Efgartigimod for Stiff Person Syndrome (ESPS)
University of South Florida
Summary
The goal of this clinical trial is to examine the proportion of patients with stiff person syndrome with no worsening in stiffness and/or spasms while on efgartigimod-hyaluronidase (EFD) when compared to their current treatment. The main questions it aims to answer are: * proportion of patients with stiff person syndrome with no worsening in DSI and HSS scores * whether there are changes in laboratory values, vital signs, electrocardiogram, or depression, and suicide risk during the trial, as well as the incidence of any adverse events. Participants will come to the clinic up to 18 times and have up to 5 phone call visits during the course of the trial. During these visits a variety of measures will be collected, including blood samples, neurological and physical exams, patient reported outcomes on stiffness, spasms, depression, and suicide risk.
Eligibility
- Age range
- 18–80 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Age 18-80, men and women, capable of giving informed consent. 2. Must have unexplained stiffness of limb and axial (trunk) muscles and involuntary striated muscle spasms. 3. DSI and HHS must be \>=2 (to allow for detection of improvement). 4. Presence of serum (or CSF) anti-GAD antibodies regardless of titers. 5. Stable dosage for at least two months before screening visit with antispasmodics (muscle relaxants, benzodiazepines, baclofen). 6. Women with children bearing potential must be willing to use highly effective contraception method during and for a minimum of 90…
Interventions
- BiologicalEfgartigimod
Efgartigiomd-hyaluroidase (EFG) is a neonatal Fc receptor (FcRn) inhibitor, which blocks the recycling of Immunoglobulin G (IgG), including pathogenic IgG
Location
- University of South Florida, Carol and Frank Morsani Center for Advanced HealthcareTampa, Florida