Could a cancer antibody reboot blood production in aplastic anemia?
NCT ID NCT07714512
First seen Jul 20, 2026 · Last updated Jul 21, 2026 · Updated 1 time
Summary
This trial tests isatuximab, an antibody that targets CD38, in adults with refractory severe aplastic anemia—a condition where the bone marrow fails to produce enough blood cells. Participants receive weekly infusions for six weeks. The study aims to see if the drug is safe and can improve blood cell counts in patients who have not responded to standard treatments.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- a CD38 monoclonal antibody called isatuximab
- What this could lead to
- If successful, this could offer a new treatment option for patients with severe aplastic anemia who do not respond to current therapies.
- What could go wrong
- This is an early-phase trial with only 32 participants, so results may not generalize. The drug may cause side effects or fail to improve blood counts.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
About 32 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Jun 2026
- Expected to finish
-
Dec 2028
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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 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 * 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
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Aplastic anemia are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
Red Blood Cell Diseases Center and Regenerative Medicine Center
RECRUITINGTianjin, Tianjin Municipality, 301617, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cells tested as a cure for severe aplastic anemia
- Can a pill revive bone marrow in aplastic anemia?
- Can a drug revive blood cell production in children with aplastic anemia?
- Could a platelet-boosting drug shorten recovery after stem cell transplants?
- A common antioxidant may help platelets recover faster after stem cell transplant
- Triple therapy aims to overcome antibody barrier in Half-Matched stem cell transplants