NCT04545762

Brief Summary

This study will assess safety and feasibility of infusing genetically modified autologous T cells transduced to express a chimeric antigen receptor targeting the B cell surface antigen Cluster of Differentiation 19 (CD19).

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
36

participants targeted

Target at P50-P75 for phase_1

Timeline
5mo left

Started Sep 2020

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress94%
Sep 2020Oct 2026

First Submitted

Initial submission to the registry

August 31, 2020

Completed
11 days until next milestone

First Posted

Study publicly available on registry

September 11, 2020

Completed
Same day until next milestone

Study Start

First participant enrolled

September 11, 2020

Completed
6.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2026

Last Updated

January 14, 2026

Status Verified

August 1, 2025

Enrollment Period

6.1 years

First QC Date

August 31, 2020

Last Update Submit

January 12, 2026

Conditions

Keywords

CAR-T Therapy

Outcome Measures

Primary Outcomes (2)

  • Proportion of participants with treatment-emergent adverse events (AEs)

    Participants treated with conforming product who received the target doses of anti-CD19 CAR-T infusion will be included in the analysis. Proportion of participants with treatment-emergent adverse events of CAR-T in B-cell NHL, as graded by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE version 5.0), revised Cytokine Release Syndrome (CRS) grading criteria, and American Society for Transplantation and Cellular Therapy (ASTCT) immune effector cell (IEC) -associated neurotoxicity syndrome (ICANS) Consensus Grading for Adults (for neurotoxicity grading).

    From initiation of study treatment to 12 months following CAR-T infusion, approximately 15 months

  • Proportion of participants who experience a dose-limiting toxicity (DLT) (Dose escalation)

    A DLT includes AEs graded according to CTCAE version 5.0, with the exceptions of CRS and neurotoxicity, which are graded using CRS and ICANS criteria. DLTs must 1) be suspected to be secondary to CAR-T cell infusion, 2) occur during the first 30 days after infusion and 3) meet the following criteria: 1. Grade 3 or 4 non-hematologic toxicities of any duration, with following exceptions: Grade 3 laboratory abnormalities without associated symptomatology or clinical consequence that resolve in \< 7 days; AEs associated with Grade \<= 2 CRS; Toxicities associated with Grade 3 CRS (except cardiac or pulmonary organ toxicity) that improves to grade \<= 2 within 3 days of intervention; isolated renal or hepatic grade 3 organ toxicity that does not resolve within 7 days; Laboratory abnormalities compatible with tumor lysis syndrome; Grade 4 hematological toxicity that persists at grade \>= 3 despite maximum supportive care for \>21 days.

    From initiation of study treatment to 30 days following CAR-T infusion

Secondary Outcomes (10)

  • Proportion of participants with CAR-T infusion related adverse events (Dose Escalation)

    From initiation of CD19 CAR T-cell manufacturing to end of infusion, approximately 18 days

  • Proportion of participants with delayed infusion due to study-related adverse events

    From T-cell collection to end of infusion, approximately 18 days

  • Overall Response Rate (ORR)

    From initiation of study treatment to 12 months after getting CAR-T infusion, approximately 15 months

  • Complete Response Rate

    From initiation of study treatment to 12 months after getting CAR-T infusion, approximately 15 months

  • Partial Response Rate

    From initiation of study treatment to 12 months after getting CAR-T infusion, approximately 15 months

  • +5 more secondary outcomes

Study Arms (2)

CLOSED TO ENROLLMENT: Dose escalation (CART-T Therapy)

EXPERIMENTAL

Participants will undergo Apheresis (1 day) to collect autologous lymphocytes/ mononuclear cells as per University of California, San Francisco (UCSF) institutional practices. CAR-T cell manufacturing (estimated \~13-14 days), during which participants will receive a lymphodepleting regimen of immunosuppressive chemotherapy (Cyclophosphamide 300 mg/m2/IV and fludarabine 30 mg/m2 /IV) followed by the infusion of CAR-T cells at an initial dose of 5 x 10\^5 cells/kg, targeting CD19 over 5-30 minutes. Participants will be followed up 30 days after infusion, for up to 12 months, if the participant in continued remission, and for survival up to 15 years.

Dose Expansion: Burkitt, Marginal Zone, or Waldenström Macroglobulinemia ONLY (CAR-T Therapy)

EXPERIMENTAL

Participants with Burkitt lymphoma, or Marginal Zone Lymphoma (MZL) and Waldenström Macroglobulinemia (WM) will undergo Apheresis (1 day) to collect autologous lymphocytes/ mononuclear cells as per University of California, San Francisco (UCSF) institutional practices. CAR-T cell manufacturing (estimated \~13-14 days), during which participants will receive a lymphodepleting regimen of immunosuppressive chemotherapy (Cyclophosphamide 300 mg/m2/IV and fludarabine 30 mg/m2 /IV) followed by the infusion of the maximum tolerated dose of CAR-T cells established in the dose escalation phase, targeting CD19 over 5-30 minutes. Participants will be followed up 30 days after infusion, for up to 12 months, if the participant in continued remission, and for survival up to 15 years.

Drug: FludarabineDrug: CyclophosphamideBiological: anti-CD19 CAR-T cells

Interventions

Given intravenously (IV)

Also known as: Fludarabine 30mg/m2, Fludara
Dose Expansion: Burkitt, Marginal Zone, or Waldenström Macroglobulinemia ONLY (CAR-T Therapy)

Given intravenously (IV)

Also known as: Cyclophosphamide 300mg/m2, Cytoxan, Endoxan, Neosar, Procytox, Revimmune, Cycloblastin
Dose Expansion: Burkitt, Marginal Zone, or Waldenström Macroglobulinemia ONLY (CAR-T Therapy)

Single infusion

Also known as: CD19-directed Chimeric Antigen Receptor T-Cell (CAR T-cell) therapy, CD19-directed chimeric antigen receptor (CAR) T-cell therapy
Dose Expansion: Burkitt, Marginal Zone, or Waldenström Macroglobulinemia ONLY (CAR-T Therapy)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Dose expansion Cohorts:
  • Cohort B (Burkitt):
  • Participants must have a diagnosis of relapsed or refractory Burkitt Lymphoma
  • Participants with Burkitt lymphoma must have relapsed or failed to respond to at least 1 prior line of multiagent chemoimmunotherapy with prior exposure to both an anti-CD20 antibody agent and an anthracycline.
  • No significant circulating disease, defined as an elevated total lymphocyte count above the upper limit of normal (ULN) due to the presence of malignant cells.
  • Participants must have measurable disease as defined below:
  • Participants with Burkitt Lymphoma must have Positron Emission Tomography (PET)-positive disease according to "Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification"
  • Cohort M/W (Marginal/Waldenström):
  • Participants must have a diagnosis of relapsed or refractory Marginal Zone Lymphoma (MZL), or Lymphoplasmacytic Lymphoma (LPL)/Waldenström Macroglobulinemia (WM):
  • o Participants with indolent lymphomas (nodal or extranodal marginal zone lymphoma, and lymphoplasmacytic lymphoma) must have relapsed after or have been refractory to ≥ 2 prior lines of multi-agent chemoimmunotherapy including prior exposure to an anti-CD20 antibody and an alkylating agent.
  • Participants must have measurable disease as defined below:
  • o Participants with Marginal Zone Lymphoma or Lymphoplasmacytic Lymphoma/Waldenström Macroglobulinemia: must either have PET-positive disease according to "Recommendations for Initial Evaluation, Staging, and Response Assessment of Hodgkin and Non-Hodgkin Lymphoma: The Lugano Classification" or serum monoclonal immunoglobulin M (IgM) paraprotein \> 0.5 g/dL.
  • Participants with indolent lymphoma (Marginal Zone Lymphoma or Lymphoplasmacytic Lymphoma/Waldenström Macroglobulinemia) must have symptomatic disease necessitating systemic treatment.
  • In addition, all participants must meet the following criteria:
  • CD19-positive by either immunohistochemistry or flow cytometry analysis on any biopsy. If prior anti-CD19 therapy has been administered, CD19-positivity has to be re-established on the most recent biopsy.
  • +15 more criteria

You may not qualify if:

  • Autologous transplant within 6 weeks of planned CAR-T cell infusion.
  • Recipient of prior CAR-T cell therapy targeting CD19 outside of this protocol.
  • Active other malignancy, other than non-melanoma skin cancer, carcinoma in situ (e.g., cervix, bladder, or breast).
  • Human immunodeficiency virus (HIV) seropositivity.
  • Serologic status reflecting active hepatitis B or C infection. Participants that are positive for hepatitis B core antibody, hepatitis B surface antigen (HBsAg), or hepatitis C antibody must have a negative polymerase chain reaction (PCR) prior to enrollment. (PCR positive participants will be excluded.)
  • Participants with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, pulmonary abnormalities or psychiatric illness/social situations that would limit compliance with study requirements.
  • Pregnant or breastfeeding women are excluded from this study because CAR-T cell therapy may be associated with the potential for teratogenic or abortifacient effects. Because there is an unknown, but potential risk for adverse events in nursing infants secondary to treatment of the mother with CAR-T cells, breastfeeding should be discontinued. These potential risks may also apply to other agents used in this study. NOTE: Women of childbearing potential must have a negative serum or urine pregnancy test.
  • Participants with history of clinically relevant central nervous system (CNS) pathology such as epilepsy, seizure disorders, paresis, aphasia, uncontrolled cerebrovascular disease, severe brain injuries, dementia and Parkinson's disease.
  • History of autoimmune disease (e.g., rheumatoid arthritis, systemic lupus erythematosus) with requirement of immunosuppressive medication within 6 months.
  • Body weight \<40 kilograms(kg).
  • Eligibility for Infusion of Investigational Product:
  • No significant laboratory abnormalities. Laboratory result abnormalities that are considered not clinically significant by the principal investigator AND are not the result of a demonstrated active infection or an active central nervous system condition.
  • ECOG performance status \< 2
  • No evidence of uncontrolled intercurrent illness including, but not limited to ongoing or active infection, inflammatory response, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, pulmonary abnormalities or psychiatric illness/social situations.
  • No new neurologic symptoms suggestive of an active central nervous system condition, or uncontrolled CNS involvement by lymphoma.
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of California, San Francisco

San Francisco, California, 94143, United States

RECRUITING

Related Publications (1)

  • Ernst M, Oeser A, Besiroglu B, Caro-Valenzuela J, Abd El Aziz M, Monsef I, Borchmann P, Estcourt LJ, Skoetz N, Goldkuhle M. Chimeric antigen receptor (CAR) T-cell therapy for people with relapsed or refractory diffuse large B-cell lymphoma. Cochrane Database Syst Rev. 2021 Sep 13;9(9):CD013365. doi: 10.1002/14651858.CD013365.pub2.

MeSH Terms

Conditions

Lymphoma, Non-HodgkinBurkitt LymphomaLymphoma, Mantle-CellLymphoma, FollicularLymphoma, B-Cell, Marginal ZoneLymphoma, Large B-Cell, DiffuseLeukemia, Lymphocytic, Chronic, B-Cell

Interventions

fludarabinefludarabine phosphateCyclophosphamideImmunotherapy, AdoptiveTherapeuticsAutomobiles

Condition Hierarchy (Ancestors)

LymphomaNeoplasms by Histologic TypeNeoplasmsLymphoproliferative DisordersLymphatic DiseasesHemic and Lymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesEpstein-Barr Virus InfectionsHerpesviridae InfectionsDNA Virus InfectionsVirus DiseasesInfectionsTumor Virus InfectionsLymphoma, B-CellLeukemia, B-CellLeukemia, LymphoidLeukemiaHematologic DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsAdoptive TransferImmunization, PassiveImmunizationImmunotherapyImmunomodulationBiological TherapyImmunologic TechniquesInvestigative TechniquesMotor VehiclesTransportationTechnology, Industry, and Agriculture

Study Officials

  • Carrie Ho, MD

    University of California, San Francisco

    PRINCIPAL INVESTIGATOR

Central Study Contacts

UCSF HDFCCC Cancer Immunotherapy Program

CONTACT

Study Design

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

Study Record Dates

First Submitted

August 31, 2020

First Posted

September 11, 2020

Study Start

September 11, 2020

Primary Completion (Estimated)

October 31, 2026

Study Completion (Estimated)

October 31, 2026

Last Updated

January 14, 2026

Record last verified: 2025-08

Data Sharing

IPD Sharing
Will not share

Locations