Engineered immune cells take on rare blood disorders
NCT ID NCT07603557
First seen Jun 25, 2026 · Last updated Jul 10, 2026 · Updated 4 times
Summary
This phase 2 trial tests a personalized CAR T cell therapy called zolacabtagene autoleucel in 52 adults with chronic immune thrombocytopenia (ITP) or autoimmune hemolytic anemia (AIHA). The treatment uses the patient's own immune cells, modified to target and destroy faulty B cells that attack blood cells. The study aims to see if this approach can safely improve blood counts and reduce symptoms.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- zolacabtagene autoleucel (a CAR T cell therapy made from the patient's own immune cells)
- What this could lead to
- If it works, this could offer a new treatment option for people with chronic ITP or AIHA who have not responded to other therapies, potentially reducing the need for ongoing medication.
- What could go wrong
- This is an early phase 2 trial with only 52 participants, so results may not apply to everyone. CAR T cell therapy can cause serious side effects like cytokine release syndrome and neurological problems.
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
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 52 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Aug 2026
An estimate. Start dates often move.
- Expected to finish
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May 2030
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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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 Inclusion Criteria for ITP * Documented clinical diagnosis of chronic ITP (cITP) without other clinical manifestations of systemic autoimmune disease. * Has relapsed after or is intolerant to corticosteroids (with or without intravenous immunoglobulin (IVIG) or anti-Rh0(D) Ig) AND has failed, relapsed after, or is intolerant to therapies with ≥ 2 mechanisms of action, with at least one being immunosuppressive or immunomodulatory. Platelet count \< 30 × 109/L. For participants on thrombopoietin receptor agonist (TPO-RA): platelet count \< 50 × 109/L. Inclusion Criteria for AIHA * Documented clinical diagnosis of AIHA (including warm autoimmune hemolytic anemia (wAIHA), cold agglutinin disease (CAD), or mixed AIHA) without other clinical manifestations of systemic autoimmune disease. o wAIHA and mixed warm and cold AIHA: Failed, relapsed after, or is intolerant to at least 2 prior lines of treatment with 2 mechanisms of action (not including corticosteroids or IVIG), one of which is an anti-CD20 monoclonal antibody unless there is a documented contraindication. o CAD (all of the following must apply): Failed, relapsed after, or is intolerant to at least 2 prior lines of treatment with 2 mechanisms of action, one of which is an anti-CD20 monoclonal antibody with or without chemotherapy unless there is a documented contraindication. * Hb \<10 g/dL without red blood cell transfusion, or transfusion dependent * Documented hemolysis Exclusion Criteria Medical Conditions * ITP or AIHA associated with: Evans syndrome, other systemic autoimmune disease or single organ autoimmune disease requiring systemic immunosuppressive therapy, hepatitis C virus, HIV, drug induced (eg, non-steroidal anti-inflammatory drug (NSAIDS), trimethoprim/sulfamethoxazole (TMP-SMX), anticonvulsants), surgical procedures, or hematologic malignancies. * COVID-19 Vaccine-induced immune thrombotic thrombocytopenia * Prior history of solid organ malignancies, unless the participant has been free of the disease for ≥ 2 years. Laboratory Test Findings * Peripheral blood ANC \< 1.5 × 109/L or requiring G-CSF or GM-CSF support o ALT/AST: ITP: ALT/AST: \> 3 × ULN AIHA: ALT \> 3 ULN. AST up to 5 × ULN may be permitted. o Bilirubin: ITP: total bilirubin \> 1.5 × ULN AIHA: direct bilirubin \> 1.5 × ULN o International normalized ratio (INR) \> 1.5 × ULN Other protocol-defined inclusion/exclusion criteria may apply.
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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.
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The places running it
8 sites in 4 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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Local Institution - 101
Boston, Massachusetts, 02114, United States
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Local Institution - 102
Seattle, Washington, 98109, United States
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Local Institution - 103
Houston, Texas, 77030-2740, United States
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Local Institution - 201
Odense, DK-5000, Denmark
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Local Institution - 301
Magdeburg, Saxony-Anhalt, 39120, Germany
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Local Institution - 302
Erlangen, 91054, Germany
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Local Institution - 401
London, Greater London, W12 OHS, United Kingdom
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Local Institution - 402
Sheffield, S10 2SJ, United Kingdom
More trials for these conditions
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- Experimental mRNA CAR-T therapy targets autoimmune diseases in first human trial
- Brain scans reveal hidden thinking problems in anemia patients
- Experimental immune therapy for stubborn anemia shows early promise but trial stalls
- Experimental CAR-T therapy takes on stubborn blood disorder