Evan SyndromePhase 22023-D-ESNCT06014775
Anti-CD38 Antibody Treating Evans Syndrome
A Phase 2 study testing Anti-CD38 antibody Injection in people with Evan Syndrome and Treatment, 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 with safety run-in to explore the clinical activity and safety of Anti-CD38 Antibody in adult ES patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Evan Syndrome, Treatment
- What is being tested?
- Anti-CD38 antibody Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2023to Aug 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.
- Prior to enrollment, a clinical diagnosis of primary Evans syndrome was made.
- Platelet count < 30×10^9/L or Hb < 100g/L or symptomatic anemia within 48 hours before the first administration of study drug;
- Failure to achieve response or relapse after corticosteroid therapy, and at least one second-line therapy or those who cannot chose other second-line therapy;
- If receiving emergency care for ES, treatment should be stopped >2 weeks before first dose.
- DAT positive (IgG+, with or without C3+).
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- The patient need to be in the state of active hemolysis.
- With normal hepatic and renal functions.
- ECOG performance status ≤2.
- Cardiac function: New York Heart Association functional class ≤2.
- For patients receiving maintenance treatment, corticosteroids must have a stable dose at least 2 weeks before the first administration, TPO receptor agonists and azathioprine, danazol, cyclosporin A, tacrolimus, sirolimus, etc. must be stopped at least 4 weeks before the first administration; The end of anti-CD20 antibody treatment was>6 months.The end of alkylating agent treatment was>2 months.
- Understand the study procedures and voluntarily sign the informed consent form in person.
Who cannot join
- Secondary Evans syndrome. Received any treatment of anti-CD38 antibody drug
- Uncontrollable primary diseases of important organs, such as malignant tumors, liver failure, heart failure, renal failure and other diseases;
- HIV positive;
- Accompanied by uncontrollable active infection, including hepatitis B, hepatitis C, cytomegalovirus, EB virus and syphilis positive;
- Accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.;
- At present, there are heart diseases, arrhythmias that need treatment or hypertension that researchers judge is poorly controlled;
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- Patients with thrombotic diseases such as pulmonary embolism, thrombosis and atherosclerosis;
- Those who have received allogeneic stem cell transplantation or organ transplantation in the past;
- Patients with mental disorders who cannot normally obtain informed consent and conduct trials and follow-up;
- Patients whose toxic symptoms caused by pre-trial treatment have not disappeared;
- Other serious diseases that may limit the subject's participation in this test (such as diabetes; Severe cardiac insufficiency; Myocardial obstruction or unstable arrhythmia or unstable angina pectoris in recent 6 months; Gastric ulcer, etc.);
- Patients with septicemia or other irregular severe bleeding;
- Patients taking antiplatelet drugs at the same time;
- Pregnant women, suspected pregnancies (positive pregnancy test for human chorionic gonadotropin in urine at screening) and lactating patients.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, 300020
Ting Sun, M.D, +86-22-23909009, sunting@ihcams.ac.cn
About this study
Official title: A Prospective, One-arm and Open Clinical Study to Assess Safety and Efficacy of Anti-CD38 Antibody in the Treatment of Evans Syndrome
A single-center, open-label, off-label use investigator-initiated clinical study with safety run-in to explore the clinical activity and safety of Anti-CD38 Antibody in adult ES patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including immunosuppressive agents, Anti-CD20 Antibody and/or TPO-RA, or those in whom no other second-line treatment options are suitable.
Read the detailed description
Evans' syndrome (ES) is defined as the concomitant or sequential association of warm auto-immune haemolytic anaemia (AIHA) with immune thrombocytopenia (ITP), and less frequently autoimmune neutropenia. ES is a rare situation that represents up to 7% of AIHA and around 2% of ITP. Due to the rarity of the disease, the treatment of ES is mostly extrapolated from what is recommended for isolated auto-immune cytopenia (AIC) and mostly relies on corticosteroids, rituximab, splenectomy, and supportive therapies.Despite continuous progress in the management of AIC and a gradual increase in ES survival, the mortality due to ES remains higher than the ones of isolated AIC, supporting the need for an improvement in ES management.
A branch of pathogenesis for ES 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.
Anti-CD38 antibody, such as Daratumumab, has been 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 ES.
This study will evaluate the safety and biologic activity of Anti-CD38 antibody in r/r primary ES who fail to respond to at least one previous second-line therapy or those who cannot chose suitable second-line therapy. The study will enroll approximately 10 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
- Evan Syndrome, Treatment
- Being tested
- Anti-CD38 antibody Injection
- Medical subject headings
- Evans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-CD38 antibody InjectionDrug
intravenous Anti-CD38 antibody administration
This study adopts a prospective, single arm, open design method. Ten subjects were enrolled in the study and were treated with Anti-CD38 antibody (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 Anti-CD38 antibody once a week for 8 weeks to observe the safety and efficacy during treatment.
The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of Anti-CD38 antibody after treatment.
| Study group | Role | What they receive |
|---|---|---|
| Intervention（Anti-CD38 antibody） | Experimental | 10 enrolled subjects : once a week x 8 doses Receives: Drug: Anti-CD38 antibody Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of overall efficacy response after Anti-CD38 antibody treatment within 8 weeks
Overall response rate defined as improvement in any cytopenias by at least one grade, without worsening any other cytopenias or stable disease at least once within 8 weeks after the first dose.
Measured: 8 weeks
Safety of Anti-CD38 antibody treatment
Incidence, severity, and relationship of treatment emergent adverse events after Anti-CD38 antibody treatment
Measured: 24 weeks
Secondary outcomes (15)
Evaluation of stable sustained response after Anti-CD38 antibody treatment at week 8
Stable sustained response rate defined as improvement in any cytopenias by at least one grade, without worsening any other cytopenias or stable disease in 3 consecutive accessible assessments at least 7 days apart 8 weeks after the first dose.
Measured: 8 weeks
Number of Participants With ES Response to Anti-CD38 antibody treatment
Complete response (CR) is complete resolution in all autoimmune cytopenias (neutropenia, anemia thrombocytopenia) maintained for more than two months, combined with an ability to wean off corticosteroids and/or other immunosuppressive medication. Partial response (PR) is improvement in any cytopenias by at least one grade, lasting more than two months, without worsening any other cytopenias or stable disease with the ability to wean corticosteroids and/or immunosuppressive medications by at least 50%. No response (NR) is no change in cytopenias with treatment, and the inability to wean corticosteroids or other immunosuppressive medications. Progressive disease (PD) refers to obtaining a CR or PR by the 3 month observation and relapsing or progressing by the 6 month observation, leading to cessation of study drug.
Measured: 24 weeks
Measurements of platelet count at each visit time point
Platelet count at each visit time point
Measured: 24 weeks
Measurements of Hb value at each visit time point
Hb value at each visit time point
Measured: 24 weeks
Measurements of hemolytic marker reticulocyte count at each visit time point
hemolytic marker reticulocyte count at each visit time point
Measured: 24 weeks
Measurements of hemolytic marker LDH at each visit time point
hemolytic marker LDH at each visit time point
Measured: 24 weeks
Measurements of hemolytic marker haptoglobin at each visit time point
hemolytic marker haptoglobin at each visit time point
Measured: 24 weeks
Measurements of hemolytic marker total bilirubin at each visit time point
hemolytic marker total bilirubin at each visit time point
Measured: 24 weeks
Emergency treatment
Percentage of subjects who received emergency treatment
Measured: 24 weeks
Duration of platelet response
The longest duration for which the subject sustained a platelet count ≥50×109/L and at least 2-fold from baseline at the meanwhile
Measured: 24 weeks
Duration of Hb response
The longest duration for which the subject sustained a Hb level ≥100 g/L, or a Hb level increased more than 20g/L than baseline
Measured: 24 weeks
Reduction of concomitant drug
Percentage of patients with reduced doses of corticosteroids and/or other concomitant immunosuppressive drugs at baseline by 8 weeks of treatment
Measured: 8 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 Anti-CD38 antibody 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: 24 weeks
Assessment of fatigue function in chronic disease treatment
The scores of Functional Assessment of Fatigue in Chronic Illness Therapy (FACIT-F) before and after Anti-CD38 antibody treatment
Measured: 24 weeks
Health status survey
The change of Health status Questionnaire (SF-36) score before and after Anti-CD38 antibody 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 posted28 Aug 2023
- Study start1 Dec 2023
- Primary completionAug 2025 (estimated)
- Study completionAug 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Evan SyndromeStudies 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
- 10 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
- NCT06014775
- Sponsor's protocol ID
- IIT2023036
- Acronym
- 2023-D-ES
- Status last verified
- Oct 2024
- First submitted
- 13 Jun 2023
- Last updated
- 21 Feb 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 36 months after study completion.
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, One-arm and Open Clinical Study to Assess Safety and Efficacy of Anti-CD38 Antibody in the Treatment of Evans Syndrome
- ClinicalTrials.gov ID
- NCT06014775
- Acronym / sponsor ID
- 2023-D-ES / IIT2023036
- Conditions
- Evan Syndrome, Treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 1 Dec 2023 (actual)
- Primary completion
- Aug 2025 (estimated)
- Study completion
- Aug 2025 (estimated)
- First posted
- 28 Aug 2023 (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 with safety run-in to explore the clinical activity and safety of Anti-CD38 Antibody in adult ES patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including immunosuppressive agents, Anti-CD20 Antibody and/or TPO-RA, or those in whom no other second-line treatment options are suitable.
Detailed description
Evans' syndrome (ES) is defined as the concomitant or sequential association of warm auto-immune haemolytic anaemia (AIHA) with immune thrombocytopenia (ITP), and less frequently autoimmune neutropenia. ES is a rare situation that represents up to 7% of AIHA and around 2% of ITP. Due to the rarity of the disease, the treatment of ES is mostly extrapolated from what is recommended for isolated auto-immune cytopenia (AIC) and mostly relies on corticosteroids, rituximab, splenectomy, and supportive therapies.Despite continuous progress in the management of AIC and a gradual increase in ES survival, the mortality due to ES remains higher than the ones of isolated AIC, supporting the need for an improvement in ES management.
A branch of pathogenesis for ES 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.
Anti-CD38 antibody, such as Daratumumab, has been 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 ES.
This study will evaluate the safety and biologic activity of Anti-CD38 antibody in r/r primary ES who fail to respond to at least one previous second-line therapy or those who cannot chose suitable second-line therapy. The study will enroll approximately 10 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（Anti-CD38 antibody） | Experimental | Drug: Anti-CD38 antibody Injection10 enrolled subjects : 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 (12 listed)
- Male or female aged ≥18 years.
- Prior to enrollment, a clinical diagnosis of primary Evans syndrome was made.
- Platelet count < 30×10^9/L or Hb < 100g/L or symptomatic anemia within 48 hours before the first administration of study drug;
- Failure to achieve response or relapse after corticosteroid therapy, and at least one second-line therapy or those who cannot chose other second-line therapy;
- If receiving emergency care for ES, treatment should be stopped >2 weeks before first dose.
Key exclusion criteria (14 listed)
- Secondary Evans syndrome. Received any treatment of anti-CD38 antibody drug
- Uncontrollable primary diseases of important organs, such as malignant tumors, liver failure, heart failure, renal failure and other diseases;
- HIV positive;
- Accompanied by uncontrollable active infection, including hepatitis B, hepatitis C, cytomegalovirus, EB virus and syphilis positive;
- Accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.;
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 |
|---|---|---|
| Anti-CD38 antibody Injectionintravenous Anti-CD38 antibody administration This study adopts a prospective, single arm, open design method. Ten subjects were enrolled in the study and were treated with Anti-CD38 antibody (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 Anti-CD38 antibody once a week for 8 weeks to observe the safety and efficacy during treatment. The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of Anti-CD38 antibody after treatment. | Drug | Intervention（Anti-CD38 antibody） |
Primary outcome
Evaluation of overall efficacy response after Anti-CD38 antibody treatment within 8 weeks
Time frame: 8 weeks
Overall response rate defined as improvement in any cytopenias by at least one grade, without worsening any other cytopenias or stable disease at least once within 8 weeks after the first dose.
Safety of Anti-CD38 antibody treatment
Time frame: 24 weeks
Incidence, severity, and relationship of treatment emergent adverse events after Anti-CD38 antibody treatment
15 secondary outcomes
Evaluation of stable sustained response after Anti-CD38 antibody treatment at week 8
Time frame: 8 weeks
Number of Participants With ES Response to Anti-CD38 antibody treatment
Time frame: 24 weeks
Measurements of platelet count at each visit time point
Time frame: 24 weeks
Measurements of Hb value at each visit time point
Time frame: 24 weeks
Measurements of hemolytic marker reticulocyte count at each visit time point
Time frame: 24 weeks
Measurements of hemolytic marker LDH at each visit time point
Time frame: 24 weeks
Measurements of hemolytic marker haptoglobin at each visit time point
Time frame: 24 weeks
Measurements of hemolytic marker total bilirubin at each visit time point
Time frame: 24 weeks
Emergency treatment
Time frame: 24 weeks
Duration of platelet response
Time frame: 24 weeks
Duration of Hb response
Time frame: 24 weeks
Reduction of concomitant drug
Time frame: 8 weeks
Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale
Time frame: 24 weeks
Assessment of fatigue function in chronic disease treatment
Time frame: 24 weeks
Health status survey
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 21 Feb 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 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, 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 Anti-CD38 antibody Injection in people with Evan Syndrome 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 12 inclusion and 14 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 21 Feb 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?
Contact Ting Sun, M.D (+86 022-23909009, sunting@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of overall efficacy response after Anti-CD38 antibody treatment within 8 weeks (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?
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 21 Feb 2025.