KYSA-8: A Phase 2 Open-Label, Single-Arm, Multicenter Study of KYV 101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T Cell (CD19 CAR T) Therapy, in Subjects With Treatment Refractory Stiff Person Syndrome
A Study of Anti-CD19 Chimeric Antigen Receptor T Cell Therapy for Subjects With Treatment Refractory Stiff Person Syndrome
• 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.