Autoimmune Cytopenia Elimination by Chimeric Antigen Receptor T-cell Therapy

NCT07790575 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 82368
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief 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)).

Conditions

Cytopenia

Outcome Measures

Primary Outcomes (1)

• 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).

  within 28 days after the CAR-T administration

Secondary Outcomes (3)

• Incidence of adverse events and serious adverse events following infusion

  until 28 days after the CAR-T administration

• Number of CAR-T cells in blood

  Day +3, +7, +10, +14, +21, +28

• Clinical response rate

  At 3, 6, 9, and 12-months post infusion

Study Arms (2)

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.

Drug: AZD0120; Drug: Bendamustin

AZD0120 (cell therapy product)

EXPERIMENTAL

Participants receive AZD0120 (CAR-T therapy product) on Day 0.

Drug: AZD0120

Interventions

AZD0120 DRUG

One time administration on Day 0.

AZD0120 (cell therapy product); AZD0120 (cell therapy product) + Bendamustine

Bendamustin DRUG

Participants will be given bendamustin for 2 days (study Day -5 and Day -4) followed by 3 days of rest.

AZD0120 (cell therapy product) + Bendamustine

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• st line treatment includes steroids (dexamethasone, prednisone or equivalent), IVIG, and/or Anti-Rhd Ig. These medications may be administered in combination or in series.
• nd 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.
• 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:
• st 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.
• nd 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.

You may not qualify if:

• 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 wAIHA - subjects with signs of hemolysis not attributable to wAIHA are excluded.
• 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)
• 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.
• 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 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.

Sponsors & Collaborators

• Saurabh Dahiya: lead

Study Sites (1)

Stanford University

Stanford, California, 94305, United States

Location

MeSH Terms

Conditions

Cytopenia

Interventions

Bendamustine Hydrochloride

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Butyrates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Benzimidazoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Saurabh Dahiya, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Professor of Medicine

Study Record Dates

• First Submitted: August 24, 2026
• First Posted: August 27, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): July 1, 2041
• Study Completion (Estimated): July 1, 2041
• Last Updated: August 27, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)