An Open, Exploratory Clinical Study of CM336 in the Treatment of Immune Thrombocytopenia

NCT06799611 | Recruiting Phase 2 DMC

• 1 other identifier: IIT2024050
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the efficacy and safety of CM336 in the treatment of refractory adult primary immune thrombocytopenia

Conditions

Immune Thrombocytopenia (ITP); Treatment

Outcome Measures

Primary Outcomes (2)

• 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

  12 weeks

• Safety of CM336

  Incidence, severity, and relationship of treatment emergent adverse events after CM336 treatment

  52 weeks

Secondary Outcomes (7)

• Proportion of subjects with a platelet count ≥ 50 × 10^9/L and ≥ 100 × 10^9/L at each visit

  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

  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

  52 weeks

• Other efficacy evaluation

  52 weeks

• Proportion of subjects receiving emergency treatment

  52 weeks

Study Arms (1)

Intervention(CM336)

EXPERIMENTAL

CM336 (Anticipated enrollment of 20 to 30 subjects)

Drug: CM336 Injection

Interventions

CM336 Injection DRUG

subcutaneous CM336 administration step-up dosing Dose and frequency of CM336 according to the protocol

Intervention(CM336)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• 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;
• 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;

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Lei Zhang, MD

  Chinese Academy of Medical Science and Blood Disease Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yunfei Chen, MD

CONTACT

+8618502220788; chenyunfei@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2025
• First Posted: January 29, 2025
• Study Start: March 3, 2025
• Primary Completion: January 31, 2026
• Study Completion (Estimated): January 31, 2027
• Last Updated: March 13, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: 12 months to 36 months after study completion
• Access Criteria: Upon request to PI

Locations

CN (1)