KITE-363 treatment for relapsed or refractory autoimmune neurologic diseases

A Phase 1 Open-label, Multiregional, Multicenter, Basket Study Evaluating the Safety and Efficacy of KITE-363, an Autologous Anti-CD19/CD20 CAR T-cell Therapy in Participants With Relapsed/Refractory Autoimmune Neurologic Diseases

This study will test whether KITE-363 is safe and can help people with relapsed or treatment-resistant autoimmune neurologic diseases such as MS, CIDP, and myasthenia gravis.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 52 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Gilead Sciences (Industry-sponsored)
• Drugs / interventions: ocrelizumab, rituximab
• Locations: 3 sites (Salt Lake City, Utah and 2 other locations)
• Trial ID: NCT07304154 on ClinicalTrials.gov

What this trial studies

This is a Phase 1a/1b interventional trial testing KITE-363 in participants with relapsed or refractory autoimmune neurologic diseases. The study uses a dose‑escalation design in Phase 1a to define safety, tolerability, and a recommended dose for Phase 1b, and then examines preliminary efficacy at the selected dose in Phase 1b. Participants receive KITE-363 with preparative lymphodepletion including fludarabine and cyclophosphamide as part of the treatment regimen. The trial is multi-center with sites in the United States and Australia and focuses on clinical safety endpoints and early signals of benefit.

Who should consider this trial

Good fit: Adults with relapsed or treatment‑resistant MS, CIDP, or myasthenia gravis who meet diagnostic criteria, have had inadequate response or intolerance to prior therapies, and who meet health and reproductive eligibility requirements are the intended candidates.

Not a fit: Patients with stable disease not needing aggressive therapy, active uncontrolled infections, serious comorbidities, or who cannot tolerate immunosuppressive preparative therapy are unlikely to benefit or may be ineligible.

Why it matters

Potential benefit: If successful, KITE-363 could offer a new therapeutic option that reduces disease activity or symptoms in patients with relapsed or treatment‑resistant autoimmune neurologic conditions.

How similar studies have performed: Related cellular and B‑cell‑targeting immunotherapies have shown promising early results in some autoimmune disorders, but applying these approaches to autoimmune neurologic diseases remains novel and experimental.

Eligibility criteria

Key Inclusion Criteria:

* Reproductive status-related eligibility and contraception requirements:

  * Participants must agree to use protocol-specified method(s) of contraception where applicable

Inclusion Criteria for multiple sclerosis (MS):

MS (Relapsing and progressive forms):

* Diagnosed with MS according to the 2017 revision of the McDonald diagnostic criteria

Relapsing forms of MS (relapsing-remitting multiple sclerosis (RRMS), active secondary-progressive multiple sclerosis (aSPMS)):

* Inadequate response to previous therapies is defined as evidence of breakthrough disease activity within 12 months prior to screening while on high efficacy disease-modifying therapy (DMT) OR Inadequate response to previous therapies defined as intolerance to ≥ 2 DMTs due to side effects prohibiting the chronic use of the DMT.
* Expanded Disability Status Scale (EDSS) 0 to 5.5

Progressive forms of MS (primary-progressive multiple sclerosis (PPMS) and non-active secondary-progressive multiple sclerosis (naSPMS)):

* Inadequate response to previous therapies is defined as evidence of disease progression within 12 months prior to screening despite standard of care therapy for naSPMS or despite ocrelizumab, where available, for PPMS
* Absence of clinical relapses for at least 24 months
* No evidence of Gadolinium enhancing (GadE+) on magnetic resonance imaging (MRI) brain at screening or baseline
* EDSS of 3 to 6.5 who are ambulatory

Inclusion Criteria for myasthenia gravis (MG):

* Documentation of autoantibodies against acetylcholine receptor (AChR), muscle-specific kinase (MuSK), or low-density lipoprotein receptor-related protein 4 (LRP4)
* Diagnosis of MG with generalized weakness meeting criteria as defined by the Myasthenia Gravis Foundation of American (MGFA) classification of II- IV at screening
* Myasthenia Gravis Activities of Daily Living (MG-ADL) score ≥ 6 (\> 50% of the total score due to non-ocular symptoms)
* Quantitative Myasthenia Gravis (QMG) score ≥ 10
* Inadequate response to previous therapies while taking at least 2 classes of immunosuppressants (ie, steroids, azathioprine (AZA), mycophenolate mofetil (MMF), intravenous immunoglobulin (IVIg), biologics (eg, rituximab, anti-neonatal fragment crystallizable (Fc) receptor (FcRN) class, and anti-complement class))
* Thymectomy allowed if completed ≥ 12 months prior to screening

Inclusion Criteria for chronic inflammatory demyelinating polyneuropathy (CIDP):

* Probable or definite CIDP as defined by the 2010 European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria, relapsing or progressive forms
* CIDP Disease Activity Status (CDAS) score ≥ 3 at screening
* Inflammatory neuropathy cause and treatment (INCAT) score ≥ 3
* Inadequate response to previous therapies despite standard of care therapy (ie, steroids, IVIg, subcutaneous immunoglobulin (SCIg), plasmapheresis exchange (PLEX), rituximab, or anti FcRN) OR Unable to tolerate standard of care due to side effects with ongoing disease activity
* Except for nodal/paranodal CIDP, historical documentation of objective improvement in the past 24 months while on IVIg, SCIg, PLEX, or anti-FcRN OR Historical documentation of objective disease worsening in the past 24 months when IVIg, SCIg, PLEX, or anti-FcRN has been reduced or interrupted

Key Exclusion Criteria:

* History or presence of central nervous system (CNS) or peripheral nervous system disorders before enrollment that may impact cognition, strength, or cause weakness
* History of autologous or allogeneic stem cell transplant and/or organ transplant

Exclusion Criteria for MS:

* Cohort 1 or 2; inability to complete 9-hole Peg Test (9-HPT) in \< 240 seconds and Timed 25 foot Walk (T25FW) \< 150 seconds
* History of hypersensitivity to parenteral administration of gadolinium-based contrast agents
* Any renal condition that would preclude the administration of gadolinium (for the relapsing forms of MS and progressive forms of MS)
* Any contraindication to lumbar puncture (LP) (for the relapsing forms of MS and progressive forms of MS)

Exclusion Criteria for MG:

* Current myasthenic crisis not effectively controlled within 2 weeks before enrollment
* Thymectomy performed within 12 months of baseline

Exclusion Criteria for CIDP:

* Pure sensory CIDP and focal CIDP
* Polyneuropathy of other causes

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Where this trial is running

Salt Lake City, Utah and 2 other locations

• LDS Hospital - Intermountain Health — Salt Lake City, Utah, United States (Recruiting)
• Fred Hutchinson Cancer Center — Seattle, Washington, United States (Recruiting)
• Concord Repatriation General Hospital — Sydney, New South Wales, Australia (Recruiting)

Study contacts

• Study coordinator: Medical Information

Conditions Chronic Inflammatory Demyelinating Polyneuropathy; Myasthenia Gravis; Multiple Sclerosis