Immune ThrombocytopeniaPhase 2NCT05562882
A Clinical Trial to Assess Safety and Efficacy of Daratumumab in the Treatment of Primary Immune Thrombocytopenia
A Phase 2 study testing Daratumumab Injection in people with Immune Thrombocytopenia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
A single-center, open-label, off-label use investigator-initiated clinical study 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Daratumumab Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteChina
- Study dates
- 10 Dec 2022to 31 Dec 2025
- Phase
- Phase 2
- 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
- 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 and/or TPO-RA.
- 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.
- February 16, 2023 After approval by the Ethics Committee on , subjects no longer require platelet glycoprotein autoantibodies positivity upon enrollment.
Who cannot join
- 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.
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- 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.
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 Investigator-initiated Clinical Trial to Assess Safety and Efficacy of Daratumumab in the Treatment of Relapsed/Refractory Primary Immune Thrombocytopenia
A single-center, open-label, off-label use investigator-initiated clinical study 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.
Read the 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.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Daratumumab 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.
Daratumumab InjectionDrug
Also called Darzalex
intravenous daratumumab administration
| Study group | Role | What they receive |
|---|---|---|
| Intervention | Experimental | Daratumumab once a week x 8 doses Receives: Drug: Daratumumab Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate of response after daratumumab treatment
The proportion of patients with 2 consecutive platelet counts of ≥ 50×109/L within 8 weeks
Measured: 8 weeks
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] of daratumumab
Incidence, severity, and relationship of treatment emergent adverse events after daratumumab treatment
Measured: 24 weeks
Secondary outcomes (10)
The proportion of patients with ≥2 consecutive platelet counts (separated by ≥7 days) of ≥30 × 109/L and a ≥2-fold increase from the baseline count within 8 weeks
The proportion of patients with ≥2 consecutive platelet counts (separated by ≥7 days) of ≥30 × 109/L and a ≥2-fold increase from the baseline count within 8 weeks.
Measured: 8 weeks
Time to first platelet count of ≥50 × 109/L without salvage therapy.
Time to first platelet count of ≥50 × 109/L without salvage therapy.
Measured: 24 weeks
Overall response rate at week 8 and week 24
Complete response (CR) : 2 or more consecutive monitored platelet counts ≥100 × 109/L without bleeding symptoms,with an interval of at least 7 days; Partial response (PR) : 2 or more consecutive monitored platelet counts ≥30 × 109/L, at least a double baseline count (separated by ≥7 days), and no bleeding symptoms. Overall response (OR) : CR or PR.
Measured: 24 weeks
Complete response rate at week 8 and week 24
Complete response (CR) : 2 or more consecutive monitored platelet counts ≥100 × 109/L without bleeding symptoms,with an interval of at least 7 days.
Measured: 24 weeks
Cumulative response duration of platelet count of ≥30 × 109/L and a platelet count doubling from the baseline within 24 weeks
Cumulative response duration of platelet count of ≥30 × 109/L and a platelet count doubling from the baseline within 24 weeks.
Measured: 24 weeks
Durable sustained platelet count response rate
Durable sustained platelet count response rate is defined as the proportion of subjects with platelet counts of ≥30 × 109/L for at least six of the eight visits between weeks 17 and 24 of the study.
Measured: 24 weeks
WHO bleeding score at baseline and at week 8 and 24 after treatment
WHO bleeding score at baseline and at week 8 and 24 after treatment. 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: 24 weeks
The proportion of subjects with concomitant medication reduction or discontinuation
The proportion of subjects with concomitant medication reduction or discontinuation within 24 weeks.
Measured: 24 weeks
The proportion of subjects receiving rescue medications
The proportion of subjects receiving rescue medications within 24 weeks.
Measured: 24 weeks
Changes in immunoglobulin levels
Changes in immunoglobulin levels before and after 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 posted3 Oct 2022
- Study start10 Dec 2022
- Primary completion31 Jan 2025
- Study completion31 Dec 2025 (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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, M.D., Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05562882
- Sponsor's protocol ID
- IIT2022039（1）
- Other IDs
- 2022-D-ITP
- Status last verified
- May 2024
- First submitted
- 28 Sep 2022
- Last updated
- 28 May 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Chen Y, Hua Z, Xu Y, Chen J, Wen Q, Sun T, Li H, Liu X, Fu R, Ju M, Xue F, Liu W, Dong H, Gu W, Dai X, Wang W, Chi Y, Pei X, Yang R, Zhang L. Daratumumab in patients with immune thrombocytopenia: a single-center, open-label, phase 2 trial. EClinicalMedicine. 2026 Mar 26;94:103849. doi: 10.1016/j.eclinm.2026.103849. eCollection 2026 Apr.
PubMed 41940000
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Investigator-initiated Clinical Trial to Assess Safety and Efficacy of Daratumumab in the Treatment of Relapsed/Refractory Primary Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT05562882
- Acronym / sponsor ID
- IIT2022039（1）
- Conditions
- Immune Thrombocytopenia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 10 Dec 2022 (actual)
- Primary completion
- 31 Jan 2025 (actual)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 3 Oct 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.
A single-center, open-label, off-label use investigator-initiated clinical study 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intervention | Experimental | Drug: Daratumumab InjectionDaratumumab 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)
- 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 and/or TPO-RA.
- 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.
Key exclusion criteria (9 listed)
- 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
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 |
|---|---|---|
| Daratumumab InjectionAlso: Darzalexintravenous daratumumab administration | Drug | Intervention |
Primary outcome
Evaluate of response after daratumumab treatment
Time frame: 8 weeks
The proportion of patients with 2 consecutive platelet counts of ≥ 50×109/L within 8 weeks
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] of daratumumab
Time frame: 24 weeks
Incidence, severity, and relationship of treatment emergent adverse events after daratumumab treatment
10 secondary outcomes
The proportion of patients with ≥2 consecutive platelet counts (separated by ≥7 days) of ≥30 × 109/L and a ≥2-fold increase from the baseline count within 8 weeks
Time frame: 8 weeks
Time to first platelet count of ≥50 × 109/L without salvage therapy.
Time frame: 24 weeks
Overall response rate at week 8 and week 24
Time frame: 24 weeks
Complete response rate at week 8 and week 24
Time frame: 24 weeks
Cumulative response duration of platelet count of ≥30 × 109/L and a platelet count doubling from the baseline within 24 weeks
Time frame: 24 weeks
Durable sustained platelet count response rate
Time frame: 24 weeks
WHO bleeding score at baseline and at week 8 and 24 after treatment
Time frame: 24 weeks
The proportion of subjects with concomitant medication reduction or discontinuation
Time frame: 24 weeks
The proportion of subjects receiving rescue medications
Time frame: 24 weeks
Changes in immunoglobulin levels
Time frame: 24 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2025.
- Derived from this study: PubMed 41940000Chen Y, Hua Z, Xu Y, Chen J, Wen Q, Sun T, Li H, Liu X, Fu R, Ju M, Xue F, Liu W, Dong H, Gu W, Dai X, Wang W, Chi Y, Pei X, Yang R, Zhang L. Daratumumab in patients with immune thrombocytopenia: a single-center, open-label, phase 2 trial. EClinicalMedicine. 2026 Mar 26;94:103849. doi: 10.1016/j.eclinm.2026.103849. eCollection 2026 Apr.
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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, M.D. principal investigator
Institute of Hematology & Blood Diseases Hospital, China
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 Daratumumab Injection in people with Immune Thrombocytopenia, 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 9 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 28 May 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?
Results are not available in the source record.
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?
Evaluate of response after daratumumab treatment (time frame: 8 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?
On the ClinicalTrials.gov record for NCT05562882, 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 28 May 2025.