Aplastic AnemiaPhase 1/2NCT07714512
Phase I/II Study of Anti-CD38 Monoclonal Antibody in Refractory Severe Aplastic Anemia
A Phase 1/2 study testing Isatuximab in people with Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is a phase I/II clinical study in adult patients with refractory severe aplastic anemia (SAA). Eligible patients must meet the criteria for refractory SAA and have a platelet count (PLT) <30 × 10^9/L and/or hemoglobin (HGB) <90 g/L at enrollment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Isatuximab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 1 siteChina
- Study dates
- 30 Jun 2026to 31 Dec 2028
- Phase
- Phase 1/2
- 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
- Diagnosed with primary acquired aplastic anemia according to the 2024 British Society for Haematology guideline, Guidelines for the Diagnosis and Management of Adult Aplastic Anaemia, and the Chinese Guideline for the Diagnosis and Treatment of Aplastic Anemia (2022 edition) issued by the Hematology Branch of the Chinese Medical Association.
- Previously diagnosed with severe aplastic anemia (SAA) or very severe aplastic anemia (VSAA), with no response or relapse after receiving anti-thymocyte/anti-lymphocyte globulin (ATG/ALG) in combination with standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin receptor agonist (TPO-RA) therapy for at least 4 months.
- Hemoglobin <90 g/L or platelet count <30×10^9/L
- Unsuitable for or unwilling to undergo hematopoietic stem cell transplantation, with no better available treatment options.
- Age ≥18 years, regardless of gender.
- Eastern Cooperative Oncology Group (ECOG) performance status score ≤2
- Willing and able to comply with the requirements for this study and written informed consent.
Who cannot join
- Diagnosed with congenital bone marrow failure syndromes
- Bone marrow reticulin fibrosis grade ≥2
- Subjects with a paroxysmal nocturnal hemoglobinuria (PNH) clone ≥50% or active hemolysis
- Subjects with clonal cytogenetic abnormalities characteristic of myelodysplastic syndromes, except +8, del(20q), and -Y
- Active bacterial, viral, or fungal infection within 2 weeks before the first dose of the investigational drug, excluding common cold and onychomycosis, or any other serious infection. Any anti-infective treatment course for infection must have been completed at least 2 weeks before the first dose. Subjects with a history of HIV infection or positive HIV antibody during screening; positive Treponema pallidum antibody during screening; active tuberculosis, defined as chest imaging or other relevant examinations within 3 months before the first dose of the investigational drug or during screening suggesting active tuberculosis infection; or active hepatitis during screening, defined as hepatitis B surface antigen (HBsAg) positivity, or hepatitis B core antibody (HBcAb) positivity with hepatitis B virus (HBV) DNA ≥30 IU/mL, or hepatitis C virus (HCV) antibody positivity with HCV RNA positivity
- Active bleeding in the gastrointestinal tract, respiratory tract, central nervous system, or other sites
Show all 15Show fewer
- A history of any clinically significant disease that, in the investigator's opinion, would pose a safety risk to the subject if participating in the study, or would affect the evaluation of efficacy or safety if the disease/condition worsens during the study, including but not limited to: a. cardiovascular diseases, such as a history of acute myocardial infarction or unstable angina within the past year, severe arrhythmia such as frequent multifocal premature ventricular contractions, ventricular tachycardia, or ventricular fibrillation, congestive heart failure, arterial or venous thrombosis, or New York Heart Association (NYHA) class III-IV cardiac function; b. a history of psychiatric disorders, severe cerebrovascular disease, or cognitive sequelae
- Use of agents targeting B cells or plasma cells within 3 months before the first dose of the investigational drug or anticipated use during the clinical trial
- Treatment with anti-lymphocyte globulin or anti-thymocyte globulin within 6 months before the first dose of the investigational drug
- Treatment with tacrolimus, sirolimus, cyclophosphamide, anti-CD52 monoclonal antibody, or similar therapies within 4 weeks or 5 half-lives, whichever is shorter, before the first dose of the investigational drug
- Planned participation in another clinical trial, or prior exposure to another investigational product before the first dose, with an interval of less than 4 weeks or 5 half-lives of the drug, whichever is shorter
- Receipt of a live attenuated vaccine within 4 weeks before the first dose of the investigational drug or planned receipt during the study, or receipt of a COVID-19 vaccine within 7 days before dosing
- Prior treatment targeting CD38
- Women who are pregnant or breastfeeding, or who plan to become pregnant or breastfeed during the study
- Patients considered to be ineligible for the study by the investigator for reasons other than the above
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
Red Blood Cell Diseases Center and Regenerative Medicine Center
Tianjin, Tianjin Municipality, 301617
Lele Zhang, PhD, 15811139278, zhanglele@ihcams.ac.cn
About this study
Official title: A Phase I/II Study on the Safety and Efficacy of CD38 Monoclonal Antibody in the Treatment of Refractory Severe Aplastic Anemia
This is a phase I/II clinical study in adult patients with refractory severe aplastic anemia (SAA). Eligible patients must meet the criteria for refractory SAA and have a platelet count (PLT) <30 × 10^9/L and/or hemoglobin (HGB) <90 g/L at enrollment. If the phase I results demonstrate an acceptable safety profile and allow determination of the maximum tolerated dose (MTD), the phase II part will be initiated directly to evaluate the efficacy of isatuximab.
- Conditions
- Aplastic Anemia
- Being tested
- Isatuximab
- Keywords
- Isatuximab, Severe aplastic anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IsatuximabDrug
Phase I: Eligible subjects will receive isatuximab at 5 mg/kg per dose or 10 mg/kg per dose. The treatment period will last 6 weeks. Isatuximab will be administered by intravenous infusion once weekly (QW) for six consecutive doses.
Phase II: Eligible subjects will receive isatuximab by intravenous infusion once weekly for six consecutive doses. The specific dose will be the recommended phase II dose from the phase I part.
| Study group | Role | What they receive |
|---|---|---|
| Isatuximab | Experimental | The phase I part is the dose-escalation stage dose escalation will follow a standard 3+3 design.
The phase II part is a single-arm study using Simon's two-stage design. Receives: Drug: Isatuximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 6 to assess the adverse event
Measured: Within 12 weeks post treatment
Overall response rate
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
Measured: Within 12 weeks post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2026
- Study start30 Jun 2026
- Primary completion30 Jun 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Sanofi
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
- Investigators
- Jun Shi, PhD, Principal investigator, Institute of Hematology & Blood Diseases Hosptial, Chinese Academy of Medical Science and Peking Union Medical School
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07714512
- Sponsor's protocol ID
- IIT2026084
- Status last verified
- Sep 2026
- First submitted
- 15 Jul 2026
- Last updated
- 21 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study on the Safety and Efficacy of CD38 Monoclonal Antibody in the Treatment of Refractory Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT07714512
- Acronym / sponsor ID
- IIT2026084
- Conditions
- Aplastic Anemia
- Keywords
- Isatuximab, Severe aplastic anemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Sanofi
- Study start
- 30 Jun 2026 (actual)
- Primary completion
- 30 Jun 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 20 Jul 2026 (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.
This is a phase I/II clinical study in adult patients with refractory severe aplastic anemia (SAA). Eligible patients must meet the criteria for refractory SAA and have a platelet count (PLT) <30 × 10^9/L and/or hemoglobin (HGB) <90 g/L at enrollment. If the phase I results demonstrate an acceptable safety profile and allow determination of the maximum tolerated dose (MTD), the phase II part will be initiated directly to evaluate the efficacy of isatuximab.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Isatuximab | Experimental | Drug: IsatuximabThe phase I part is the dose-escalation stage dose escalation will follow a standard 3+3 design. The phase II part is a single-arm study using Simon's two-stage design. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosed with primary acquired aplastic anemia according to the 2024 British Society for Haematology guideline, Guidelines for the Diagnosis and Management of Adult Aplastic Anaemia, and the Chinese Guideline for the Diagnosis and Treatment of Aplastic Anemia (2022 edition) issued by the Hematology Branch of the Chinese Medical Association.
- Previously diagnosed with severe aplastic anemia (SAA) or very severe aplastic anemia (VSAA), with no response or relapse after receiving anti-thymocyte/anti-lymphocyte globulin (ATG/ALG) in combination with standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin receptor agonist (TPO-RA) therapy for at least 4 months.
- Hemoglobin <90 g/L or platelet count <30×10^9/L
- Unsuitable for or unwilling to undergo hematopoietic stem cell transplantation, with no better available treatment options.
- Age ≥18 years, regardless of gender.
Key exclusion criteria (15 listed)
- Diagnosed with congenital bone marrow failure syndromes
- Bone marrow reticulin fibrosis grade ≥2
- Subjects with a paroxysmal nocturnal hemoglobinuria (PNH) clone ≥50% or active hemolysis
- Subjects with clonal cytogenetic abnormalities characteristic of myelodysplastic syndromes, except +8, del(20q), and -Y
- Active bacterial, viral, or fungal infection within 2 weeks before the first dose of the investigational drug, excluding common cold and onychomycosis, or any other serious infection. Any anti-infective treatment course for infection must have been completed at least 2 weeks before the first dose. Subjects with a history of HIV infection or positive HIV antibody during screening; positive Treponema pallidum antibody during screening; active tuberculosis, defined as chest imaging or other relevant examinations within 3 months before the first dose of the investigational drug or during screening suggesting active tuberculosis infection; or active hepatitis during screening, defined as hepatitis B surface antigen (HBsAg) positivity, or hepatitis B core antibody (HBcAb) positivity with hepatitis B virus (HBV) DNA ≥30 IU/mL, or hepatitis C virus (HCV) antibody positivity with HCV RNA positivity
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 |
|---|---|---|
| IsatuximabPhase I: Eligible subjects will receive isatuximab at 5 mg/kg per dose or 10 mg/kg per dose. The treatment period will last 6 weeks. Isatuximab will be administered by intravenous infusion once weekly (QW) for six consecutive doses. Phase II: Eligible subjects will receive isatuximab by intravenous infusion once weekly for six consecutive doses. The specific dose will be the recommended phase II dose from the phase I part. | Drug | Isatuximab |
Primary outcome
Incidence of adverse events
Time frame: Within 12 weeks post treatment
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 6 to assess the adverse event
Overall response rate
Time frame: Within 12 weeks post treatment
Percentage of patients with hematological response, including complete response (CR) or partial response (PR). Hematological response is evaluated by hemoglobin (Hb), platelet count (PLT) and absolute neutrophil count (ANC).
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 21 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Aplastic Anemia
RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
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.
Jun Shi, PhD principal investigator
Institute of Hematology & Blood Diseases Hosptial, Chinese Academy of Medical Science and Peking Union Medical School
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Isatuximab in people with Aplastic Anemia, 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 7 inclusion and 15 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 21 Sep 2026. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Lele Zhang, PhD (15811139278, zhanglele@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: Within 12 weeks post treatment). 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 Sep 2026.