B-cell Mediated Autoimmune DisordersPhase 1NCT06680037
A Study to Assess the Safety and Clinical Activity of Azer-cel in Participants With B-cell Mediated Autoimmune Disorders
A Phase 1 study testing Azercabtagene zapreleucel (azer-cel) in people with B-cell Mediated Autoimmune Disorders, run by TG Therapeutics, Inc. at 8 sites in United States.
Trial summary
The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- B-cell Mediated Autoimmune Disorders
- What is being tested?
- Azercabtagene zapreleucel (azer-cel)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 8 sitesUnited States
- Study dates
- 6 May 2025to 1 Jan 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- PMS and RMS inclusion criteria:
- 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.
- 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.
Show all 35Show fewer
- NMOSD inclusion criteria:
- Between age 18 and ≤ 75 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.
- MG Inclusion criteria:
- Age ≥18 and ≤75 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).
- Demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician.
- MG activities of daily living (MG-ADL) score ≥6 at screening.
- CIDP Inclusion criteria
- Age ≥18 and ≤75 years of age at the time of signing the informed consent.
- 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.
- CIDP Disease Activity Status (CDAS): CDAS score ≥3 at screening.
- INCAT Disability Score: INCAT disability score ≥4 to ≤9 score at screening.
- General Exclusion Criteria:
- History of malignancy that has not been in remission for at least 2 years.
- 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).
- Viral and Tuberculosis (TB) 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 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.
Who cannot join
None listed.
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
United States8 sites, 8 recruiting
- Recruiting
TG Therapeutics Investigational Trial Site
Ann Arbor, Michigan, 48019
- Recruiting
TG Therapeutics Investigational Trial Site
Cleveland, Ohio, 44195
- Recruiting
TG Therapeutics Investigational Trial Site
Columbus, Ohio, 43210
- Recruiting
TG Therapeutics Investigational Trial Site
La Jolla, California, 92093
- Recruiting
TG Therapeutics Investigational Trial Site
Milwaukee, Wisconsin, 53226
- Recruiting
TG Therapeutics Investigational Trial Site
New York, New York, 10025
- Recruiting
TG Therapeutics Investigational Trial Site
Omaha, Nebraska, 68198
- Recruiting
TG Therapeutics Investigational Trial Site
Rochester, New York, 14642
About this study
Official title: A Phase 1, Open-label Study to Evaluate the Safety and Clinical Activity of Azercabtagene Zapreleucel in Participants With B-cell Mediated Autoimmune Disorders
The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel).
- Conditions
- B-cell Mediated Autoimmune Disorders
- Being tested
- Azercabtagene zapreleucel (azer-cel)
- 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)
- Medical subject headings
- Multiple Sclerosis, Neuromyelitis Optica, Myasthenia Gravis, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Azercabtagene zapreleucel (azer-cel)Drug
IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Azer-cel | Experimental | Participants will receive a single dose of intravenous (IV) infusion of azer-cel at different dose levels, on Day 0 of the treatment period. Receives: Drug: Azercabtagene zapreleucel (azer-cel) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
Measured: Up to Day 720
Change From Baseline in CAR T-cell Toxicities
Measured: Baseline, up to Day 720
Pharmacokinetics (PK) Plasma Concentrations of Azer-cel
Measured: Up to Day 720
Pharmacodynamics (PD) Plasma Concentrations of Azer-cel
Measured: Up to Day 720
Time to Confirmed Disability Progression (CDP)
Measured: Up to Day 720
Time to Confirmed Disability Improvement (CDI)
Measured: Up to Day 720
Change From Baseline in Brain MRI Gadolinium Enhancing T1, New or Enlarging Hyperintense T2
Measured: Baseline, up to Day 720
Change From Baseline in Whole Brain Atrophy
Measured: Baseline, up to Day 720
Change From Baseline in Modified Rankin Scale (mRS)
Measured: Baseline, up to Day 720
Change From Baseline in Visual Acuity on Landolt C Broken Ring Chart (Low Contrast Visual Acuity [LCVA]) and High Contrast Visual Acuity (HCVA)
Measured: Baseline, up to Day 720
Change from Baseline in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score
Measured: Baseline, up to Day 720
Change from Baseline in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r)
Measured: Baseline, up to Day 720
Time to Relapse, as Defined by a 1 Point Change in Adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT)
Measured: Baseline, up to Day 720
Change From Baseline in Inflammatory Neuropathy Cause and Treatment (INCAT) Score
Measured: Baseline, up to Day 720
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2024
- Study start6 May 2025
- Primary completion1 Dec 2026 (estimated)
- Study completion1 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for B-cell Mediated Autoimmune DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- TG Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06680037
- Sponsor's protocol ID
- TG-Azercel-101
- Status last verified
- Sep 2026
- First submitted
- 1 Nov 2024
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-label Study to Evaluate the Safety and Clinical Activity of Azercabtagene Zapreleucel in Participants With B-cell Mediated Autoimmune Disorders
- ClinicalTrials.gov ID
- NCT06680037
- Acronym / sponsor ID
- TG-Azercel-101
- 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)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- TG Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 6 May 2025 (actual)
- Primary completion
- 1 Dec 2026 (estimated)
- Study completion
- 1 Jan 2029 (estimated)
- First posted
- 8 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Azer-cel | Experimental | Drug: Azercabtagene zapreleucel (azer-cel)Participants will receive a single dose of intravenous (IV) infusion of azer-cel at different dose levels, on Day 0 of the treatment period. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (35 listed)
- 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.
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Azercabtagene zapreleucel (azer-cel)IV infusion | Drug | Azer-cel |
Primary outcome
Number of Participants with Dose-Limiting Toxicities (DLTs)
Time frame: 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.
14 secondary outcomes
Number of Participants with Treatment Emergent Adverse Events (TEAEs), Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Events of Special Interest (AESIs)
Time frame: Up to Day 720
Change From Baseline in CAR T-cell Toxicities
Time frame: Baseline, up to Day 720
Pharmacokinetics (PK) Plasma Concentrations of Azer-cel
Time frame: Up to Day 720
Pharmacodynamics (PD) Plasma Concentrations of Azer-cel
Time frame: Up to Day 720
Time to Confirmed Disability Progression (CDP)
Time frame: Up to Day 720
Time to Confirmed Disability Improvement (CDI)
Time frame: Up to Day 720
Change From Baseline in Brain MRI Gadolinium Enhancing T1, New or Enlarging Hyperintense T2
Time frame: Baseline, up to Day 720
Change From Baseline in Whole Brain Atrophy
Time frame: Baseline, up to Day 720
Change From Baseline in Modified Rankin Scale (mRS)
Time frame: Baseline, up to Day 720
Change From Baseline in Visual Acuity on Landolt C Broken Ring Chart (Low Contrast Visual Acuity [LCVA]) and High Contrast Visual Acuity (HCVA)
Time frame: Baseline, up to Day 720
Change from Baseline in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score
Time frame: Baseline, up to Day 720
Change from Baseline in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r)
Time frame: Baseline, up to Day 720
Time to Relapse, as Defined by a 1 Point Change in Adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT)
Time frame: Baseline, up to Day 720
Change From Baseline in Inflammatory Neuropathy Cause and Treatment (INCAT) Score
Time frame: Baseline, up to Day 720
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Azercabtagene zapreleucel (azer-cel) in people with B-cell Mediated Autoimmune Disorders, run by TG Therapeutics, Inc. at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 35 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact TG Therapeutics Clinical Support Team (1-877-575-8489, clinicalsupport@tgtxinc.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Dose-Limiting Toxicities (DLTs) (time frame: From Day 0 to Day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2026; AzTrials last refreshed its copy on 26 Oct 2026.