A Study to Assess the Safety and Clinical Activity of Azer-cel in Participants With B-cell Mediated Autoimmune Disorders

NCT06680037 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: TG-Azercel-101
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 8

Brief Summary

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

Conditions

B-cell Mediated Autoimmune Disorders

Keywords

Progressive forms of multiple sclerosis (PMS); Primary Progressive MS (PPMS); Secondary Progressive MS (SPMS); Multiple Sclerosis (MS); Neuromyelitis optica spectrum disorder (MNOSD); Myasthenia Gravis (MG); Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Outcome Measures

Primary Outcomes (1)

• 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.

  From Day 0 to Day 28

Secondary Outcomes (14)

• Number of Participants with Treatment Emergent Adverse Events (TEAEs), Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs)

  Up to Day 720

• Change From Baseline in CAR T-cell Toxicities

  Baseline, up to Day 720

• Pharmacokinetics (PK) Plasma Concentrations of Azer-cel

  Up to Day 720

• Pharmacodynamics (PD) Plasma Concentrations of Azer-cel

  Up to Day 720

• Time to Confirmed Disability Progression (CDP)

  Up to Day 720

Study Arms (1)

Azer-cel

EXPERIMENTAL

Participants will receive single dose of intravenous (IV) infusion of azer-cel at different dose levels, on Day 0 of the treatment period.

Drug: Azercabtagene zapreleucel (azer-cel)

Interventions

Azercabtagene zapreleucel (azer-cel) DRUG

IV infusion

Azer-cel

Eligibility Criteria

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

You may qualify if:

• Participants with Progressive forms of Multiple Sclerosis including Primary Progressive and Secondary Progressive MS.
• Participants must have discontinued disease modifying therapy (DMT) prior to signing the ICF and meet the following washout criteria prior to receiving lymphodepletion.

You may not qualify if:

• History of malignancy that has not been in remission for at least 2 years.
• Viral Screening
• Evidence of chronic active or history of hepatitis B virus (HBV).
• Seropositive for human immunodeficiency virus (HIV) antibody.
• History of bone marrow/hematopoietic stem cell or solid organ transplantation.
• Prior treatment with adoptive T-cell therapy or any gene therapy product directed at any target (e.g. CAR T-cell therapy).

Sponsors & Collaborators

• TG Therapeutics, Inc.: lead

Study Sites (8)

TG Therapeutics Investigational Trial Site

La Jolla, California, 92093, United States

RECRUITING

TG Therapeutics Investigational Trial Site

Lexington, Kentucky, 40506, United States

NOT YET RECRUITING

TG Therapeutics Investigational Trial Site

Ann Arbor, Michigan, 48019, United States

RECRUITING

TG Therapeutics Investigational Trial Site

Omaha, Nebraska, 68198, United States

RECRUITING

TG Therapeutics Investigational Trial Site

New York, New York, 10025, United States

RECRUITING

TG Therapeutics Investigational Trial Site

Rochester, New York, 14642, United States

RECRUITING

TG Therapeutics Investigational Trial Site

Cleveland, Ohio, 44195, United States

RECRUITING

TG Therapeutics Investigational Trial Site

Columbus, Ohio, 43210, United States

RECRUITING

MeSH Terms

Conditions

Multiple Sclerosis; Neuromyelitis Optica; Myasthenia Gravis; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Myelitis, Transverse; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Eye Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Polyradiculoneuropathy; Polyneuropathies; Peripheral Nervous System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

TG Therapeutics Clinical Support Team

CONTACT

1-877-575-8489; clinicalsupport@tgtxinc.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 1, 2024
• First Posted: November 8, 2024
• Study Start: May 6, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): January 1, 2029
• Last Updated: February 27, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)