neutrophil count < 1500/mm3.
- hemoglobin < 9g/dL; IgG < 500 mg/dL.
- lymphocyte count < 500/mm3. g) Creatinine clearance (CrCl) < 30 mL/min (i.e., CrCl ≥30 mL/min is allowed)
- Positive for HIV antibodies or syphilis antibodies.
- Subjects test positive for Hepatitis B surface antigen (HBsAg) or subjects test positive for hepatitis B core antibody and HBV-DNA (through polymerase chain reaction testing), or subjects test positive for hepatitis C virus antibody and HCV-RNA during the screening period. Subjects with positive hepatitis B core antibody but negative HBV-DNA can be enrolled, with HBV-DNA monitoring every 4 weeks.
- Pregnant or lactating women, or those intending to conceive or breastfeed during the study; and male partners intending to induce pregnancy during the study.
- Any other conditions unsuitable for participation in this study, as assessed by the investigator.
Trial locations and contacts
1 site in 1 country.
China1 site
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, 300020
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, and Multi-center Clinical Study to Assess Safety and Efficacy of Anti-Human CD38 Monoclonal Antibody CM313 in the Treatment of Primary Immune Thrombocytopenia
To evaluate the efficacy and safety of CM313 in the treatment of immune thrombocytopenia in patients who have failed glucocorticoid therapy.
Read the detailed description
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Therefore, there is still a lack of effective treatment for adult ITP, especially for recurrent and refractory ITP patients, which is one of the problems that have attracted more attention and need to be solved urgently.
The main pathogenesis of ITP is the loss of platelet autoantigen immune tolerance, which leads to abnormal activation of humoral and cellular immunity. It is characterized by antibody mediated platelet destruction and insufficient platelet production by megakaryocytes. The residual long-term autoreactive plasma cells may be a source of therapeutic resistance to autoimmune cytopenia. Antiplatelet specific plasma cells have been detected in the spleen of patients with rituximab refractory ITP. Therefore, the strategy of simply eliminating B cells may not work, because LLPC will continue to produce pathogenic antibodies. However, targeting LLPC becomes a new strategy to treat autoimmune diseases.
CM313, a kind of anti-CD38 antibody, is a new type of monoclonal antibody targeting CD38. It targets plasma cells and has carried out some clinical studies in multiple myeloma, with good therapeutic effects. In addition, the clinical trials of similar CD38 monoclonal antibody drugs, such as daratumumab, in the treatment of autoimmune diseases, including membranous nephropathy, systemic lupus erythematosus (SLE) and ITP, are also being carried out simultaneously. We assume that autologous reaction LLPC may be the cause of treatment failure in some ITP patients. Therefore, the use of CD38 monoclonal antibody to clear long-term surviving plasma cells in ITP patients may be a new strategy for treating ITP patients.
Therefore, the investigators designed this clinical trial to evaluate the efficacy and safety of CM313 in the treatment of immune thrombocytopenia in patients who have failed glucocorticoid therapy.
- Conditions
- Immune Thrombocytopenia, Treatment
- Being tested
- CM313 Injection, Placebo Injection
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CM313 InjectionDrug
Intravenous CM313 administration
This study adopts a randomized, double-blind, placebo-controlled design method. A total of 45 subjects were randomly assigned to experimental group (CM313, n = 30) and placebo comparator group (Placebo, n = 15) at a ratio of 2:1. Subjects assigned to experimental group were treated with CD38 monoclonal antibody (CM313: 16mg/kg/w) for 8 weeks.
The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of 16mg/kg CM313 once a week for 8 weeks to observe the efficacy and safety during treatment.
The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of CM313 after treatment.
Placebo InjectionDrug
Intravenous Placebo administration
This study adopts a randomized, double-blind, placebo-controlled design method. A total of 45 subjects were randomly assigned to experimental group (CM313, n = 30) and placebo comparator group (Placebo, n = 15) at a ratio of 2:1. Subjects assigned to placebo comparator group were treated with placebo of CM313 once a week for 8 weeks.
The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of placebo of CM313 once a week for 8 weeks to observe the efficacy and safety during treatment.
The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy after placebo administration.
| Study group | Role | What they receive |
|---|---|---|
| Intervention (CM313) | Experimental | 30 from 45 enrolled subjects receive CM313: once a week x 8 doses Receives: Drug: CM313 Injection |
| Intervention (Placebo) | Placebo comparator | 15 from 45 enrolled subjects receive placebo: once a week x 8 doses Receives: Drug: Placebo Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of the efficacy after CM313/Placebo treatment at week 8
Proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least twice the baseline platelet count without bleeding at week 8 after initial administration in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period
Measured: 8 weeks
Secondary outcomes (10)
Evaluation of the efficacy after CM313/Placebo treatment at each visit period
Proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least twice the baseline platelet count without bleeding at each visit period after initial administration in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period
Measured: 24 weeks
Evaluation of the complete remission rate after CM313/Placebo treatment at each visit period
Proportion of subjects with a platelet count ≥ 100 × 10^9/L without bleeding at each visit period after initial administration in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period
Measured: 24 weeks
Proportion of subjects with a platelet count ≥ 50 × 10^9/L at each visit period
Proportion of subjects with a platelet count ≥ 50 × 10^9/L at each visit period in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period
Measured: 24 weeks
Time to response (TTR)
Time needed from treatment initiation to platelet count ≥30×10^9/L and at least twice the baseline platelet count
Measured: 24 weeks
Cumulative weeks of platelet ≥30×10^9/L
Cumulative weeks of platelet ≥30×10^9/L without having received any platelet elevating therapy or having had dose increment of TPO-RA and/or corticosteroids
Measured: 24 weeks
Cumulative weeks of platelet ≥50×10^9/L
Cumulative weeks of platelet ≥50×10^9/L without having received any platelet elevating therapy or having had dose increment of TPO-RA and/or corticosteroids
Measured: 24 weeks
Emergency treatment
Percentage of patients who need emergency treatment after CM313/Placebo treatment
Measured: 24 weeks
Concomitant maintenance drug
Changes in concomitant maintenance therapy at week 12 compared with that before CM313/Placebo treatment
Measured: 12 weeks
Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale
Changes of the subjects' numbers in WHO bleeding score after CM313/Placebo treatment according to the reported World Health Organization's Bleeding Scale at week 12. The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Measured: 12 weeks
Adverse events assessment
Incidence, severity, and relationship of treatment emergent adverse events after CM313/Placebo treatment
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2024
- Study start16 Jan 2024
- Primary completion28 Nov 2024
- Study completionDec 2024 (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 Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Tianjin People's Hospital, The Second Affiliated Hospital of Kunming Medical University, Henan Cancer Hospital, Tianjin Medical University Second Hospital, North China University of Science and Technology
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, MD, Principal investigator, Chinese Academy of Medical Science and Blood Disease Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06199089
- Sponsor's protocol ID
- IIT2023068
- Status last verified
- Dec 2024
- First submitted
- 25 Dec 2023
- Last updated
- 30 Dec 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Researchers qualified can request the dataset, including de-identified individual subject data. Data may be requested from PI from 12 months 36 months after study completion.
Publications
Chen Y, Xu Y, Dai J, Sun T, Li H, Hua Z, Zhou Z, Zhou H, Yan Z, Zhao X, Xue F, Liu W, Liu X, Fu R, Wang W, Chi Y, Dong H, Ju M, Dai X, Gu W, Pei X, Yang R, Zhang L. Anti-CD38 monoclonal antibody CM313 for primary immune thrombocytopenia: multicentre, randomised, placebo controlled, phase 2 trial. BMJ. 2025 Oct 21;391:e084314. doi: 10.1136/bmj-2025-084314.
PubMed 41120215
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, and Multi-center Clinical Study to Assess Safety and Efficacy of Anti-Human CD38 Monoclonal Antibody CM313 in the Treatment of Primary Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT06199089
- Acronym / sponsor ID
- IIT2023068
- Conditions
- Immune Thrombocytopenia, Treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Tianjin People's Hospital, The Second Affiliated Hospital of Kunming Medical University, Henan Cancer Hospital, Tianjin Medical University Second Hospital, North China University of Science and Technology
- Study start
- 16 Jan 2024 (actual)
- Primary completion
- 28 Nov 2024 (actual)
- Study completion
- Dec 2024 (estimated)
- First posted
- 10 Jan 2024 (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.
To evaluate the efficacy and safety of CM313 in the treatment of immune thrombocytopenia in patients who have failed glucocorticoid therapy.
Detailed description
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Therefore, there is still a lack of effective treatment for adult ITP, especially for recurrent and refractory ITP patients, which is one of the problems that have attracted more attention and need to be solved urgently.
The main pathogenesis of ITP is the loss of platelet autoantigen immune tolerance, which leads to abnormal activation of humoral and cellular immunity. It is characterized by antibody mediated platelet destruction and insufficient platelet production by megakaryocytes. The residual long-term autoreactive plasma cells may be a source of therapeutic resistance to autoimmune cytopenia. Antiplatelet specific plasma cells have been detected in the spleen of patients with rituximab refractory ITP. Therefore, the strategy of simply eliminating B cells may not work, because LLPC will continue to produce pathogenic antibodies. However, targeting LLPC becomes a new strategy to treat autoimmune diseases.
CM313, a kind of anti-CD38 antibody, is a new type of monoclonal antibody targeting CD38. It targets plasma cells and has carried out some clinical studies in multiple myeloma, with good therapeutic effects. In addition, the clinical trials of similar CD38 monoclonal antibody drugs, such as daratumumab, in the treatment of autoimmune diseases, including membranous nephropathy, systemic lupus erythematosus (SLE) and ITP, are also being carried out simultaneously. We assume that autologous reaction LLPC may be the cause of treatment failure in some ITP patients. Therefore, the use of CD38 monoclonal antibody to clear long-term surviving plasma cells in ITP patients may be a new strategy for treating ITP patients.
Therefore, the investigators designed this clinical trial to evaluate the efficacy and safety of CM313 in the treatment of immune thrombocytopenia in patients who have failed glucocorticoid therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention (CM313) | Experimental | Drug: CM313 Injection30 from 45 enrolled subjects receive CM313: once a week x 8 doses |
| Intervention (Placebo) | Placebo comparator | Drug: Placebo Injection15 from 45 enrolled subjects receive placebo: once a week x 8 doses |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age ≥18 years, male or female.
- Before enrollment, the subjects have been clinically diagnosed with primary immune thrombocytopenia for no less than three months according to the American Society of Hematology guidelines 2011 Evidence-Based Practice Guideline (Neunert et al. 2011) or the International Consensus Report for the Investigation and Management of Primary Immune Thrombocytopenia (Provan et al. 2010), as applicable locally.
- Patients have failed glucocorticoid therapy (either due to inefficacy, efficacy could not be maintained, or relapse). Patients were required to have a response history (PLT≥50×10^9/L) to standard first-line treatment of ITP (glucocorticoid and/or intravenous immunoglobulin).
- Subjects with a platelet count of <30×10^9/L within the 24 hours prior to the first dose of the study drug; The mean platelet count of at least two separate assessments (at least 1 week apart) <30×10^9/L during the screening visit, and no platelet count > 35×10^9/L.
- ECOG performance status score of ≤2.
Key exclusion criteria (24 listed)
- Subjects with a known allergy to anti-CD38 monoclonal antibodies or excipients, or those who have previously received anti-CD38 monoclonal antibodies with ineffective therapeutic outcomes.
- Subjects who are diagnosed with autoimmune hemolytic anemia or various secondary thrombocytopenic disorders.
- Subjects with history of any thrombotic or embolic events or extensive and severe bleeding, such as hemoptysis, major upper gastrointestinal bleeding, intracranial hemorrhage, or the presence of sepsis or other irregular bleeding within the 12 months preceding the initiation of the first dose of study drug.
- Subjects who have participated in any other investigational drug studies (including vaccine studies) or been exposed to other investigational drugs within the first 4 weeks or 5 half-lives (whichever was longer) prior to the first dose of study drug.
- Subjects who have used anticoagulants or any agents with antiplatelet effects, such as aspirin, within 3 weeks prior to the first dose of study drug.
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 |
|---|---|---|
| CM313 InjectionIntravenous CM313 administration This study adopts a randomized, double-blind, placebo-controlled design method. A total of 45 subjects were randomly assigned to experimental group (CM313, n = 30) and placebo comparator group (Placebo, n = 15) at a ratio of 2:1. Subjects assigned to experimental group were treated with CD38 monoclonal antibody (CM313: 16mg/kg/w) for 8 weeks. The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of 16mg/kg CM313 once a week for 8 weeks to observe the efficacy and safety during treatment. The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of CM313 after treatment. | Drug | Intervention (CM313) |
| Placebo InjectionIntravenous Placebo administration This study adopts a randomized, double-blind, placebo-controlled design method. A total of 45 subjects were randomly assigned to experimental group (CM313, n = 30) and placebo comparator group (Placebo, n = 15) at a ratio of 2:1. Subjects assigned to placebo comparator group were treated with placebo of CM313 once a week for 8 weeks. The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of placebo of CM313 once a week for 8 weeks to observe the efficacy and safety during treatment. The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy after placebo administration. | Drug | Intervention (Placebo) |
Primary outcome
Evaluation of the efficacy after CM313/Placebo treatment at week 8
Time frame: 8 weeks
Proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least twice the baseline platelet count without bleeding at week 8 after initial administration in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period
10 secondary outcomes
Evaluation of the efficacy after CM313/Placebo treatment at each visit period
Time frame: 24 weeks
Evaluation of the complete remission rate after CM313/Placebo treatment at each visit period
Time frame: 24 weeks
Proportion of subjects with a platelet count ≥ 50 × 10^9/L at each visit period
Time frame: 24 weeks
Time to response (TTR)
Time frame: 24 weeks
Cumulative weeks of platelet ≥30×10^9/L
Time frame: 24 weeks
Cumulative weeks of platelet ≥50×10^9/L
Time frame: 24 weeks
Emergency treatment
Time frame: 24 weeks
Concomitant maintenance drug
Time frame: 12 weeks
Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale
Time frame: 12 weeks
Adverse events assessment
Time frame: 24 weeks
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 30 Dec 2024.
- Derived from this study: PubMed 41120215Chen Y, Xu Y, Dai J, Sun T, Li H, Hua Z, Zhou Z, Zhou H, Yan Z, Zhao X, Xue F, Liu W, Liu X, Fu R, Wang W, Chi Y, Dong H, Ju M, Dai X, Gu W, Pei X, Yang R, Zhang L. Anti-CD38 monoclonal antibody CM313 for primary immune thrombocytopenia: multicentre, randomised, placebo controlled, phase 2 trial. BMJ. 2025 Oct 21;391:e084314. doi: 10.1136/bmj-2025-084314.
- Individual participant dataSharing planned: Yes. Researchers qualified can request the dataset, including de-identified individual subject data. Data may be requested from PI from 12 months 36 months after study completion.
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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.
Lei Zhang, MD principal investigator
Chinese Academy of Medical Science and Blood Disease Hospital
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 CM313 Injection in people with Immune Thrombocytopenia and Treatment, run by Institute of Hematology & Blood Diseases Hospital, China 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 8 inclusion and 24 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: under way, not looking for participants, as of the sponsor's last update on 30 Dec 2024. 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. Some participants in this study may receive a placebo. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of the efficacy after CM313/Placebo treatment at week 8 (time frame: 8 weeks). 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 NCT06199089, 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 30 Dec 2024; AzTrials last refreshed its copy on 2 Nov 2026.