Status:

ACTIVE_NOT_RECRUITING

KYSA-8: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects With Treatment Refractory Stiff Person Syndrome

Lead Sponsor:

Kyverna Therapeutics

Conditions:

Stiff-Person Syndrome

SPS

Eligibility:

All Genders

18-75 years

Phase:

PHASE2

Brief Summary

A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy for Subjects with Treatment Refractory Stiff Person Syndrome

Detailed Description

Stiff person syndrome (SPS) is a rare progressive immune-mediated disorder of the central nervous system (CNS) that is characterized by progressive rigidity and painful spasms of predominantly axial a...

Eligibility Criteria

Inclusion

  • Key
  • Subject must have been diagnosed SPS per the following criteria:
  • Rigidity of limb and axial (trunk) muscles prominent in the abdominal and thoracolumbar paraspinal areas and making bending difficult
  • Clinical or electrophysiological evidence of continuous contraction of agonist and antagonist muscles
  • Episodic spasms precipitated by unexpected noises, tactile stimuli, or emotional upset
  • Absence of any other neurologic disease that could explain the stiffness and rigidity
  • High titer serum anti-GAD65 antibodies shown at screening -OR- seropositive for anti-glycine antibodies. If anti-GAD65 antibodies are lower than the high titer threshold peripherally but positive in the cerebrospinal fluid (CSF), the subject can be included. A prior documented high titer anti-GAD65 antibody level may be acceptable subject to sponsor review.
  • Active symptoms with inadequate response to at least one immunomodulatory therapy.
  • Stiffness index ≥2.
  • At least 20 of the 25 enrolled subjects should be ambulatory.
  • Key

Exclusion

  • Bedridden subjects for more than 3 months.
  • History of CNS or spinal cord tumor, metabolic or infectious cause of myelopathy, genetically inherited progressive CNS disorder, sarcoidosis, non-SPS progressive neurologic condition or progressive multifocal leukoencephalopathy (PML).
  • History of stroke, seizure, dementia, Parkinson's disease, cerebellar diseases, psychosis, aphasia, and any other neurologic disorder that is of a nature and severity that the investigator considers would increase the risk for the subject.
  • Cardiac ejection fraction ≤ 40%.

Key Trial Info

Start Date :

September 25 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

December 1 2026

Estimated Enrollment :

25 Patients enrolled

Trial Details

Trial ID

NCT06588491

Start Date

September 25 2024

End Date

December 1 2026

Last Update

December 22 2025

Active Locations (3)

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Page 1 of 1 (3 locations)

1

University of Colorado Anschutz Medical Campus

Aurora, Colorado, United States, 80045

2

Mayo Clinic

Rochester, Minnesota, United States, 55905

3

Thomas Jefferson University Hospital

Philadelphia, Pennsylvania, United States, 19107