CytopeniaPhase 1ACE-CAR TNCT07790575
Autoimmune Cytopenia Elimination by Chimeric Antigen Receptor T-cell Therapy
A Phase 1 study testing AZD0120 and Bendamustin in people with Cytopenia, run by Saurabh Dahiya at 1 site in United States.
Trial summary
This is a phase 1 study to evaluate the safety and tolerability of AZD0120 in patients with relapsed or refractory immune mediated cytopenia (primary chronic Immune Thrombocytopenia (ITP) or autoimmune hemolytic anemia (wAIHA)). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Cytopenia
- What is being tested?
- AZD0120 and Bendamustin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2026to Jul 2041
- Phase
- Phase 1
- 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of primary chronic immune thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA) according to the published consensus criteria
- a. 2 or more prior treatments for ITP wherein the following conditions are required to define prior treatments:
- 1st line treatment includes steroids (dexamethasone, prednisone or equivalent), IVIG, and/or Anti-Rhd Ig. These medications may be administered in combination or in series.
- 2nd line treatment includes the use of thrombopoietic agents, and anti-CD20 mAbs.
- These medications may be given in combination or in series.
- A failure to respond to splenectomy is considered an additional line of therapy.
Show all 33Show fewer
- Thus, in order to be study eligible, a potential participant must have refractory chronic ITP and have received high dose steroids, IVIG, TPO agonists, and anti-CD20mAbs, with evidence of treatment failure, resistance, or relapse following steroids, TPO agonists, and anti-CD20 mAbs. Participants must have failed both steroids and TPO-RA to be eligible. b. 2 or more prior treatments for AIHA wherein the following conditions are required to define prior treatments:
- 1st line treatment includes high dose-steroids (dexamethasone, prednisone or equivalent), IVIG, and/or Anti-Rhd Ig. These medications may be administered in combination or in series.
- 2nd line treatment includes the use of corticosteroids, anti-CD20 mAbs. These medications may be given in combination or in series.
- A failure to respond to splenectomy is considered an additional line of therapy.
- Thus, in order to be study eligible, a potential participant must have refractory AIHA and have received high dose steroids, IVIG, and anti-CD20mAbs, with evidence of treatment failure, resistance, or relapse following steroids and anti-CD20 mAbs.
- Participants must have failed both steroids and anti-CD20 mAbs (i.e., rituximab) to be eligible.
- Organ and Marrow Function
- ANC ≥ 1000/uL.
- Absolute lymphocyte count ≥ 600/uL.
- Adequate renal, hepatic, pulmonary and cardiac function defined as:
- Creatinine ≤ 2 mg/dL or creatinine clearance (as estimated by Cockcroft Gault Equation) ≥ 50 mL/min.
- Serum ALT/AST <2.5 times ULN.
- Total bilirubin ≤ 1.5 mg/dl, except in subjects with Gilbert's syndrome.
- Cardiac ejection fraction ≥ 50%, no evidence of physiologically significant pericardial effusion as determined by an ECHO, and no clinically significant ECG findings.
- Baseline oxygen saturation > 92% on room air.
- Testing for i) HBc Ab ii) HCV Ab iii) Hep B surf. AG iv) HIV 1\&2 Ab v) Syphilis Screen vi) HTLV Ab I \& II vii) NAT MPX (nucleic acid test multiplex) for HIV, HCV, HBV viii) Herpes Simplex Virus 1 \& 2 IgG panel ix) VZ IgG x) CMV Total Ab Must be seronegative for HIV-1 RNA PCR; HIV 1 and HIV 2 Ab (antibody); HTLV-1 and HTLV-2 Ab; PCR- or sAg negative (surface antigen for hepatitis B); negative for the Treponema pallidum antibody Syphilis screen; and negative for HIV-1 and hepatitis C by nucleic acid testing (NAT) within 40 days of apheresis procedures.
- 6. Females of childbearing potential have a negative serum or urine pregnancy test because of the potentially dangerous/unknown effects on the fetus. Women not of child-bearing potential are defined as females who are either permanently sterilized (hysterectomy, bilateral oophorectomy, or bilateral salpingectomy), or who are post-menopausal. Females will be considered post-menopausal if they have been amenorrhoeic for 12 months prior to the planned date of assignment to treatment arm without an alternative medical cause. A high FSH level in post-menopausal range may be used to confirm a post-menopausal state in women not using hormonal contraception or hormonal replacement therapy. A post-menopausal state is confirmed by 2 consecutive high FSH values (at least 4 weeks apart) in the post-menopausal range.
- 7. Contraception: Subjects of child-bearing or child-fathering potential must be willing to practice effective birth control from the time of enrollment on this study. Contraceptive use by participants or participant partners should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. For AZD0120: effective contraception must be used for at least 12 months after receiving AZD0120 infusion or CAR T-cell DNA is no longer detectable by PCR, whichever occurs later. For participants who receive only bendamustine for some reason but not AZD0120: males must use contraception for 3 months after last dose; females must use contraception for 6 months after last dose.
- Ability to understand and the willingness to sign a written informed consent document. Patients must have signed informed consent to participate in the trial.
- Adequate vital sign criterion with acceptable numerical ranges of:
- Systolic Blood Pressure (mmHg) ≥ 100 and ≤ 150
- Diastolic Blood pressure (mmHg) ≥ 60 and ≤ 90
- To ensure subject safety and stability, any subject who is noted to have a BP > 150/90 mm Hg should be stable on anti-hypertensive medications with repeated BP ≤150/90 for at least one month prior to enrollment in the study
- Baseline Heart Rate ≥ 60 and ≤ 100 bpm
- Oral Temperature ≤ 37.7 C/afebrile
- Respiratory rate ≥ 12 and ≤ 20bpm
- Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 at screening to be eligible for enrollment.
Who cannot join
- For ITP - ITP Bleeding Scale Grade 3 or higher at the screening visit (with the exception of menorrhagia controlled with hormonal therapy +/- tranexamic acid)
- For ITP - Hgb < 8 g/dL (NOTE: Exclusion does not apply to Evans Syndrome).
- For wAIHA - subjects with signs of hemolysis not attributable to wAIHA are excluded.
- For wAIHA - platelet count <100,000/uL (NOTE: ITP subjects may be below this threshold if due to active ITP. Exclusion does not apply to Evans Syndrome).
- For ITP - Secondary ITP (including but not limited to associated with a lymphoproliferative disorder, positive screening tests for HIV, HCV, H. pylori, Common Variable Immunodeficiency)
- For wAIHA - Secondary wAIHA (including but not limited to associated with lymphoproliferative disorder, positive screening tests for HIV, HCV, Common Variable Immunodeficiency)
Show all 27Show fewer
- Prior treatment with any investigational agent within 3 months, or 5 half-lives, whichever is longer. Agents authorized by the FDA for prevention or treatment of SARS-CoV-2 are not considered investigational.
- Subjects who received immunosuppressants (i.e., mycophenolate mofetil, azathioprine, methotrexate, calcineurin inhibitors, tyrosine kinase inhibitors) within one month prior to the CAR T infusion are excluded.
- History of sickle cell anemia or other hemoglobinopathy (hemoglobinopathy trait is not an exclusion criteria).
- Coagulation abnormalities defined by: INR > 1.6, PT > 15 seconds, PTT > 45 seconds to the exclusion criteria. Patients with positive antiphospholipid antibodies, including anti-cardiolipin, or lupus anticoagulant.
- Presence of fungal, bacterial, viral, or other infection that is not controlled and/ or requiring hospitalization or treatment with IV antimicrobials within 4 weeks of screening. Simple UTI and uncomplicated bacterial pharyngitis are permitted if responding to active treatment.
- Psychiatric disorder(s) or psychosocial circumstance(s) which in the opinion of the Stanford Transplant team caring for this potential patient would place the patient at an unacceptable risk. Participant has significant neurological or psychiatric condition (active or history of) including but not limited to severe brain injury, dementia, cerebellar disease, Parkinson's disease, clinical evidence of dementia or altered mental status, or stroke, intracranial hemorrhage, or seizure within 6 months before signing ICF. Participants with existing stable, mild neurological conditions that have not progressed in the last 6 months (e.g., essential tremor) may be considered eligible at the Investigator's discretion, following appropriate neurological consultation.
- Presence or history of liver cirrhosis.
- Presence or history of MDS; presence of bone marrow failure and malignant bone marrow disorders confirmed by bone marrow examination (aspirate and/or biopsy).
- Patients without a bone marrow aspirate and/or biopsy performed within 12 months prior to screening to confirm the primary cause of the disease.
- History of malignancy other than non-melanoma skin cancer or carcinoma in situ (e.g., cervix, bladder, breast) or localized prostate cancer requiring no treatment unless disease free for at least 2 years.
- Active infection with HIV, hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive) as the immunosuppression contained in this study may pose unacceptable risk. A prior history of hepatitis B or hepatitis C is permitted providing the viral load is undetectable per quantitative PCR and/or nucleic acid testing. Hepatitis B surface antibody following hepatitis B immunization is not considered to be evidence of past infection.
- History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease (uncontrolled congestive heart failure) within 3 months of enrollment. Subjects with stable cardiac disease fulfilling inclusion criteria are allowed.
- History of Crohn's, rheumatoid arthritis, systemic lupus that required continued systemic immunosuppression/systemic disease modifying agents within the 2 years prior to trial enrollment.
- A primary immune deficiency disease.
- In the investigator's judgment, the subject is unlikely to complete protocol-required study visits or procedures, including follow-up visits, or comply with the study requirements for participation.
- History of severe immediate hypersensitivity reaction to any of the agents used in this study. This includes contraindications or life-threatening allergies, hypersensitivity, or intolerance to AZD0120 or its excipients, including dimethyl sulfoxide; bendamustine; or tocilizumab.
- Any medical condition that in the judgement of the investigator is likely to interfere with assessment of safety or efficacy of study treatment.
- Prior treatment with autologous cellular immunotherapy (e.g., CAR T) or gene therapy directed at any target.
- Treatment with anti-CD20+, CD19+ or BCMA+ monoclonal antibody therapy within 3 months of trial initiation or with B cell counts less than 50% of the lower limit of normal.
- Prior history of solid organ transplantation
- QTc >450msec in males or >470msecs in females
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase 1, Open-Label, Single Center Study of AZD0120 (Also Known as GC012F), a Chimeric Antigen Receptor T Cell Therapy Targeting CD19 and B Cell Maturation Antigen (BCMA), in Subjects With Relapsed and Refractory Persistent Autoimmune Cytopenia
This is a phase 1 study to evaluate the safety and tolerability of AZD0120 in patients with relapsed or refractory immune mediated cytopenia (primary chronic Immune Thrombocytopenia (ITP) or autoimmune hemolytic anemia (wAIHA)).
- Conditions
- Cytopenia
- Being tested
- AZD0120, Bendamustin
- Medical subject headings
- Cytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AZD0120Drug
One time administration on Day 0.
BendamustinDrug
Participants will be given bendamustin for 2 days (study Day -5 and Day -4) followed by 3 days of rest.
| Study group | Role | What they receive |
|---|---|---|
| AZD0120 (cell therapy product) + Bendamustine | Experimental | Participants receive AZD0120 (CAR-T therapy product) on Day 0 and Bendamustine for 2 days prior to the CAR-T therapy administration. Receives: Drug: AZD0120; Drug: Bendamustin |
| AZD0120 (cell therapy product) | Experimental | Participants receive AZD0120 (CAR-T therapy product) on Day 0. Receives: Drug: AZD0120 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
The incidence of adverse events defined as dose-limiting toxicities (DLTs), cytokine release syndrome (CRS), and immune effector cell-associated neurotoxicity syndrome (ICANS).
Measured: within 28 days after the CAR-T administration
Secondary outcomes (3)
Incidence of adverse events and serious adverse events following infusion
Evaluate the safety of AZD0120 cells by the incidence and severity of adverse events, and serious adverse events, following infusion. Events will be recorded and graded according to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 or ASTCT Consensus Grading.
Measured: until 28 days after the CAR-T administration
Number of CAR-T cells in blood
The CAR T cell expansion and persistence will be followed at designated time points after infusion. Study participants should have detectable CAR T cells in the blood in at least 2 consecutive time points (Day +3, +7, +10, +14, +21, +28).
Measured: Day +3, +7, +10, +14, +21, +28
Clinical response rate
Measured by complete response (CR), response (R), durable response (DR), and treatment-free remission for each disease arm as determined by published guidelines.
Measured: At 3, 6, 9, and 12-months post infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Aug 2026
- Study startSep 2026 (estimated)
- Primary completionJul 2041 (estimated)
- Study completionJul 2041 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CytopeniaStudies of the same condition, open and under way.
- 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
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 16 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Saurabh Dahiya
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Saurabh Dahiya, Stanford University
- Investigators
- Saurabh Dahiya, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07790575
- Sponsor's protocol ID
- 82368
- Acronym
- ACE-CAR T
- Status last verified
- Aug 2026
- First submitted
- 24 Aug 2026
- Last updated
- 27 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-Label, Single Center Study of AZD0120 (Also Known as GC012F), a Chimeric Antigen Receptor T Cell Therapy Targeting CD19 and B Cell Maturation Antigen (BCMA), in Subjects With Relapsed and Refractory Persistent Autoimmune Cytopenia
- ClinicalTrials.gov ID
- NCT07790575
- Acronym / sponsor ID
- ACE-CAR T / 82368
- Conditions
- Cytopenia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 16 participants (estimated)
- Lead sponsor
- Saurabh DahiyaUniversity, hospital or other
- Study start
- Sep 2026 (estimated)
- Primary completion
- Jul 2041 (estimated)
- Study completion
- Jul 2041 (estimated)
- First posted
- 27 Aug 2026 (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 is a phase 1 study to evaluate the safety and tolerability of AZD0120 in patients with relapsed or refractory immune mediated cytopenia (primary chronic Immune Thrombocytopenia (ITP) or autoimmune hemolytic anemia (wAIHA)).
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AZD0120 (cell therapy product) + Bendamustine | Experimental | Drug: AZD0120, Drug: BendamustinParticipants receive AZD0120 (CAR-T therapy product) on Day 0 and Bendamustine for 2 days prior to the CAR-T therapy administration. |
| AZD0120 (cell therapy product) | Experimental | Drug: AZD0120Participants receive AZD0120 (CAR-T therapy product) on Day 0. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (33 listed)
- Diagnosis of primary chronic immune thrombocytopenia (ITP) or warm autoimmune hemolytic anemia (wAIHA) according to the published consensus criteria
- a. 2 or more prior treatments for ITP wherein the following conditions are required to define prior treatments:
- These medications may be given in combination or in series.
- Thus, in order to be study eligible, a potential participant must have refractory chronic ITP and have received high dose steroids, IVIG, TPO agonists, and anti-CD20mAbs, with evidence of treatment failure, resistance, or relapse following steroids, TPO agonists, and anti-CD20 mAbs. Participants must have failed both steroids and TPO-RA to be eligible. b. 2 or more prior treatments for AIHA wherein the following conditions are required to define prior treatments:
- Thus, in order to be study eligible, a potential participant must have refractory AIHA and have received high dose steroids, IVIG, and anti-CD20mAbs, with evidence of treatment failure, resistance, or relapse following steroids and anti-CD20 mAbs.
Key exclusion criteria (27 listed)
- For ITP - ITP Bleeding Scale Grade 3 or higher at the screening visit (with the exception of menorrhagia controlled with hormonal therapy +/- tranexamic acid)
- For ITP - Hgb < 8 g/dL (NOTE: Exclusion does not apply to Evans Syndrome).
- For wAIHA - subjects with signs of hemolysis not attributable to wAIHA are excluded.
- For wAIHA - platelet count <100,000/uL (NOTE: ITP subjects may be below this threshold if due to active ITP. Exclusion does not apply to Evans Syndrome).
- For ITP - Secondary ITP (including but not limited to associated with a lymphoproliferative disorder, positive screening tests for HIV, HCV, H. pylori, Common Variable Immunodeficiency)
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 |
|---|---|---|
| AZD0120One time administration on Day 0. | Drug | AZD0120 (cell therapy product), AZD0120 (cell therapy product) + Bendamustine |
| BendamustinParticipants will be given bendamustin for 2 days (study Day -5 and Day -4) followed by 3 days of rest. | Drug | AZD0120 (cell therapy product) + Bendamustine |
Primary outcome
Incidence of adverse events
Time frame: within 28 days after the CAR-T administration
The incidence of adverse events defined as dose-limiting toxicities (DLTs), cytokine release syndrome (CRS), and immune effector cell-associated neurotoxicity syndrome (ICANS).
3 secondary outcomes
Incidence of adverse events and serious adverse events following infusion
Time frame: until 28 days after the CAR-T administration
Number of CAR-T cells in blood
Time frame: Day +3, +7, +10, +14, +21, +28
Clinical response rate
Time frame: At 3, 6, 9, and 12-months post infusion
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 27 Aug 2026.
- Individual participant dataSharing planned: No
Related trials for Cytopenia
RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
RecruitingPhase 3
rhTPO for Thrombocytopenia in Chronic Liver Disease Patients Undergoing Elective Invasive Procedures.
Thrombocytopenia Associated With Chronic Liver Disease
RecruitingPhase 4
Hematological Safety of Dipyrone Versus Paracetamol After Dental Extractions
Hematologic Test, Agranulocytosis
RecruitingObservational study
Clonal Hematopoiesis and Therapy-Emergent Myeloid Neoplasms in Patients With Cancers, CHANCES Study
Recurrent Fallopian Tube Carcinoma, Recurrent Ovarian Carcinoma
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
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.
Saurabh Dahiya, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AZD0120 and Bendamustin in people with Cytopenia, run by Saurabh Dahiya at 1 site in United States.
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 33 inclusion and 27 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: opening soon, as of the sponsor's last update on 27 Aug 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 United States. 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 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: within 28 days after the CAR-T administration). 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 27 Aug 2026.