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The main objective of the study is to determine the recommended phase 2 dose (RP2D) of Azercabtagene zapreleucel (azer-cel).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Azer-cel | Experimental | Participants will receive single dose of intravenous (IV) infusion of azer-cel at different dose levels, on Day 0 of the treatment period. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Azercabtagene zapreleucel (azer-cel) | Drug | IV infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | From Day 0 to Day 28 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
PMS and RMS inclusion criteria:
NMOSD inclusion criteria:
MG Inclusion criteria:
Age ≥18 and ≤70 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):
MG activities of daily living (MG-ADL) score ≥6 at screening.
CIDP Inclusion criteria
General Exclusion Criteria:
History of malignancy that has not been in remission for at least 2 years.
Viral Screening
History of bone marrow/hematopoietic stem cell or solid organ transplantation.
Prior treatment with adoptive T-cell therapy or any gene therapy product directed at any target (e.g. CAR T-cell therapy).
Note: Other protocol-specified Inclusion/Exclusion criteria may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| TG Therapeutics Clinical Support Team | Contact | 1-877-575-8489 | clinicalsupport@tgtxinc.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| TG Therapeutics Investigational Trial Site | Recruiting | La Jolla | California | 92093 | United States | |
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| 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 |
| Change From Baseline in Modified Rankin Scale (mRS) | 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) | Baseline, up to Day 720 |
| Change from Baseline in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score | Baseline, up to Day 720 |
| Change from Baseline in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r) | Baseline, up to Day 720 |
| Time to Relapse, as Defined by a 1 Point Change in Adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT) | Baseline, up to Day 720 |
| Change From Baseline in Inflammatory Neuropathy Cause and Treatment (INCAT) Score | Baseline, up to Day 720 |
| TG Therapeutics Investigational Trial Site |
| Recruiting |
| Ann Arbor |
| Michigan |
| 48019 |
| United States |
| TG Therapeutics Investigational Trial Site | Recruiting | Omaha | Nebraska | 68198 | United States |
| TG Therapeutics Investigational Trial Site | Recruiting | New York | New York | 10025 | United States |
| TG Therapeutics Investigational Trial Site | Recruiting | Rochester | New York | 14642 | United States |
| TG Therapeutics Investigational Trial Site | Recruiting | Cleveland | Ohio | 44195 | United States |
| TG Therapeutics Investigational Trial Site | Recruiting | Columbus | Ohio | 43210 | United States |
| TG Therapeutics Investigational Trial Site | Recruiting | Milwaukee | Wisconsin | 53226 | United States |
| ID | Term |
|---|---|
| D009103 | Multiple Sclerosis |
| D009471 | Neuromyelitis Optica |
| D009157 | Myasthenia Gravis |
| D020277 | Polyradiculoneuropathy, Chronic Inflammatory Demyelinating |
| ID | Term |
|---|---|
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D003711 | Demyelinating Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D009188 | Myelitis, Transverse |
| D009902 | Optic Neuritis |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D005128 | Eye Diseases |
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D011129 | Polyradiculoneuropathy |
| D011115 | Polyneuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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