The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia (NCT07287228) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
China37 participantsStarted 2025-12-10
Plain-language 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. 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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:
.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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Overall response rate
Timeframe: Within 12 weeks after treatment
Trial details
NCT IDNCT07287228
SponsorInstitute of Hematology & Blood Diseases Hospital, China
. 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.
Exclusion criteria
. 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.
. 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:
.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.