A Phase 1, Open-label Study to Evaluate the Safety and Clinical Activity of Azercabtagene Zapreleucel in Participants With B-cell Mediated Autoimmune Disorders
Summary
The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel).
Arms & interventions
- DrugAzercabtagene zapreleucel (azer-cel)
IV infusion
Outcome measures
Primary
Number of Participants with Dose-Limiting Toxicities (DLTs)
DLT will be determined using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time frame: From Day 0 to Day 28
Secondary
Number of Participants with Treatment Emergent Adverse Events (TEAEs), Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs)
Time frame: Up to Day 720
Change From Baseline in CAR T-cell Toxicities
Time frame: Baseline, up to Day 720
Pharmacokinetics (PK) Plasma Concentrations of Azer-cel
Time frame: Up to Day 720
Pharmacodynamics (PD) Plasma Concentrations of Azer-cel
Time frame: Up to Day 720
Time to Confirmed Disability Progression (CDP)
Time frame: Up to Day 720
Time to Confirmed Disability Improvement (CDI)
Time frame: Up to Day 720
Change From Baseline in Brain MRI Gadolinium Enhancing T1, New or Enlarging Hyperintense T2
Time frame: Baseline, up to Day 720
Change From Baseline in Whole Brain Atrophy
Time frame: Baseline, up to Day 720
Change From Baseline in Modified Rankin Scale (mRS)
Time frame: Baseline, up to Day 720
Change From Baseline in Visual Acuity on Landolt C Broken Ring Chart (Low Contrast Visual Acuity [LCVA]) and High Contrast Visual Acuity (HCVA)
Time frame: Baseline, up to Day 720
Change from Baseline in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score
Time frame: Baseline, up to Day 720
Change from Baseline in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r)
Time frame: Baseline, up to Day 720
Time to Relapse, as Defined by a 1 Point Change in Adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT)
Time frame: Baseline, up to Day 720
Change From Baseline in Inflammatory Neuropathy Cause and Treatment (INCAT) Score
Time frame: Baseline, up to Day 720
Eligibility criteria
Study locations (8)
TG Therapeutics Investigational Trial Site
La Jolla, California, 92093
TG Therapeutics Investigational Trial Site
Ann Arbor, Michigan, 48019
TG Therapeutics Investigational Trial Site
Omaha, Nebraska, 68198
TG Therapeutics Investigational Trial Site
New York, New York, 10025
TG Therapeutics Investigational Trial Site
Rochester, New York, 14642
TG Therapeutics Investigational Trial Site
Cleveland, Ohio, 44195
TG Therapeutics Investigational Trial Site
Columbus, Ohio, 43210
TG Therapeutics Investigational Trial Site
Milwaukee, Wisconsin, 53226