KYSA-6: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Patients With Generalized Myasthenia Gravis

NCT06193889 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: KYSA-6KYV101-006
• Study Type: interventional
• Target: 66
• Locations: 3 countries
• Sites: 14

Brief Summary

A Study of the Anti-CD 19 Chimeric Antigen Receptor T Cell Therapy for Patients with Myasthenia Gravis

Conditions

Myasthenia Gravis; Generalized Myasthenia Gravis

Keywords

KYV-101; myasthenia gravis; autoimmune disease; anti-CD19 CAR-T Therapy; cellular therapy; MG; KYSA-6; KYV101-006

Outcome Measures

Primary Outcomes (4)

• Incidence and severity of adverse events (AEs) and laboratory abnormalities (Phase 2)

  2 years

• Efficacy of KYV-101 via Myasthenia Gravis Activities of Daily Living (MG-ADL) change from baseline (Phase 2)

  24 weeks

• Efficacy of KYV-101 via Myasthenia Gravis Activities of Daily Living (MG-ADL) change from baseline for KYV-101 Treatment Arm to Standard of Care Arm (Phase 3)

  24 weeks

• Efficacy of KYV-101 via Quantitative Myasthenia Gravis (QMG) change from baseline for KYV-101 Treatment arm to Standard of Care arm

  24 weeks

Secondary Outcomes (7)

• Efficacy of KYV-101 via Myasthenia Gravis Composite (MGC) score change from baseline for KYV-101 arm to Standard of Care arm (Phase 3)

  24 weeks

• Efficacy of KYV-101 via Percent change from baseline in anti acetylcholine receptors (anti-AChR) antibody levels for KYV-101 Treatment arm to Standard of Care arm (Phase 3)

  24 weeks

• Efficacy of KYV-101 via Percent change from baseline in anti muscle-specific tyrosine kinase (anti-MuSK) antibody levels for KYV-101 Treatment arm to Standard of Care arm (Phase 3)

  24 weeks

• Efficacy of KYV-101 via Proportion of patients with a ≥3 point improvement from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) for KYV-101 Treatment arm to Standard of Care arm (Phase 3)

  24 weeks

• Efficacy of KYV-101 via Proportion of patients with minimal symptom expression (MSE) for KYV-101 Treatment arm to Standard of Care arm (Phase 3)

  24 weeks

Study Arms (3)

KYV-101 CAR-T cells with lymphodepletion conditioning

EXPERIMENTAL

Phase 2: Dosing with KYV-101 CAR-T cells

Drug: Standard lymphodepletion regimen; Biological: KYV-101

KYV-101 Treatment

EXPERIMENTAL

Phase 3

Drug: Standard lymphodepletion regimen; Biological: KYV-101

Standard of Care

ACTIVE COMPARATOR

Phase 3 Optional crossover to receive KYV-101 Treatment after 24 weeks

Drug: Standard of Care Treatment; Drug: Standard lymphodepletion regimen; Biological: KYV-101

Interventions

Standard of Care Treatment DRUG

Standard of Care Medications Optional Crossover to receive KYV-101 treatment

Standard of Care

Standard lymphodepletion regimen DRUG

Standard lymphodepletion regimen

Also known as: Cyclophosphamide, Fludarabine

KYV-101 CAR-T cells with lymphodepletion conditioning; KYV-101 Treatment; Standard of Care

KYV-101 BIOLOGICAL

Anti-CD19 CAR-T cell therapy

KYV-101 CAR-T cells with lymphodepletion conditioning; KYV-101 Treatment; Standard of Care

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Presence of autoantibodies to AChR or MuSK at screening.
• Myasthenia Gravis Foundation of America (MGFA) Class II-IV
• MG-Activities of Daily Living (MG-ADL) total score of ≥6 at screening and confirmed at pre-dose baseline
• QMG total score of ≥11 at screening an confirmed at pre-dose baseline
• Failed treatment with 2 or more immunosuppressive/immunomodulatory therapies, or failed at least 1 immunosuppressive therapy and required chronic plasmapheresis, or IVIG (\>4 times/year over ≥12 months) to control symptoms
• On a stable dose of glucocorticoids and/or other immunotherapies for ≥1 month prior to screening. For patients treated with azathioprine, a stable dose for ≥2 months prior to screening is required
• No change in dose of acetylcholinesterase inhibitors for ≥2 weeks prior to screening
• No use of intravenous immune globulin (IVIG) or plasmapheresis (PLEX) within 4 weeks of screening or pre-dose baseline (unless this is part of their SOC treatment regimen)
• No use of rituximab (or any other anti-CD20 or CD19 monoclonal antibody) within 12 weeks prior to screening
• No use of FcRn inhibitors within 4 weeks prior to screening

You may not qualify if:

• Unable to washout or interrupt autoimmune disease therapy prior to apheresis
• Co-occurring neurological autoimmune disease (ie, Lambert-Eaton Myasthenic Syndrome) or any disease affecting the neuromuscular junction or muscle causing weakness (eg, myositis, myopathy, motor neuropathy)
• History of stroke (with residual sequalae and/or risk for recurrence), seizure (even if well controlled on antiepileptics), neurodegenerative disease, altered mental status (unexplained and/or recent/current), or uncontrolled/severe psychiatric disease
• Any serious and/or uncontrolled medical condition that, in the investigator's judgment, would cause unacceptable safety risk, interfere with study procedures or results, or compromise compliance with the protocol, including but not limited to, clinically significant cardiac or pulmonary disease
• History of primary immunodeficiency, organ or allogeneic bone marrow transplant, or splenectomy
• Active, uncontrolled, viral, bacterial, or systemic fungal infection or recent history of repeated infections
• Thymectomy \<12 months of screening or planned during the study
• Prior treatment with gene therapy product or cellular immunotherapy (eg, CAR T) requiring vector integration and directed at any target
• Patients requiring chronic anticoagulation therapy that cannot be discontinued for medical procedures

Sponsors & Collaborators

• Kyverna Therapeutics: lead

Study Sites (14)

University of California, Irvine

Orange, California, 92868, United States

RECRUITING

Stanford University Medical Center

Palo Alto, California, 94305, United States

RECRUITING

University of Miami

Miami, Florida, 33149, United States

RECRUITING

Indiana University Health

Indianapolis, Indiana, 46202, United States

RECRUITING

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, 19107, United States

RECRUITING

Houston Methodist Hospital

Houston, Texas, 77030, United States

RECRUITING

Intermountain Medical Center

Murray, Utah, 84107, United States

RECRUITING

Hospital Israelita Albert Einstein

São Paulo, Brazil

RECRUITING

Charite- Universitätsklinikum Berlin

Berlin, Germany

RECRUITING

Universitätsklinikum der Ruhr-Universität Bochum

Bochum, Germany

RECRUITING

Universitätsklinikum Hamburg-Eppendorf

Hamburg, Germany

RECRUITING

Medizinische Hochscule Hannover

Hanover, Germany

RECRUITING

Friedrich-Schiller-Universität Jena

Jena, Germany

RECRUITING

Universitätsklinik Magdeburg

Magdeburg, Germany

RECRUITING

Related Publications (1)

• Gilhus NE, Tzartos S, Evoli A, Palace J, Burns TM, Verschuuren JJGM. Myasthenia gravis. Nat Rev Dis Primers. 2019 May 2;5(1):30. doi: 10.1038/s41572-019-0079-y.

  PMID: 31048702 BACKGROUND

MeSH Terms

Conditions

Myasthenia Gravis; Autoimmune Diseases

Interventions

Cyclophosphamide; fludarabine

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Officials

• MD

  Kyverna Therapeutics, Inc.

  STUDY DIRECTOR

Central Study Contacts

Kyverna Therapeutics, Inc.

CONTACT

510-925-2484; Clinicaltrials@kyvernatx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 11, 2023
• First Posted: January 5, 2024
• Study Start: August 28, 2024
• Primary Completion (Estimated): September 1, 2027
• Study Completion (Estimated): September 1, 2028
• Last Updated: January 30, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

BR (1); DE (6); US (7)