Efgartigimod for Stiff Person Syndrome (ESPS)

Conditions

• Stiff-Person Syndrome

Eligibility Criteria

Sex

ALL

Age

18 Years - 80 Years

Healthy Volunteers

Not accepted

Interventions

• Efgartigimod — BIOLOGICAL
  Efgartigiomd-hyaluroidase (EFG) is a neonatal Fc receptor (FcRn) inhibitor, which blocks the recycling of Immunoglobulin G (IgG), including pathogenic IgG

Study Details

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.

Key Dates

Start date

Apr 1, 2025

Status verified

Apr 2025

Primary completion

Dec 31, 2026

Completion

Dec 31, 2027

Study Design

Enrollment

10 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: subcutaneous Efgartigimod-hyaluronidase (EFG)
  Participants with GAD65-antibody positive SPS will be given subcutaneous Efgartigimod-hyaluronidase (EFG).

Primary Outcome Measure

Distribution of Stiffness Index (DSI) [ Time Frame: From enrollment to the end of treatment at week 13 ]

Central Contacts

• Tuan H Vu, MD
  813-974-9413
  [email protected]
• Jessica D Shaw, MPH
  813-974-9413
  [email protected]

Locations (1)

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