Cell and Gene Therapy Clinical Studies

Principal Investigator: Thomas H. Brannagan, MD
NCT Number: NCT07304154

This study will have two Phases: Phase 1a and Phase 1b. The goals of this clinical study are to learn more about the study drug KITE-363, by evaluating its safety, tolerability and efficacy in participants with relapsed/refractory autoimmune neurologic diseases. ​​​​

Status: Open to enrollment

Principal Investigator: Rebecca Straus Farber, MD
NCT Number: NCT07224373

This study will evaluate AZD0120 for safety, including DLTs and TEAEs, by the SRC for determination of the Recommended Phase 2 dose for each disease cohort. Approximately 9-12 participants will be evaluated per disease cohort.

Status: Open to enrollment

Principal Investigator: Rebecca Straus Farber, MD
NCT Number: NCT06680037

The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel).

Status: Open to enrollment

Principal Investigator: Christina Ulane, MD, PhD
NCT Number: NCT06626919

A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia gravis (GMG). Anitocabtagene autoleucel (anito-cel) is a BCMA-directed CAR-T cell therapy.

Status: Open to enrollment

Principal Investigator: Christina Ulane, MD, PhD
NCT Number: NCT06799247

The AURORA Study is evaluating the safety, tolerability, and efficacy of an investigational mRNA CAR T-cell therapy known as Descartes-08 in adults with acetylcholine receptor autoantibody -positive generalized myasthenia gravis. Part 1 of the study will last around 6 months. For eligible participants, Part 2 will last around 8 months.

Status: Closed to enrollment

Principal Investigator: Christina Ulane, MD, PhD
NCT Number: NCT06359041

Myasthenia gravis (MG) is a rare autoimmune disorder characterized by autoantibody responses that cause defective transmission of signals at the neuromuscular junction, resulting in a distinctive pattern of weakness. Patients with generalized MG (gMG) typically experience symptoms associated with ocular disease in addition to weakness of many other voluntary muscle groups, including extremity, bulbar, and respiratory muscles. MG is considered a classic example of a B-cell mediated autoimmune disease. Currently, there are no curative treatments for MG. This study is being conducted to evaluate the safety and efficacy of an investigational cell therapy, CABA-201, that can be given to patients with gMG. A single dose of CABA-201 in combination with cyclophosphamide (CY) and fludarabine (FLU) will be evaluated.

Status: Open to enrollment

Principal Investigator: Rebecca Straus Farber, MD
NCT Number: NCT06220201

The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG).

Status: Closed to enrollment

Principal Investigator: Laura Geraldino Pardilla, MD, MSc
NCT Number: NCT06897930

This is a Phase 1b/2, single-arm, open-label, multi-center, clinical study of AZD0120, a CD19/BCMA dual CAR T cell therapy, to evaluate the safety, tolerability, and efficacy in adult participants with refractory Systemic Lupus Erythematosus.

Status: Open to enrollment

Principal Investigator: Elana Bernstein, MD
NCT Number: NCT07295847

This is a Phase 1b, open-label, multi-center, multi-cohort clinical study of AZD0120, a CD19/BCMA dual CAR T-cell therapy, to evaluate the safety in adult participants with systemic sclerosis (SSc), idiopathic inflammatory myopathies (IIM), or difficult-to-treat rheumatoid arthritis (D2T RA) for determination of the recommended phase 2 dose for each disease cohort. Approximately 9-12 participants will be evaluated per disease cohort.

Status: Open to enrollment

Principal Investigator: Elana Bernstein, MD
NCT Number: NCT06328777

Systemic sclerosis (SSc), also known as scleroderma, is a rare autoimmune disorder characterized by autoantibody production and abnormal B cell function. Though the cause of SSc is not well understood, it is thought to involve B cells that cause the body to attack different tissues in one's own body, causing skin and organ fibrosis. This study is being conducted to evaluate the safety and efficacy of an investigational therapy, CABA-201, a CD19-CAR T cell therapy, that can be given to patients with SSc who have active disease. A single dose of CABA-201 in combination with cyclophosphamide (CY) and fludarabine (FLU) will be evaluated.

Status: Open to enrollment

Principal Investigator: Laura Geraldino Pardilla, MD, MSc
NCT Number: NCT07015983

The purpose of this study is to evaluate the efficacy, safety and drug levels of CC-97540 in participants with active systemic lupus erythematosus (SLE) including lupus nephritis with inadequate response to glucocorticoids and at least 2 immunosuppressants.

Status: Open to enrollment, not recruiting

Principal Investigator: Laura Geraldino Pardilla, MD, MSc
NCT Number: NCT06518668

This is an open-label, non-randomized, Phase 1 study. Subjects with SLE will receive cyclophosphamide LD followed by NKX019 to determine safety and preliminary efficacy.

The study will consist of 4 study periods for each study subject inclusive of Screening, Active Treatment, Follow-up, and Extended Follow-up. Disease assessments will occur every 90 days for 2 years.

Status: Closed to enrollment

Principal Investigator: Elana Bernstein, MD
NCT Number: NCT06544330

SYNCAR-001 + STK-009 is a 2-component human orthogonal (ho) IL-2 receptor-ligand cell therapy consisting of (1) SYNCAR-001, a CD19-directed chimeric antigen receptor T cell (CAR-T) co-expressing an engineered IL-2 beta receptor (hoRb); and (2) STK-009, an engineered pegylated IL-2 cytokine (hoIL-2) selective for hoRb. This study is being conducted to evaluate the safety and efficacy of a single dose of SYNCAR-001 followed by multiple subcutaneously administered doses of STK-009. No conditioning chemotherapy (lymphodepletion) will be administered. The study will follow a 3+3 design during dose escalation followed by dose expansion at the recommended phase 2 dose (RP2D).

Status: Closed to enrollment

Principal Investigator: Mark N. Stein, MD
NCT Number: NCT06267729

This is a first-time in human, multi-center, open label, Phase I/II study of AZD0754 autologous CAR T-cell therapy administered intravenously to participants with metastatic prostate cancer. The study is intended to assess the safety, cellular kinetics, pharmacodynamics, preliminary efficacy, and feasibility of manufacturing AZD0754 for patients with metastatic prostate cancer.

Status: Open to enrollment

Principal Investigator: Suzanne Lentzsch, MD, PhD
NCT Number: NCT07081646

Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.

Status: Open to enrollment

Principal Investigator: Ran Reshef, MD, MSc
NCT Number: NCT06500273

This is a randomized, open-label study in adult patients who have completed standard first line therapy for large B-cell lymphoma (LBCL) and achieved a complete response or partial response suitable for observation, but who have minimal residual disease (MRD) as detected by the Foresight CLARITY™ Investigational Use Only (IUO) MRD test, powered by PhasED-Seq™. The purpose of the trial is to assess the efficacy and safety of consolidation with cemacabtagene ansegedleucel (cema-cel), an allogeneic CD19 CAR T product, as compared to standard of care observation.

In this study, participants with MRD are randomized 1:1 to treatment with cema-cel or an observation arm. Treatment includes cema-cel following a lymphodepletion regimen of fludarabine and cyclophosphamide.

Prior to August 2025, participants may also have received an anti-CD52 monoclonal antibody, ALLO-647, as part of their lymphodepletion regimen.

Status: Open to enrollment

Principal Investigator: Ran Reshef, MD, MSc
NCT Number: NCT04989803

The goal of this clinical study is to learn more about the safety and effectiveness of the study drugs, KITE-363 and KITE-753, in participants with relapsed and/or refractory B-cell lymphoma.

Status: Open to enrollment

Principal Investigator: Ran Reshef, MD, MSc
NCT Number: NCT06420089

This is an open-label phase I study to determine the safety and recommended phase 2 dose (RP2D) of Senza5 CART5 cells in patients with relapsed or refractory CD5 positive nodal T cell NHL. RP2D will be based on the safety, tolerability, pharmacokinetics, and preliminary efficacy of Senza5 CART5 cells. This trial will evaluate up to 5 dose levels using the Bayesian Optimal Interval (BOIN) design enrolling 3 patients in each cohort to assess safety and achieve therapeutic levels so that the RP2D of Senza5 CART5 cells given as a single IV infusion can be determined.

Status: Open to enrollment, not currently recruiting

Principal Investigator: Ran Reshef, MD, MSc
NCT Number: NCT03666000

This is a Phase 1/1b, nonrandomized, open-label, parallel assignment, dose-escalation, and dose-expansion study to evaluate the safety and clinical activity of azer-cel, an allogeneic anti-CD19 CAR T, in adults with r/r B ALL, r/r B-cell NHL and CLL/SLL.

Status: Open to enrollment

Principal Investigator: Ran Reshef, MD, MSc
NCT Number: NCT05605899

The goal of this clinical study is to compare the study drug, axicabtagene ciloleucel, versus standard of care (SOC) in first-line therapy in participants with high-risk large B-cell lymphoma.

Status: Closed to enrollment

Principal Investigator: Ran Reshef, MD, MSc
NCT Number: NCT05473910

This is a multi-center, non-randomized, concurrent controlled, multi-arm, Phase 1 interventional, open-label, biologic assignment-based umbrella study evaluating the feasibility, safety and preliminary efficacy of an escalating dose regimen of up to 2 doses of TSC-100 and TSC-101 in patients with AML, MDS, or ALL following HCT from a haploidentical donor, MMUD, or MUD.

Status: Closed to enrollment

Principal Investigator: Ran Reshef, MD, MSc
NCT Number: NCT04923893

The purpose of this study is to compare the efficacy of Bortezomib, Lenalidomide and Dexamethasone (VRd) induction followed by a single administration of ciltacabtagene autoleucel (cilta-cel) versus VRd induction followed by Lenalidomide and Dexamethasone (Rd) maintenance in newly diagnosed multiple myeloma participants for whom ASCT is not planned as initial therapy in terms of Progression Free Survival (PFS).

Status: Closed to enrollment

Principal Investigator: Markus Mapara, MD, PhD
NCT Number: NCT04819841

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

Status: Open to enrollment, not recruiting

Principal Investigator: Markus Mapara, MD, PhD
NCT Number: NCT05456880

This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD.

Status: Closed to enrollment

Principal Investigator: Pawel Muranski, MD
NCT Number: NCT05183490

The primary objective is to determine the safety and feasibility of administering R-MVST cells to patients with refractory viral reactivation and/or symptomatic disease caused by Epstein Barr Virus (EBV), cytomegalovirus (CMV), adenovirus (ADV) or BK virus. R-MVST cells will be generated on-demand from the closest partially human leukocyte antigen (HLA)-matched (minimum haploidentical) healthy donors or from the original allo-transplant donor if available. The investigator will closely monitor the recipients for potential toxicities including graft-versus-host disease (GVHD) post-infusion.

Secondary objectives are to determine the effect of R-MVST infusion on viral load, possible recovery of antiviral immunity post-infusion and for evidence of clinical responses and overall survival. Recipients will be monitored for secondary graft failure at day 28 post R-MVST infusion.

Status: Open to enrollment

Principal Investigator: Alexandra J Coromilas, MD
NCT Number: NCT04422912

Pemphigus vulgaris (PV) is a B-cell mediated autoimmune disorder in which painful blisters are formed on the skin or mucosal membrane, including the mouth, nose, throat, eyelids, anus, and genitals.

This phase 1/2 study is being conducted in two parts. The first part is the main study conducted to find the maximum tolerated dose and optimal fractionated infusion schedule of an investigational cell therapy, DSG3-CAART, that can be given to patients with mucosal PV who are inadequately managed by standard therapies. This study is closed to enrollment.

The second part is a sub-study is being conducted to investigate if CABA-201, also called resecabtagene autoleucel, or "rese-cel", can be safely administered while achieving clinical responses without the need for preconditioning in mucosal-dominant PV (mPV) and mucocutaneous PV (mcPV) patients. This sub-study is open to enrollment.

DSG3-CAART or CABA-201 may potentially lead to complete and durable remission of disease.

Status: Open to enrollment

Principal Investigator: Jacqueline Yuey Lonier, MD
NCT Number: NCT06919354

This is a 78-week single arm, multi-center, Phase 1 study to evaluate the safety, tolerability, cellular kinetics, and biomarker changes in C-peptide over time of GNTI-122, an investigational cell therapy manufactured from a participant's own blood cells in adult participants with recently diagnosed T1D. After assessment of eligibility, participants who qualify for the study will be enrolled sequentially in 1 of 3 cohorts. Cohort 1 participants (n=3) receive a low dose of GNTI-122 . Cohort 2 participants (n=3) receive a high dose of GNTI-122. Cohort 3 participants (n=10) receive a high dose of GNTI-122 in combination with rapamycin. Participants are followed for 78 weeks after the administration of GNTI-122 during which safety and efficacy assessments are made, including vital signs, ECG, physical exam, clinical labs, and monitoring of adverse events and concomitant medications. Disease markers (e.g., MMTT-stimulated C-peptide, HbA1c) and pharmacodynamic activity (e.g., lymphocyte subsets and phenotypes, effector T cell responses to islet antigens ex vivo, T1D autoantibodies) will be monitored serially throughout the study. The study will include sentinel dosing and a Safety Review Committee to ensure participant safety.

Status: Open to enrollment