Azer-cel for Autoimmune Disease

Recruiting at 6 trial locations

Overseen By TG Therapeutics Clinical Support Team

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: TG Therapeutics, Inc.

Disqualifiers: Cancer, Hepatitis B, HIV, Transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 4 weeks

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called azer-cel (Azercabtagene zapreleucel) for individuals with specific types of multiple sclerosis (MS), namely primary progressive or secondary progressive MS. The trial aims to determine the optimal dose for future studies. Participants will receive a single dose through an IV on the first day of treatment. Ideal candidates for this trial are those who have discontinued other MS treatments and do not have certain health issues, such as a recent history of cancer or specific viral infections. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, providing participants the opportunity to be among the first to receive this new therapy.

Will I have to stop taking my current medications?

Yes, you will need to stop taking your current disease modifying therapy (DMT) before joining the trial, and there is a specific washout period (time without taking certain medications) required before receiving treatment.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research shows that azercabtagene zapreleucel, or azer-cel, is under testing for use in autoimmune diseases. This early-phase study primarily aims to determine the appropriate dose for future research. At this stage, limited safety information is available. However, similar treatments have shown promise in related conditions.

Early-phase trials ensure treatments are safe for people, checking for side effects and patient tolerance. Since azer-cel is being tested in individuals with autoimmune diseases, researchers closely monitor participants for any negative reactions.

Specific results from this trial are not yet available, but the study is a step toward understanding the safety and effectiveness of azer-cel for autoimmune conditions. If successful, it could lead to new treatment options in the future. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Azer-cel is unique because it uses azercabtagene zapreleucel, a CAR T-cell therapy, which is quite different from the usual immunosuppressants or biologics used to treat autoimmune diseases. Traditional treatments often aim to suppress the immune system broadly, which can lead to various side effects. However, azer-cel is designed to specifically target and modulate immune cells that are causing the disease, potentially offering a more precise treatment. Researchers are excited because this targeted approach could lead to better control of the disease with fewer side effects, offering new hope for patients who don't respond well to existing therapies.

What evidence suggests that azer-cel might be an effective treatment for autoimmune disease?

Studies have shown that azercabtagene zapreleucel (azer-cel) holds promise for treating autoimmune diseases. Research indicates that azer-cel, a therapy using modified immune cells, achieved impressive results in patients with diffuse large B-cell lymphoma, even in those unresponsive to other treatments. Additionally, this therapy was successfully used in a person with multiple sclerosis, marking an important step forward in treating autoimmune conditions. The treatment changes immune cells to target and fight specific harmful cells in the body. Overall, early findings suggest that azer-cel could effectively manage autoimmune diseases by harnessing the body's own immune system. ^{2 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with progressive forms of Multiple Sclerosis, including Primary Progressive and Secondary Progressive MS. Participants must have stopped any disease modifying therapy before joining the study and meet specific criteria for medication washout.

Inclusion Criteria

My condition is Progressive Multiple Sclerosis.

I have stopped all disease modifying treatments as required.

Exclusion Criteria

Viral Screening: Evidence of chronic active or history of hepatitis B virus (HBV), Seropositive for human immunodeficiency virus (HIV) antibody

I have not had treatments like CAR T-cell therapy.

I have had cancer that was not in remission for at least 2 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of intravenous (IV) infusion of azer-cel at different dose levels

4 weeks

1 visit (in-person) on Day 0

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Azercabtagene zapreleucel (azer-cel)

Trial Overview The trial is testing Azercabtagene zapreleucel (azer-cel) to find out the safest and most effective dose for treating B-cell mediated autoimmune disorders, specifically focusing on its use in Multiple Sclerosis.

How Is the Trial Designed?

Group I: Azer-cel Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

TG Therapeutics, Inc.

Lead Sponsor

Trials

41

Recruited

6,900+

Citations

1. cgtlive.com cgtlive.com/view/allogeneic-car-t-azer-cel-achieves-complete-responses-patients-diffuse-large-b-cell-lymphoma

Allogeneic CAR-T Azer-Cel Achieves Complete ... We are pleased with the results, which suggest improved outcomes in patients, and we look forward to amassing more data using this dosing ...

2. insideprecisionmedicine.com insideprecisionmedicine.com/topics/oncology/take-2-off-the-shelf-car-t-shines-in-relapsed-refractory-lymphoma-patients/

Take II: Off-the-Shelf CAR T Shines in Relapsed/Refractory ... The allogeneic Azer-cel showed impressive response rates in a Phase Ib with DLBCL patients who had failed autologous CAR T cell therapy treatments.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06680037

NCT06680037 | A Study to Assess the Safety and ... A Study to Assess the Safety and Clinical Activity of Azer-cel in Participants With B-cell Mediated Autoimmune Disorders. ClinicalTrials.gov ID NCT06680037.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12313632/

CAR T-cells meet autoimmune neurological diseases This review will examine recent advancements in CAR T-cell therapy for autoimmune disorders, highlighting how CAR T cells can be engineered to ...

5. multiplesclerosisnewstoday.com multiplesclerosisnewstoday.com/news-posts/2025/08/11/first-ms-patient-treated-off-shelf-car-t-cell-therapy/

In first, MS patient is treated with 'off-the-shelf' CAR T-cell... A person with multiple sclerosis (MS) has for the first time been treated with an “off-the-shelf” CAR T-cell therapy called azercabtagene ...

6. allclinicaltrials.com allclinicaltrials.com/study/NCT06680037

Azer-cel - A Study to Assess the Safety and ... The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel). .

7. trialx.com trialx.com/clinical-trials/listings/311866/a-study-to-assess-the-safety-and-clinical-activity-of-azer-cel-in-participants-with-b-cell-mediated-autoimmune-disorders/

A Study to Assess the Safety and Clinical Activity of Azer ... Overview. The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel).

8. clinicaltrial.be clinicaltrial.be/en/details/458148?per_page=20&only_recruiting=0&only_eligible=0&only_active=0

A Study to Assess the Safety and Clinical Activity of Aze... Trial description. The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel).