Assessing the safety and effectiveness of Azer-cel for autoimmune disorders

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to evaluate the safety and clinical activity of Azercabtagene zapreleucel (azer-cel) in participants suffering from B-cell mediated autoimmune disorders, specifically focusing on progressive forms of multiple sclerosis. The primary goal is to determine the recommended phase 2 dose (RP2D) of the treatment. Participants will be required to have discontinued any disease-modifying therapy prior to enrollment and will undergo specific eligibility assessments to ensure safety. The study is interventional and is currently in Phase 1.

Why it matters

Who can join: the full rules

Inclusion Criteria:

PMS and RMS inclusion criteria:

1. Age 18 years to ≤60 years (inclusive) at screening.
2. Expanded Disability Status Scale (EDSS) score 3.0 - 6.5 (inclusive) at screening.
3. Diagnosis of primary progressive multiple sclerosis (PPMS), secondary progressive multiple sclerosis (non-active or active), or Relapsing MS (RMS).
4. Documented evidence of disability progression independent of relapse (PIRA) at any point over the 12 months prior to the screening visit.
5. Willingness and ability to undergo lumbar puncture (LP) for cerebrospinal fluid (CSF) collection at protocol-specified timepoints, with no contraindications to the procedure in the opinion of the Investigator.

NMOSD inclusion criteria:

1. Between age 18 and ≤ 75 years, inclusive at the time of signing the informed consent.
2. 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).
3. Diagnosis of anti-aquaporin-4 immunoglobulin G (AQP4-IgG) seropositive at screening (verified by the allocated central laboratory) and Neuromyelitis Optica Spectrum Disorder (NMOSD).
4. Must meet the appropriate NMOSD treatment washout criteria prior to receiving lymphodepletion.

MG Inclusion criteria:

1. Age ≥18 and ≤75 years of age at the time of signing the informed consent.
2. Diagnosed with gMG at least 1 year prior to the date of signing the informed consent.
3. Confirmation of MG Diagnosis:

   1. Positive serologic test for anti-acetylcholine receptor (AChR) antibodies or anti-muscle-specific kinase (MuSK) antibodies confirmed at screening AND
   2. 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).
      * Demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician.
4. MG activities of daily living (MG-ADL) score ≥6 at screening.

CIDP Inclusion criteria

1. Age ≥18 and ≤75 years of age at the time of signing the informed consent.
2. Participant must have either typical CIDP, or one of the following two CIDP variants: motor CIDP, multifocal CIDP (also known as Lewis Sumner Syndrome). Separately, Autoimmune Nodopathy (AN) participants with anti-CNTN1 or anti-NF155/anti-NF186, as confirmed by serological testing, may be included.
3. CIDP Disease Activity Status (CDAS): CDAS score ≥3 at screening.
4. INCAT Disability Score: INCAT disability score ≥4 to ≤9 score at screening.

General Exclusion Criteria:

1. History of malignancy that has not been in remission for at least 2 years.
2. Active venous thromboembolism (e.g., deep vein thrombosis or pulmonary embolism), clinically significant coagulopathy (e.g., disseminated intravascular coagulation), or current therapeutic anticoagulation (e.g., warfarin).
3. Viral and Tuberculosis (TB) Screening

   1. Evidence of chronic active or history of hepatitis B virus (HBV).
   2. Seropositive for human immunodeficiency virus (HIV) antibody.
4. History of bone marrow/hematopoietic stem cell or solid organ transplantation.
5. Prior treatment with any gene therapy product directed at any target (e.g. CAR T-cell therapy), with the exception of azer-cel.

Note: Other protocol-specified Inclusion/Exclusion criteria may apply.

Where it's running

• TG Therapeutics Investigational Trial Site
  La Jolla, California, United States Recruiting
• TG Therapeutics Investigational Trial Site
  Ann Arbor, Michigan, United States Recruiting
• TG Therapeutics Investigational Trial Site
  Omaha, Nebraska, United States Recruiting
• TG Therapeutics Investigational Trial Site
  New York, New York, United States Recruiting
• TG Therapeutics Investigational Trial Site
  Rochester, New York, United States Recruiting
• TG Therapeutics Investigational Trial Site
  Cleveland, Ohio, United States Recruiting

Study contacts

• Study coordinator: TG Therapeutics Clinical Support Team
• Email: clinicalsupport@tgtxinc.com
• Phone: 1-877-575-8489

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Long-term safety follow-up after Azer-Cel in B-cell autoimmune disorders

  This observational study follows people who already received Azer-Cel to see if any long-term safety events occur over up to 15 years.

  Recruiting TG Therapeutics, Inc. · Morrisville, North Carolina · NCT07577583

• Anti-BCMA-CD19 CAR-T therapy for relapsed or refractory IgG4-related disease

  This trial will test a personalized CAR-T cell therapy that targets BCMA and CD19 in adults (18–75) whose IgG4-related disease has come back or not responded to standard treatments like steroids or rituximab.

  Recruiting Phase 2 Chinese PLA General Hospital · Beijing, Beijing Municipality · NCT07148791

• Ublituximab in people with schizophrenia

  This study tests whether ublituximab can help reduce symptoms in adults with treatment‑resistant schizophrenia who are already taking standard antipsychotic therapy.

  Recruiting at 2 sites Phase 2 TG Therapeutics, Inc. · Little Rock, Arkansas and 1 other locations · NCT07680946

• Ublituximab maintenance after efgartigimod response in myasthenia gravis

  This study tests whether giving ublituximab as maintenance after a positive response to efgartigimod helps adults with myasthenia gravis.

  Recruiting at 7 sites Phase 2 TG Therapeutics, Inc. · Colorado Springs, Colorado and 6 other locations · NCT07673744

• Ublituximab in MS: how injection site and delivery method affect absorption

  This study tests how different SC injection sites and a prefilled pen delivery compare in how ublituximab behaves in the body for people with relapsing MS.

  Recruiting at 44 sites Phase 2 TG Therapeutics, Inc. · Bihać and 43 other locations · NCT07503873

• Ublituximab in Pediatric Relapsing MS: See if a B-cell therapy works in kids

  This study tests whether ublituximab can work in children and teens with relapsing MS and compares it to fingolimod in eligible participants.

  Recruiting at 5 sites Phase 2/3 TG Therapeutics, Inc. · Salt Lake City, Utah and 5 other locations · NCT07220252

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.