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

Phase 1

Recruiting

• Conditions: B-cell Mediated Autoimmune Disorders
• Interventions: Drug: Azercabtagene zapreleucel (azer-cel)

• Registration Number: NCT06680037
• Lead Sponsor: TG Therapeutics, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-11-01
• Study First Submitted QC: 2024-11-07
• Study First Posted: 2024-11-08
• Study Start: 2025-01-15
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-30
• Last Update Posted: 2025-05-04
• Primary Completion: 2026-12-01
• Study Completion: 2029-01-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

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

Exclusion Criteria

1. History of malignancy that has not been in remission for at least 2 years.

2. Viral Screening

   1. Evidence of chronic active or history of hepatitis B virus (HBV).
   2. Seropositive for human immunodeficiency virus (HIV) antibody.

3. History of bone marrow/hematopoietic stem cell or solid organ transplantation.

4. Prior treatment with adoptive T-cell therapy or any gene therapy product directed at any target (e.g. CAR T-cell therapy).

Note: Please note, other protocol specified Inclusion/Exclusion criteria may apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Number of Participants with Dose-Limiting Toxicities (DLTs)	From Day 0 to Day 28	DLT will be determined using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

Secondary Outcome Measures

Name	Time	Method
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
Time to Confirmed Disability Improvement (CDI)	Up to Day 720
Change From Baseline in Brain MRI Gadolinium Enhancing T1, New or Enlarging Hyperintense T2	Baseline, up to Day 720
Change From Baseline in Whole Brain Atrophy	Baseline, up to Day 720

Trial Locations

Locations (1)

TG Therapeutics Investigational Trial Site; 🇺🇸 Omaha, Nebraska, United States