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
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests the safety and effects of azer-cel and aims to choose a dose for the next stage of testing. It is for adults with certain autoimmune disorders, in which the immune system attacks the body.
The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel).
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- For multiple sclerosis (MS): Be age 18–60 and have primary progressive, secondary progressive (active or not active), or relapsing MS.
- For MS: Have an EDSS disability score of 3.0–6.5 and records showing worsening disability apart from a relapse in the past 12 months. Be willing and safely able to have spinal taps to collect fluid around the brain and spinal cord.
- For neuromyelitis optica spectrum disorder (NMOSD): Be age 18–75, have this disorder, and test positive for AQP4-IgG antibodies at screening through the study's central lab.
- For NMOSD: Have an EDSS disability score of 2.0–7.0. For higher scores, the study doctor must judge that you can reasonably take part. Meet the required waiting periods after prior treatments before receiving treatment that lowers immune cell levels.
- For generalized myasthenia gravis (gMG), a muscle weakness disorder: Be age 18–75 and have been diagnosed at least 1 year before consent. Test positive for AChR or MuSK antibodies at screening.
- For gMG: Have an abnormal nerve-to-muscle signal test, a positive anticholinesterase drug test, or doctor-confirmed improvement with oral cholinesterase inhibitors. Have an MG-ADL score of at least 6, which measures effects on daily activities.
- For chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder: Be age 18–75 and have typical, motor, or multifocal CIDP. People with autoimmune nodopathy, another nerve disorder, may also qualify if blood tests show anti-CNTN1 or anti-NF155/anti-NF186 antibodies.
- For CIDP: Have a CDAS disease activity score of at least 3 and an INCAT disability score of 4–9.
Who can't join
- A history of cancer that has not been in remission for at least 2 years.
- An active blood clot in a vein or lung, a serious blood-clotting disorder, or current use of treatment-dose blood thinners, such as warfarin.
- Ongoing active hepatitis B or a history of hepatitis B.
- A positive blood test for HIV antibodies.
- A past bone marrow, blood-forming stem cell, or solid organ transplant.
- Past gene therapy, including CAR T-cell therapy, other than azer-cel.
- Other study entry and exclusion rules may apply.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
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.
Study sites (8)
CA
- TG Therapeutics Investigational Trial Site · La Jolla, CA 92093More trials in La Jolla
MI
- TG Therapeutics Investigational Trial Site · Ann Arbor, MI 48019More trials in Ann Arbor
NE
- TG Therapeutics Investigational Trial Site · Omaha, NE 68198More trials in Omaha
NY
- TG Therapeutics Investigational Trial Site · New York, NY 10025More trials in New York
- TG Therapeutics Investigational Trial Site · Rochester, NY 14642More trials in Rochester
OH
- TG Therapeutics Investigational Trial Site · Cleveland, OH 44195More trials in Cleveland
- TG Therapeutics Investigational Trial Site · Columbus, OH 43210More trials in Columbus
WI
- TG Therapeutics Investigational Trial Site · Milwaukee, WI 53226More trials in Milwaukee