Efgartigimod for Stiff Person Syndrome (ESPS)

NCT06528392 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: STUDY006922
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Stiff-Person Syndrome

Keywords

Stiff-Person Syndrome; Efgartigimod

Outcome Measures

Primary Outcomes (2)

• Distribution of Stiffness Index (DSI)

  DSI is a validated indicator or stiffness. Scores range from 0 to 6 and reflect the extent of stiffness. One point is given for stiffness in each of the following areas: lower trunk, upper trunk, legs, arms, face and abdomen.

  From enrollment to the end of treatment at week 13

• heightened sensitivity score (HSS)

  HSS measures changes in the frequency of spasms. Scores range from 1 to 7; one point given for each source or type of spasm: unexpected noises, visual stimuli, somatosensory stimuli, voluntary activities, emotional upset or stress, no specific stimuli, or nocturnal spasms.

  From enrollment to end of treatment at week 13

Secondary Outcomes (3)

• Proportion of patients with SPS who are treatment responders

  enrollment to end of 13 weeks

• The duration of time patients maintain responder status

  from enrollment to end of treatment at week 13

• Modified Rankin Scale

  From enrollment to end of treatment at week 13

Study Arms (1)

subcutaneous Efgartigimod-hyaluronidase (EFG)

EXPERIMENTAL

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

Biological: Efgartigimod

Interventions

Efgartigimod BIOLOGICAL

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

subcutaneous Efgartigimod-hyaluronidase (EFG)

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18-80, men and women, capable of giving informed consent.
• Must have unexplained stiffness of limb and axial (trunk) muscles and involuntary striated muscle spasms.
• DSI and HHS must be \>=2 (to allow for detection of improvement).
• Presence of serum (or CSF) anti-GAD antibodies regardless of titers.
• Stable dosage for at least two months before screening visit with antispasmodics (muscle relaxants, benzodiazepines, baclofen).
• Women with children bearing potential must be willing to use highly effective contraception method during and for a minimum of 90 days after the last dose of IP.

You may not qualify if:

• a. type 1 DM
• b. Cancer, except basal cell skin cancer
• c. Infections: HIV, Hepatitis B or C, clinically significant active infection not sufficiently resolved in the investigator's opinion
• d. Serum IgG (\<6 g/L) at screening

Sponsors & Collaborators

• University of South Florida: lead

Study Sites (1)

University of South Florida, Carol and Frank Morsani Center for Advanced Healthcare

Tampa, Florida, 33612, United States

Location

MeSH Terms

Conditions

Stiff-Person Syndrome

Interventions

efgartigimod alfa

Condition Hierarchy (Ancestors)

Autoimmune Diseases of the Nervous System; Nervous System Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Tuan H Vu, MD

  University of South Florida

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tuan H Vu, MD

CONTACT

813-974-9413; tvu6@usf.edu

Jessica D Shaw, MPH

CONTACT

813-974-9413; jessshaw@usf.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is an exploratory, single arm treatment, non-inferiority trial.

Study Record Dates

• First Submitted: July 25, 2024
• First Posted: July 30, 2024
• Study Start: April 1, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 11, 2025

Record last verified: 2025-04

Locations

US (1)