Feasibility and Safety of Collecting and Combining Autologous Hematopoietic Stem Cells With Chimeric Antigen Receptor (CAR) T-Cell Therapy in Subjects With Relapsed/Refractory Hematological Malignancies

August 6, 2026 updated by: Joshua Sasine, MD, PhD

IIT2022-04-Sasine-CAR-T: A Phase 1 Single-arm, Open-label Study to Evaluate the Feasibility and Safety of Collecting and Combining Autologous Hematopoietic Stem Cells With Chimeric Antigen Receptor (CAR) T-Cell Therapy in Subjects With Relapsed/Refractory Hematological Malignancies

The study is designed to examine the feasibility and safety of collecting autologous hematopoietic stem cells (HSCs) to be combined with CAR T-cell therapy for patients with relapsed/refractory (r/r) hematological disease. The study will evaluate feasibility of collecting the target dose of HSCs from at least 50% of enrolled patients. The study will assess safety based on incidence and severity of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) in the first 60 days post CAR T dosing, and also through the collection of adverse events (AEs) and serious adverse events (SAEs) as well as the durability of response after treatment with HSCs with CAR T. The study follows an open-label, single-center and single non-randomized cohort design. 20 subjects with r/r hematological malignancies will be enrolled and treated to evaluate the feasibility and preliminary safety of collecting autologous HSCs and combining them with CAR T-cell therapy.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
20

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90048
        • Recruiting
        • Cedars-Sinai Medical Center
        • Sub-Investigator:
          • Ronald Paquette, MD
        • Sub-Investigator:
          • Akil Merchant, MD
        • Sub-Investigator:
          • Justin Darrah, MD
        • Sub-Investigator:
          • John Chute, MD
        • Sub-Investigator:
          • Robert Vescio, MD
        • Sub-Investigator:
          • Noah Merin, MD, PhD
        • Sub-Investigator:
          • Hannah Lee, MD
        • Sub-Investigator:
          • David Oveisi, MD
        • Sub-Investigator:
          • Leslie Ballas, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Age 18 - 85 years.
  • Histologically proven hematological malignancy according to the World Health Organization 2016 classification criteria for which a commercially available, FDA-approved CAR T product exists.

  • Relapsed or refractory disease, defined by the following:

    • Disease progression after last regimen, or
    • Refractory disease: failure to achieve a partial response (PR) or complete remission (CR) to the last regimen
  • At least 2 weeks or 5 half-lives, whichever is shorter, must have elapsed since any prior systemic therapy for the malignancy at the time the subject is planned for leukapheresis.
  • Toxicities due to prior therapy must be stable or recovered to ≤ Grade 1 with the exception of alopecia.
  • Subjects with an active uncontrolled infection should not start CAR T treatment until the infection has resolved.
  • Eastern cooperative oncology group (ECOG) performance status 0 - 2.
  • Adequate hematologic, hepatic, and cardiac function
  • Serum pregnancy test for women of childbearing potential (WOCBP) at Screening.
  • Willing to comply to research specimen collection as specified in the protocol.
  • Written informed consent obtained from subject and ability for subject to comply with the requirements of the study.

Exclusion Criteria:

  • Autologous hematopoietic cell transplant intent or execution within 8 weeks of planned CAR T infusion.
  • History of allogeneic cell transplantation within 8 weeks of planned CAR T infusion.
  • Presence or suspicion of fungal, bacterial, viral, or other infection that is uncontrolled or requiring IV antimicrobials for management at time of screening.
  • History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease within 6 months of enrollment.
  • History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, or any autoimmune disease with CNS involvement.
  • Doses of corticosteroids of greater than or equal to 5 mg/day of prednisone or equivalent doses of other corticosteroids and other immunosuppressive drugs are not allowed prior to enrollment. A washout period of 10 days prior to leukapheresis and 10 days prior to anti-CD19 CAR T cell administration is required.
  • Any medical condition likely to interfere with assessment of feasibility or safety of study treatment.
  • Live vaccine ≤ 6 weeks prior to planned start of conditioning regimen.
  • History of severe immediate hypersensitivity reaction to any of the agents used in this study.
  • Current pregnancy or breastfeeding because of the potentially dangerous effects of the preparative chemotherapy on the fetus or infant.
  • Subjects of both sexes who are not willing to practice birth control from the time of consent through 6 months after the completion of conditioning chemotherapy. Females who have undergone surgical sterilization or who have been postmenopausal for at least 1 year are not considered to be of childbearing potential.
  • In the investigator's judgment, the subject is unlikely to complete all protocol-required study visits or procedures, including follow-up visits, or comply with the study requirements for participation.
  • Patients with obvious myeloid clonal hematopoiesis on the screening bone marrow biopsy will be excluded based on the risk of developing myeloid neoplasms with aHSC infusion.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: CAR T Therapy with Autologous Hematopoietic Stem Cells (aHSCs)
Biological: autologous hematopoietic stem cells added to planned CAR T
Autologous hematopoietic stem cells (aHSCs) infused on Day 10 after CAR T (any FDA-approved CAR T product) infusion on Day 0.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
To assess feasibility of collecting the target HSC cell dose for at least 50% of enrolled patients.
Time Frame: From Day 0 (CAR T infusion) to Day 10 (aHSC infusion).
Target dose collection of autologous HSCs (2 to 5 x 106 CD34+ cells/kg) defined as collection from at least 50% of patients enrolled in this study by Day 10.
From Day 0 (CAR T infusion) to Day 10 (aHSC infusion).
To assess safety of aHSC to planned CAR T therapy in the first 60 days through the incidence, severity, and duration of CRS based on the American Society for Transplantation and Cellular Therapy (ASTCT) consensus grading system.
Time Frame: From Day 0 to Day 60.
Safety will be assessed by incidence, severity, and duration of CRS per ASTCT consensus grading system. ASTCT CRS Consensus grading (Grade scale is 1-4) is based on 3 CRS parameters: fever, hypotension, and hypoxia. Higher grade indicates worse outcome.
From Day 0 to Day 60.
To assess safety of aHSC to planned CAR T therapy in the first 60 days through the incidence, severity, and duration of ICANS based on the American Society for Transplantation and Cellular Therapy (ASTCT) consensus grading system.
Time Frame: From Day 0 to Day 60.
Safety will be assessed by incidence, severity, and duration ICANS per ASTCT consensus grading system. ASTCT ICANS Consensus grading (Grade scale is 1-4) is based on 5 neurotoxicity domains: Immune Effector Cell-Associated Encephalopathy (ICE) score, level of consciousness, seizure, motor findings, raised intracranial pressure (ICP)/cerebral edema. Higher grade indicates worse outcome.
From Day 0 to Day 60.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Response rate of CAR T at 6 weeks.
Time Frame: From Day 0 to Day 60.
Response rate of CAR T will be defined as the proportion of subjects who experience partial response (PR) or complete response (CR) assessed at 6 weeks or day 60. Response criteria used will be according to type of disease: Response by International Myeloma Working Group (IMWG) Criteria (for multiple myeloma); Response by Center for International Blood and Marrow Transplant Research (CIMBTR) Criteria (for acute lymphoblastic leukemia); Response by Lugano Criterial (for lymphoma).
From Day 0 to Day 60.
Assess rate of recovery of absolute neutrophil count (ANC) by Day 28.
Time Frame: From Day 0 to Day 28.
Recovery assessed by the proportion of patients with ANC recover to ≥ 500/µL.
From Day 0 to Day 28.
Assess red blood cell (RBC) count and transfusion independence by Day 28.
Time Frame: From Day 0 to Day 28.
The proportion of patients with RBC transfusion independence by Day 28.
From Day 0 to Day 28.
Assess platelet count and transfusion independence rate by Day 28.
Time Frame: From Day 0 to Day 28.
The proportion of patients with platelet transfusion independence by Day 28.
From Day 0 to Day 28.
Assess safety and tolerability of combining aHSCs with an FDA-approved CAR T regimen within first 52 weeks of aHSC infusion.
Time Frame: From Day 0 to Week 52.
Safety and tolerability will be assessed by the number of adverse events (AEs) and serious adverse events (SAEs) related to study treatment according to CTCAE v.5, up to Week 52.
From Day 0 to Week 52.
Median progression-free survival (PFS) for the duration of the study
Time Frame: From Day 0 until disease progression or withdrawal. Assessed up to 3 years.
PFS will be assessed from time of CAR T infusion until confirmation of disease progression or withdrawal of consent, whichever comes first.
From Day 0 until disease progression or withdrawal. Assessed up to 3 years.
Median overall survival (OS) for the duration of the study
Time Frame: From Day 0 until death due to any cause or withdrawal of consent. Assessed up to 3 years.
OS will be assessed from date of CAR T infusion until date of death due to any cause or withdrawal of consent, whichever comes first.
From Day 0 until death due to any cause or withdrawal of consent. Assessed up to 3 years.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Joshua Sasine, MD, PhD

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Joshua Sasine, MD, PhD, Cedars-Sinai Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 2, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 15, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 15, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 17, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 31, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 2, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 10, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 6, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • IIT2022-04-Sasine-CAR-T

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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