Aplastic AnemiaPhase 1/2DARA-AA-001NCT07287228
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
A Phase 1/2 study testing Daratumumab in people with Aplastic Anemia and Refractory, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This study consists of two phases, phase Ib and phase II. The primary objective of phase Ib is to evaluate the safety and tolerability of multiple doses of daratumumab in patients with refractory aplastic anemia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aplastic Anemia, Refractory, Relapse
- What is being tested?
- Daratumumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 1 siteChina
- Study dates
- 17 Dec 2025to 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
- Clearly diagnosed with primary acquired aplastic anemia according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition).
- Refractory aplastic anemia meeting one of the following criteria:
- 2.1 Patients previously diagnosed with severe aplastic anemia (SAA) # or transfusion-dependent non-severe aplastic anemia (TD-NSAA) \&: have received antithymocyte/antilymphocyte globulin (ATG/ALG) combined with standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin (TPO) receptor agonist therapy for at least 4 months, but showed no response or relapsed; 2.2 Patients previously diagnosed with non-transfusion-dependent non-severe aplastic anemia (NTD-NSAA): have received standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin (TPO) receptor agonist therapy for at least 4 months, but showed no response or relapsed.
- Hemoglobin <90 g/L or platelet count <30×10⁹/L.
- Not suitable for or unwilling to undergo hematopoietic stem cell transplantation; no other better treatment options available.
- Age ≥18 years, no sex limitation.
- Eastern Cooperative Oncology Group (ECOG) performance status score ≤2 (see Appendix 2).
- Voluntarily signed the informed consent form, understands the nature, purpose, and procedures of the trial, and is willing to comply with the trial requirements.
Who cannot join
- Patients with congenital bone marrow failure syndromes.
- Patients with bone marrow reticulin staining ≥ Grade 2.
- Patients with paroxysmal nocturnal hemoglobinuria (PNH) clone ≥50% or with active hemolysis.
- Patients with clonal chromosomal abnormalities characteristic of myelodysplastic syndrome (MDS) (except +8, 20q-, and -Y).
- Presence of active bacterial, viral, or fungal infection within 2 weeks prior to the first administration of the investigational drug (excluding common cold and onychomycosis), or any other severe infection. Any anti-infective therapy for infections must be completed at least 2 weeks before the first dose. History of HIV infection or HIV antibody positive during screening; positive treponema pallidum antibody during screening; active pulmonary tuberculosis (evidenced by chest imaging or other relevant examinations within 3 months prior to first administration or during screening indicating active TB infection); active hepatitis during screening (positive hepatitis B surface antigen [HBsAg], or positive hepatitis B core antibody [HBcAb] with HBV DNA ≥30 IU/mL, or positive hepatitis C antibody [HCV Ab] with positive HCV RNA).
- Presence of active bleeding in the gastrointestinal tract, respiratory tract, central nervous system, or other sites.
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- History of significant clinical diseases that, in the investigator's judgment, would pose a safety risk for the subject's participation or affect the evaluation of efficacy or safety if the disease/condition worsens during the study. Examples include:
- 7.1 Cardiovascular disease: acute myocardial infarction or unstable angina within the past year, severe arrhythmias (such as frequent multifocal PVCs, ventricular tachycardia, ventricular fibrillation, etc.), congestive heart failure, arterial or venous thrombosis, NYHA Class III-IV cardiac function.
- 7.2 History of psychiatric disorders or severe cerebrovascular disease or cognitive sequelae.
- Use of B-cell or plasma-cell targeting agents within 3 months prior to the first administration of the investigational drug or expected use during the study period.
- Receipt of antithymocyte globulin or antilymphocyte globulin within 6 months prior to the first administration of the investigational drug.
- Treatment with tacrolimus, sirolimus, cyclophosphamide, anti-CD52 monoclonal antibodies, or similar agents within 4 weeks or 5 half-lives (whichever is shorter) prior to the first administration of the investigational drug.
- Planned participation in another clinical trial or prior exposure to another investigational product within less than 4 weeks or 5 half-lives (whichever is shorter) before the first dose.
- Vaccination with live attenuated vaccines within 4 weeks prior to the first administration of the investigational drug or planned during the study period, or COVID-19 vaccination within 7 days prior to first administration.
- Prior exposure to CD38-targeted therapy.
- Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study; female subjects of childbearing potential and male subjects with partners of childbearing potential who are unwilling to use highly effective contraception (see Appendix 3 for specific methods) throughout the study (from signing the ICF to 6 months after the last dose of study drug), or who plan to donate eggs or sperm during the study.
- Any other conditions deemed unsuitable for participation in this study by the investigator.
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
Institute of Hematology & Blood Diseases Hosptial, Chinese Academy of Medical Science and Peking Union Medical School
Tianjin, Tianjin Municipality, 300020
Lele Zhang, M.D., Ph.D., 86-15811139278, zhanglele@ihcams.ac.cn
About this study
Official title: An Ib/II Phase Clinical Study Evaluating the Safety and Preliminary Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
This study consists of two phases, phase Ib and phase II. The primary objective of phase Ib is to evaluate the safety and tolerability of multiple doses of daratumumab in patients with refractory aplastic anemia. The primary objective of phase II is to preliminarily assess the efficacy of multiple doses of daratumumab in patients with refractory aplastic anemia. Secondary objectives include evaluation of other efficacy endpoints, safety (phase II), and pharmacodynamic characteristics of multiple doses of daratumumab in these patients. The exploratory objective is to assess biological functional changes in peripheral blood and bone marrow before and after daratumumab treatment.
Read the detailed description
This study is a seamless phase Ib/II design. The target population comprises adult patients with refractory aplastic anemia (AA) who meet the criteria for refractory AA at enrollment, with platelet counts (PLT) < 30×10⁹/L or hemoglobin (HGB) < 90 g/L. A Safety Review Committee (SRC), led by the principal investigator, will be established. If the phase Ib results demonstrate sufficient safety and allow determination of the maximum tolerated dose (MTD), the phase II study will proceed directly.
The phase Ib study serves as the dose-escalation stage, with three dose cohorts: low-dose (8 mg/kg), medium-dose (12 mg/kg), and high-dose (16 mg/kg), administered once weekly for six consecutive doses. Dose escalation follows the "3+3" design.
- Conditions
- Aplastic Anemia, Refractory, Relapse
- Being tested
- Daratumumab
- Keywords
- Aplastic anemia, Daratumumab
- Medical subject headings
- Anemia, Aplastic, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DaratumumabDrug
In Phase I, eligible subjects who meet the inclusion criteria will receive daratumumab at doses of 8 mg/kg, 12 mg/kg, or 16 mg/kg. The treatment period will last 6 weeks, with intravenous infusion administered once weekly (QW) for a total of 6 consecutive doses.
During the conduct of the clinical trial, based on the clinical trial data obtained (safety, MTD, etc.) and after discussion by the SRC, the RP2D for Phase II will be determined. In Phase II, eligible subjects who meet the inclusion criteria will receive intravenous daratumumab once weekly (specific dose based on the RP2D determined from Phase Ib clinical trial data after SRC discussion) for a total of 6 consecutive doses.
| Study group | Role | What they receive |
|---|---|---|
| After enrollment, patients will receive daratumumab treatment. | Experimental | This study is designed as a seamless Phase Ib/II trial. The target population is adult patients with refractory aplastic anemia (AA), who must meet the criteria for refractory AA at enrollment. After enrollment, patients will receive daratumumab treatment. Receives: Drug: Daratumumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 after treatment
Secondary outcomes (1)
Incidence of adverse events
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Measured: Within 12 weeks after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2025
- Study start17 Dec 2025
- Primary completion31 Dec 2027 (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
- Model description
- The target population is adult patients with refractory aplastic anemia (AA).
- Masking
- None (open label)
- Enrolment
- 37 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jun Shi, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07287228
- Sponsor's protocol ID
- IIT2025084
- Acronym
- DARA-AA-001
- Status last verified
- Nov 2026
- First submitted
- 25 Sep 2025
- Last updated
- 6 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Ib/II Phase Clinical Study Evaluating the Safety and Preliminary Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
- ClinicalTrials.gov ID
- NCT07287228
- Acronym / sponsor ID
- DARA-AA-001 / IIT2025084
- Conditions
- Aplastic Anemia, Refractory, Relapse
- Keywords
- Aplastic anemia, Daratumumab
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 37 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 17 Dec 2025 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 17 Dec 2025 (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 study consists of two phases, phase Ib and phase II. The primary objective of phase Ib is to evaluate the safety and tolerability of multiple doses of daratumumab in patients with refractory aplastic anemia. The primary objective of phase II is to preliminarily assess the efficacy of multiple doses of daratumumab in patients with refractory aplastic anemia. Secondary objectives include evaluation of other efficacy endpoints, safety (phase II), and pharmacodynamic characteristics of multiple doses of daratumumab in these patients. The exploratory objective is to assess biological functional changes in peripheral blood and bone marrow before and after daratumumab treatment.
Detailed description
This study is a seamless phase Ib/II design. The target population comprises adult patients with refractory aplastic anemia (AA) who meet the criteria for refractory AA at enrollment, with platelet counts (PLT) < 30×10⁹/L or hemoglobin (HGB) < 90 g/L. A Safety Review Committee (SRC), led by the principal investigator, will be established. If the phase Ib results demonstrate sufficient safety and allow determination of the maximum tolerated dose (MTD), the phase II study will proceed directly.
The phase Ib study serves as the dose-escalation stage, with three dose cohorts: low-dose (8 mg/kg), medium-dose (12 mg/kg), and high-dose (16 mg/kg), administered once weekly for six consecutive doses. Dose escalation follows the "3+3" design.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The target population is adult patients with refractory aplastic anemia (AA).
| Arm | Type | Receives |
|---|---|---|
| After enrollment, patients will receive daratumumab treatment. | Experimental | Drug: DaratumumabThis study is designed as a seamless Phase Ib/II trial. The target population is adult patients with refractory aplastic anemia (AA), who must meet the criteria for refractory AA at enrollment. After enrollment, patients will receive daratumumab treatment. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Clearly diagnosed with primary acquired aplastic anemia according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition).
- Refractory aplastic anemia meeting one of the following criteria:
- 2.1 Patients previously diagnosed with severe aplastic anemia (SAA) # or transfusion-dependent non-severe aplastic anemia (TD-NSAA) \&: have received antithymocyte/antilymphocyte globulin (ATG/ALG) combined with standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin (TPO) receptor agonist therapy for at least 4 months, but showed no response or relapsed; 2.2 Patients previously diagnosed with non-transfusion-dependent non-severe aplastic anemia (NTD-NSAA): have received standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin (TPO) receptor agonist therapy for at least 4 months, but showed no response or relapsed.
- Hemoglobin <90 g/L or platelet count <30×10⁹/L.
- Not suitable for or unwilling to undergo hematopoietic stem cell transplantation; no other better treatment options available.
Key exclusion criteria (17 listed)
- Patients with congenital bone marrow failure syndromes.
- Patients with bone marrow reticulin staining ≥ Grade 2.
- Patients with paroxysmal nocturnal hemoglobinuria (PNH) clone ≥50% or with active hemolysis.
- Patients with clonal chromosomal abnormalities characteristic of myelodysplastic syndrome (MDS) (except +8, 20q-, and -Y).
- Presence of active bacterial, viral, or fungal infection within 2 weeks prior to the first administration of the investigational drug (excluding common cold and onychomycosis), or any other severe infection. Any anti-infective therapy for infections must be completed at least 2 weeks before the first dose. History of HIV infection or HIV antibody positive during screening; positive treponema pallidum antibody during screening; active pulmonary tuberculosis (evidenced by chest imaging or other relevant examinations within 3 months prior to first administration or during screening indicating active TB infection); active hepatitis during screening (positive hepatitis B surface antigen [HBsAg], or positive hepatitis B core antibody [HBcAb] with HBV DNA ≥30 IU/mL, or positive hepatitis C antibody [HCV Ab] with positive HCV RNA).
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 |
|---|---|---|
| DaratumumabIn Phase I, eligible subjects who meet the inclusion criteria will receive daratumumab at doses of 8 mg/kg, 12 mg/kg, or 16 mg/kg. The treatment period will last 6 weeks, with intravenous infusion administered once weekly (QW) for a total of 6 consecutive doses. During the conduct of the clinical trial, based on the clinical trial data obtained (safety, MTD, etc.) and after discussion by the SRC, the RP2D for Phase II will be determined. In Phase II, eligible subjects who meet the inclusion criteria will receive intravenous daratumumab once weekly (specific dose based on the RP2D determined from Phase Ib clinical trial data after SRC discussion) for a total of 6 consecutive doses. | Drug | After enrollment, patients will receive daratumumab treatment. |
Primary outcome
Overall response rate
Time frame: Within 12 weeks after 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).
1 secondary outcome
Incidence of adverse events
Time frame: Within 12 weeks after treatment
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 6 Nov 2026.
- Individual participant dataSharing planned: Undecided
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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.
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 Daratumumab in people with Aplastic Anemia and Refractory, 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 17 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 6 Nov 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, Dr (02223608328, zhanglele@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: Within 12 weeks after treatment). 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 6 Nov 2026.