RecruitingPhase 1/2

Phase I/II Study of Anti-CD38 Monoclonal Antibody in Refractory Severe Aplastic Anemia

China32 participantsStarted 2026-06-30

Plain-language 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. 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.

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: * 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. Exclusion Criteria: * 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 …

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

Incidence of adverse events

Timeframe: Within 12 weeks post treatment

Overall response rate

Timeframe: Within 12 weeks post treatment

Trial details

NCT IDNCT07714512

SponsorInstitute of Hematology & Blood Diseases Hospital, China

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-06-30

Contact for this trial

Lele Zhang, PhD

‭15811139278‬ zhanglele@ihcams.ac.cn

View on ClinicalTrials.gov More Aplastic Anemia trials