A Clinical Trial to Assess Safety and Efficacy of Daratumumab in the Treatment of Primary Immune Thrombocytopenia
Primary Purpose
Immune Thrombocytopenia
Status
Not yet recruiting
Phase
Early Phase 1
Locations
Study Type
Interventional
Intervention
Daratumumab Injection
Sponsored by
About this trial
This is an interventional treatment trial for Immune Thrombocytopenia
Eligibility Criteria
Inclusion Criteria:
- Male or female aged ≥18 years.
- Diagnosed with ITP that has persisted for ≥3 months and with a platelet count of <30 X 109/L measured within 2 days prior to inclusion.
- Failure to achieve response or relapse after corticosteroid therapy, and at least one second-line therapy including rituximab.
- If receiving emergency care for ITP, treatment should be stopped >2 weeks before first dose.
- A positive result to the ELISA test to detect antibody against GPIIb/IIIa or GPIIb/IIIa and GPIb/IX within 1 week prior to inclusion.
- With normal hepatic and renal functions.
- ECOG Performance Status ≤ 2.
Exclusion Criteria:
- Received any treatment of anti-CD38 antibody drug.
- Has been diagnosed with malignancy and/or liver failure, heart failure and renal failure.
- Known previous infection or seropositivity for HIV, Hepatitis B, Hepatitis C, Cytomegalovirus, EB virus, Syphilis.
- Any clinically overt hemorrhage.
- Has been diagnosed with cardiac disease, arrhythmia and/or severe or uncontrollable hypertension
- Known pulmonary embolism, thrombosis and/or atherosclerosis.
- Has been received allogeneic stem cell transplantation or organ transplantation.
- Patients with history of current or past psychiatric disease that might interfere with the ability to comply with the study protocol or give informed consent.
- Pregnancy or lactation.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Intervention
Arm Description
Daratumumab once a week x 8 doses
Outcomes
Primary Outcome Measures
Evaluate of response after daratumumab treatment
Response defined as platelet count ≥50 x109/L .8 weeks after the first dose.
Safety of daratumumab
Incidence of treatment emergent adverse events after daratumumab treatment
Secondary Outcome Measures
Full Information
NCT ID
NCT05562882
First Posted
September 28, 2022
Last Updated
September 28, 2022
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
1. Study Identification
Unique Protocol Identification Number
NCT05562882
Brief Title
A Clinical Trial to Assess Safety and Efficacy of Daratumumab in the Treatment of Primary Immune Thrombocytopenia
Official Title
A Investigator-initiated Clinical Trial to Assess Safety and Efficacy of Daratumumab in the Treatment of Relapsed/Refractory Primary Immune Thrombocytopenia
Study Type
Interventional
2. Study Status
Record Verification Date
September 2022
Overall Recruitment Status
Not yet recruiting
Study Start Date
October 15, 2022 (Anticipated)
Primary Completion Date
December 31, 2023 (Anticipated)
Study Completion Date
May 31, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Institute of Hematology & Blood Diseases Hospital, China
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
5. Study Description
Brief Summary
A single-center, open-label, off-label use investigator-initiated clinical study with safety run-in to explore the clinical activity and safety of daratumumab in adult ITP patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including rituximab and/or TPO-RA.
Detailed Description
Primary immune thrombocytopenia is an autoimmune disease associated with a reduced peripheral blood platelet count. The first-line treatment is corticosteroids. Splenectomy, rituximab, and thrombopoietin receptor agonists (TPO RAs, such as Etrapopar and Romistine) are commonly used as second-line therapy. However, many of the treatments used achieve few lasting remissions. About 20% - 30% of patients have inadequate or no response to first-line and second-line treatment, and would develop into recurrent/refractory (r/r) ITP.
A branch of pathogenesis for ITP has been revealed that plasma cells secrete pathogenic antibodies directed against platelet and red blood cell antigens. Antiplatelet specific plasma cells have been detected in the spleen of patients with rituximab refractory ITP. In those refractory cases, persistent autoreactive long-lived plasma cells in the bone marrow could explain treatment failure.
Daratumumab, an anti-CD38 monoclonal antibody developed to target tumoral plasma cells in multiple myeloma, was recently found to be effective in antibody-mediated diseases, such as autoimmune cytopenia following hematopoietic stem cell transplantation, systemic lupus and also ITP.
This study will evaluate the safety and biologic activity of Daratumumab in r/r primary ITP who fail to respond to at least one previous second-line therapy. The study will enroll approximately 20 participants. This trial will be conducted in China. All participants will be followed for at least 16 weeks after the 8 weeks of treatment.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Intervention
Arm Type
Experimental
Arm Description
Daratumumab once a week x 8 doses
Intervention Type
Drug
Intervention Name(s)
Daratumumab Injection
Other Intervention Name(s)
Darzalex
Intervention Description
intravenous daratumumab administration
Primary Outcome Measure Information:
Title
Evaluate of response after daratumumab treatment
Description
Response defined as platelet count ≥50 x109/L .8 weeks after the first dose.
Time Frame
9-24 weeks
Title
Safety of daratumumab
Description
Incidence of treatment emergent adverse events after daratumumab treatment
Time Frame
24 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female aged ≥18 years.
Diagnosed with ITP that has persisted for ≥3 months and with a platelet count of <30 X 109/L measured within 2 days prior to inclusion.
Failure to achieve response or relapse after corticosteroid therapy, and at least one second-line therapy including rituximab.
If receiving emergency care for ITP, treatment should be stopped >2 weeks before first dose.
A positive result to the ELISA test to detect antibody against GPIIb/IIIa or GPIIb/IIIa and GPIb/IX within 1 week prior to inclusion.
With normal hepatic and renal functions.
ECOG Performance Status ≤ 2.
Exclusion Criteria:
Received any treatment of anti-CD38 antibody drug.
Has been diagnosed with malignancy and/or liver failure, heart failure and renal failure.
Known previous infection or seropositivity for HIV, Hepatitis B, Hepatitis C, Cytomegalovirus, EB virus, Syphilis.
Any clinically overt hemorrhage.
Has been diagnosed with cardiac disease, arrhythmia and/or severe or uncontrollable hypertension
Known pulmonary embolism, thrombosis and/or atherosclerosis.
Has been received allogeneic stem cell transplantation or organ transplantation.
Patients with history of current or past psychiatric disease that might interfere with the ability to comply with the study protocol or give informed consent.
Pregnancy or lactation.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Lei Zhang, M.D.
Phone
+86 022-23909083
Email
zhanglei1@ihcams.ac.cn
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lei Zhang, M.D.
Organizational Affiliation
Institute of Hematology & Blood Diseases Hospital, China
Official's Role
Principal Investigator
12. IPD Sharing Statement
Learn more about this trial
A Clinical Trial to Assess Safety and Efficacy of Daratumumab in the Treatment of Primary Immune Thrombocytopenia
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