NCT06680037|RecruitingPhase 1DMCFDA Drug

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

A Phase 1, Open-label Study to Evaluate the Safety and Clinical Activity of Azercabtagene Zapreleucel in Participants With B-cell Mediated Autoimmune Disorders

TG Therapeutics, Inc.ClinicalTrials.gov

Compare

1 other identifier

TG-Azercel-101

Study Type

interventional

Target

100

Locations

1 country

Sites

Timeline

RegisteredNov 2024

StartedMay 2025

CompletionJan 2030

Brief Summary

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

Trial Health

On Track

Trial Health Score

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

Enrollment

100

participants targeted

Target at P75+ for phase_1

Timeline

42mo left

Started May 2025

Longer than P75 for phase_1

Geographic Reach

1 country

8 active sites

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

4.7 years study duration

Study Progress27%

May 2025Jan 2030

First Submitted

Initial submission to the registry

November 1, 2024

Completed

7 days until next milestone

First Posted

Study publicly available on registry

November 8, 2024

Completed

6 months until next milestone

Study Start

First participant enrolled

May 6, 2025

Completed

2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2027

Expected

2.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2030

Last Updated

July 20, 2026

Status Verified

July 1, 2026

Enrollment Period

2.6 years

First QC Date

November 1, 2024

Last Update Submit

July 16, 2026

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
+9 more secondary outcomes

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

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

Age 18 years to ≤60 years (inclusive) at screening.
Expanded Disability Status Scale (EDSS) score 3.0 - 6.5 (inclusive) at screening.
Diagnosis of primary progressive multiple sclerosis (PPMS), secondary progressive multiple sclerosis (non-active or active), or Relapsing MS (RMS).
Documented evidence of disability progression independent of relapse (PIRA) at any point over the 12 months prior to the screening visit.
Between age 18 and 65 years, inclusive at the time of signing the informed consent.
EDSS score between 2.0 and 7.0 at screening, inclusive (for higher EDSS, the Investigator must assess that the participant is reasonably able to participate in the study).
Diagnosis of anti-aquaporin-4 immunoglobulin G (AQP4-IgG) seropositive at screening (verified by the allocated central laboratory) and Neuromyelitis Optica Spectrum Disorder (NMOSD).
Must meet the appropriate NMOSD treatment washout criteria prior to receiving lymphodepletion.
Age ≥18 and ≤70 years of age at the time of signing the informed consent.
Diagnosed with gMG at least 1 year prior to the date of signing the informed consent.
Confirmation of MG Diagnosis:
Positive serologic test for anti-acetylcholine receptor (AChR) antibodies or anti-muscle-specific kinase (MuSK) antibodies confirmed at screening AND
One of the following (either historical or during screening):
Abnormal neuromuscular transmission test demonstrated by single-fiber electromyography or repetitive nerve stimulation.
Positive anticholinesterase test (e.g., edrophonium chloride test).
+6 more criteria

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).

Contact the study team to confirm eligibility.