Immune Thrombocytopenia (ITP)Phase 2NCT06799611
An Open, Exploratory Clinical Study of CM336 in the Treatment of Immune Thrombocytopenia
A Phase 2 study testing CM336 Injection in people with Immune Thrombocytopenia (ITP) 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 CM336 in the treatment of refractory adult primary immune thrombocytopenia Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia (ITP), Treatment
- What is being tested?
- CM336 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 3 Mar 2025to 31 Jan 2027
- 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 and above, male or female;
- Conform to the diagnostic criteria of persistent or chronic immune thrombocytopenia (ITP);
- Failure of previous glucocorticoid therapy;
- In the second-line treatment phase, eligible subjects must meet any of the following criteria: (1) Demonstrate no response to treatment with at least one thrombopoietin receptor agonist (including but not limited to recombinant human thrombopoietin [rhTPO], eltrombopag, hetrombopag, avatrombopag, or romiplostim); Fail to achieve sustained response (manifested as non-response, loss of response, or disease relapse) following anti-CD20 monoclonal antibody therapy (e.g., rituximab) or anti-CD38 monoclonal antibody therapy; (2) Exhibit no therapeutic response or experience disease relapse after splenectomy.
- The platelet count was <30×109/L within 48 hours before the first administration;
- ECOG physical state score ≤ 2 points;
- Patients receiving maintenance treatment (including corticosteroids (less than or equal to 20mg prednisone), TPO receptor agonists, etc.) must have a stable dose at least 4 weeks before the first administration;
- Signed and dated written informed consent;
Who cannot join
- Received any treatment of anti-BCMA antibody drug;
- Accompanied by autoimmune hemolytic anemia, or various secondary and hereditary thrombocytopenia;
- History of any thrombotic or embolic events in the 12 months prior to the first dose or accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc;
- Participated in any other study drug or exposure to other study drugs within 4 weeks or 5 half-lives before the first dose (whichever is longer);
- Use of anticoagulants or any drug with antiplatelet effects (such as aspirin) within 3 weeks before the first dose;
- Treatment with ITP (methylprednisolone, platelet, gamma-globulin infusion or TPO receptor agonist therapy) within 2 weeks before the first dose;
Show all 20Show fewer
- Splenectomy was performed within 6 months before the first dose;
- Patients who received azathioprine, danazol, cyclosporine A, tacrolimus, sirolimus, etc., within 4 weeks prior to the first dose; or received treatments such as CD20 monoclonal antibodies (e.g., rituximab), CD38 monoclonal antibodies, cyclophosphamide, or vindesine within 3 months prior to the first dose；
- Received a live vaccine within 4 weeks before the first dose, or planned to receive any live vaccine during the clinical trial;
- Those who have received allogeneic stem cell transplantation or organ transplantation in the past;
- Other serious diseases that may limit the subject's participation in this trial (such as diabetes; Hepatic and renal insufficiency; Severe cardiac insufficiency; Myocardial obstruction or unstable arrhythmia or unstable angina pectoris in recent 6 months; Gastric ulcer, etc.);
- Patients with malignant tumors within 5 years before the screening;
- A history of severe recurrent or chronic infection;
- A known or suspected history of immunosuppression, including a history of invasive opportunistic infections;
- Clinically significant laboratory abnormalities at the time of screening;
- HIV antibody or syphilis antibody positive;
- Accompanied by uncontrollable active infection, including hepatitis B, hepatitis C; Pregnant women, suspected pregnancies (positive pregnancy test for human chorionic gonadotropin in urine at screening) and lactating patients;
- Patients with mental disorders who cannot normally obtain informed consent and conduct trials and follow-up;
- Patients whose symptoms of toxicity from pre-trial treatment have not resolved;
- Any other ineligibility for subjects in the study was assessed by the investigator.
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, 1 recruiting
- Recruiting
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, 300020
Yunfei Chen, MD, +86-22-23909009, chenyunfei@ihcams.ac.cn
About this study
Official title: An Open, Exploratory Clinical Study to Assess Safety and Efficacy of BCMA-CD3 Bispecific Antibody CM336 in the Treatment of Primary Immune Thrombocytopenia
To evaluate the efficacy and safety of CM336 in the treatment of refractory adult primary immune thrombocytopenia
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.
B-cell maturation antigen (BCMA) is mainly restricted in some B-cell subsets and plasma cells, but not in other normal human cells and tissues. BCMA does not maintain normal B-cell homeostasis, but is necessary for long-lived plasma cell survival. Plasma cells are the main antibody-producing cells in the body, and the production of autoantibodies is the main pathogenesis of ITP. BCMA-CD3 bispecific antibody CM336 can bind BCMA-positive B cells and plasma cells, and bind CD3-positive T cells at the same time to induce T cell activation and play the role of T-cell-dependent cellular cytotoxicity (TDCC). CM336 can eliminate BCMA-positive B cells and plasma cells through the above mechanism, directly reduce the production of pathogenic antibodies, and then achieve therapeutic effect. Due to the deep clearance of immune cells by bispecific antibodies, CM336 may achieve the effect of immune reset and cure autoimmune diseases such as ITP. The BCMA-CD3 bispecific antibody teclistamab has been reported to be used in the treatment of refractory systemic lupus erythematosus, systemic sclerosis, primary Sjogren's syndrome, idiopathic inflammatory myopathy, rheumatoid arthritis and other autoimmune diseases, with significant clinical effect and good safety.
Therefore, the investigators designed this clinical trial to evaluate the safety and efficacy of CM336 in the treatment of refractory ITP.
- Conditions
- Immune Thrombocytopenia (ITP), Treatment
- Being tested
- CM336 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.
CM336 InjectionDrug
subcutaneous CM336 administration step-up dosing Dose and frequency of CM336 according to the protocol
| Study group | Role | What they receive |
|---|---|---|
| Intervention(CM336) | Experimental | CM336 (Anticipated enrollment of 20 to 30 subjects) Receives: Drug: CM336 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the efficacy after CM336 treatment at week 12
Proportion of subjects whose platelet counts ≥ 30×10^9/L and at least two times of baseline platelet count without bleeding at week 12
Measured: 12 weeks
Safety of CM336
Incidence, severity, and relationship of treatment emergent adverse events after CM336 treatment
Measured: 52 weeks
Secondary outcomes (7)
Proportion of subjects with a platelet count ≥ 50 × 10^9/L and ≥ 100 × 10^9/L at each visit
Proportion of subjects with a platelet count ≥ 50 × 10^9/L and ≥ 100 × 10^9/L at each visit after administration.
Measured: 52 weeks
Duration from treatment initiation to platelet count ≥30×10^9/L and at least two times of baseline platelet count, platelet count ≥50×10^9/L, platelet count ≥100×10^9/L
Duration from treatment initiation to platelet count ≥30×10^9/L and at least two times of baseline platelet count, platelet count ≥50×10^9/L, platelet count ≥100×10^9/L
Measured: 52 weeks
Cumulative weeks of platelet ≥30×10^9/L and at least two times of baseline platelet count, platelet count ≥50×10^9/L, platelet count ≥100×10^9/L
Cumulative weeks of platelet ≥30×10^9/L and at least two times of baseline platelet count, platelet count ≥50×10^9/L, platelet count ≥100×10^9/L
Measured: 52 weeks
Other efficacy evaluation
Platelet counts at each visit
Measured: 52 weeks
Proportion of subjects receiving emergency treatment
Proportion of subjects receiving emergency treatment within 12 weeks and throughout the clinical trial
Measured: 52 weeks
Changes in world health organization (WHO) bleeding scale
Changes in world health organization (WHO) bleeding scale without emergency treatment. Changes of every subject in WHO bleeding score after CM336 treatment according to the reported World Health Organization's Bleeding Scale. 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: 52 weeks
Measurements of platelet glycoprotein (GP) autoantibodies
level of anti-GPIIb/IIIa and Ib/IX antibodies before and after CM336 therapy
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2025
- Study start3 Mar 2025
- Primary completion31 Jan 2026 (estimated)
- Study completion31 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune Thrombocytopenia (ITP)Studies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- 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
- NCT06799611
- Sponsor's protocol ID
- IIT2024050
- Status last verified
- Jan 2025
- First submitted
- 23 Jan 2025
- Last updated
- 13 Mar 2025
- 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 to 36 months after study completion.
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open, Exploratory Clinical Study to Assess Safety and Efficacy of BCMA-CD3 Bispecific Antibody CM336 in the Treatment of Primary Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT06799611
- Acronym / sponsor ID
- IIT2024050
- Conditions
- Immune Thrombocytopenia (ITP), Treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 3 Mar 2025 (actual)
- Primary completion
- 31 Jan 2026 (estimated)
- Study completion
- 31 Jan 2027 (estimated)
- First posted
- 29 Jan 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.
To evaluate the efficacy and safety of CM336 in the treatment of refractory adult primary immune thrombocytopenia
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.
B-cell maturation antigen (BCMA) is mainly restricted in some B-cell subsets and plasma cells, but not in other normal human cells and tissues. BCMA does not maintain normal B-cell homeostasis, but is necessary for long-lived plasma cell survival. Plasma cells are the main antibody-producing cells in the body, and the production of autoantibodies is the main pathogenesis of ITP. BCMA-CD3 bispecific antibody CM336 can bind BCMA-positive B cells and plasma cells, and bind CD3-positive T cells at the same time to induce T cell activation and play the role of T-cell-dependent cellular cytotoxicity (TDCC). CM336 can eliminate BCMA-positive B cells and plasma cells through the above mechanism, directly reduce the production of pathogenic antibodies, and then achieve therapeutic effect. Due to the deep clearance of immune cells by bispecific antibodies, CM336 may achieve the effect of immune reset and cure autoimmune diseases such as ITP. The BCMA-CD3 bispecific antibody teclistamab has been reported to be used in the treatment of refractory systemic lupus erythematosus, systemic sclerosis, primary Sjogren's syndrome, idiopathic inflammatory myopathy, rheumatoid arthritis and other autoimmune diseases, with significant clinical effect and good safety.
Therefore, the investigators designed this clinical trial to evaluate the safety and efficacy of CM336 in the treatment of refractory ITP.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intervention(CM336) | Experimental | Drug: CM336 InjectionCM336 (Anticipated enrollment of 20 to 30 subjects) |
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 and above, male or female;
- Conform to the diagnostic criteria of persistent or chronic immune thrombocytopenia (ITP);
- Failure of previous glucocorticoid therapy;
- In the second-line treatment phase, eligible subjects must meet any of the following criteria: (1) Demonstrate no response to treatment with at least one thrombopoietin receptor agonist (including but not limited to recombinant human thrombopoietin [rhTPO], eltrombopag, hetrombopag, avatrombopag, or romiplostim); Fail to achieve sustained response (manifested as non-response, loss of response, or disease relapse) following anti-CD20 monoclonal antibody therapy (e.g., rituximab) or anti-CD38 monoclonal antibody therapy; (2) Exhibit no therapeutic response or experience disease relapse after splenectomy.
- The platelet count was <30×109/L within 48 hours before the first administration;
Key exclusion criteria (20 listed)
- Received any treatment of anti-BCMA antibody drug;
- Accompanied by autoimmune hemolytic anemia, or various secondary and hereditary thrombocytopenia;
- History of any thrombotic or embolic events in the 12 months prior to the first dose or accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc;
- Participated in any other study drug or exposure to other study drugs within 4 weeks or 5 half-lives before the first dose (whichever is longer);
- Use of anticoagulants or any drug with antiplatelet effects (such as aspirin) within 3 weeks before the first dose;
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 |
|---|---|---|
| CM336 Injectionsubcutaneous CM336 administration step-up dosing Dose and frequency of CM336 according to the protocol | Drug | Intervention(CM336) |
Primary outcome
To evaluate the efficacy after CM336 treatment at week 12
Time frame: 12 weeks
Proportion of subjects whose platelet counts ≥ 30×10^9/L and at least two times of baseline platelet count without bleeding at week 12
Safety of CM336
Time frame: 52 weeks
Incidence, severity, and relationship of treatment emergent adverse events after CM336 treatment
7 secondary outcomes
Proportion of subjects with a platelet count ≥ 50 × 10^9/L and ≥ 100 × 10^9/L at each visit
Time frame: 52 weeks
Duration from treatment initiation to platelet count ≥30×10^9/L and at least two times of baseline platelet count, platelet count ≥50×10^9/L, platelet count ≥100×10^9/L
Time frame: 52 weeks
Cumulative weeks of platelet ≥30×10^9/L and at least two times of baseline platelet count, platelet count ≥50×10^9/L, platelet count ≥100×10^9/L
Time frame: 52 weeks
Other efficacy evaluation
Time frame: 52 weeks
Proportion of subjects receiving emergency treatment
Time frame: 52 weeks
Changes in world health organization (WHO) bleeding scale
Time frame: 52 weeks
Measurements of platelet glycoprotein (GP) autoantibodies
Time frame: 52 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 13 Mar 2025.
- 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 to 36 months after study completion.
Related trials for Immune Thrombocytopenia (ITP)
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
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.
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 CM336 Injection in people with Immune Thrombocytopenia (ITP) 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 20 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: looking for participants now, as of the sponsor's last update on 13 Mar 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 Yunfei Chen, MD (+8618502220788, chenyunfei@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the efficacy after CM336 treatment at week 12 (time frame: 12 weeks). 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 13 Mar 2025.