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NCT Number: NCT06528392

Efgartigimod for Stiff Person Syndrome (ESPS)

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.

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Key information

Conditions

Stiff-Person Syndrome Autoimmune Diseases Autoimmune Diseases of the Nervous System Central Nervous System Diseases Immune System Diseases Nervous System Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

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

Tampa, Florida, 33612, United States

Location contact

Jessica D Shaw, MPH

CONTACT

[email protected]

813-974-9413

Tuan H Vu, MD

PRINCIPAL_INVESTIGATOR

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • Exclusionary conditions:

1a. type 1 DM

1b. Cancer, except basal cell skin cancer

1c. Infections: HIV, Hepatitis B or C, clinically significant active infection not sufficiently resolved in the investigator's opinion

1d. Serum IgG (<6 g/L) at screening

1e. pregnancy/breast feeding. 2. Exclusionary medications: rituximab within 6 months of screening. 3. IVIG or PLEX treatment 4 weeks prior to screening. 4. History of any type of solid organ or bone marrow transplant.

Treatment and study plan

Efgartigimod

Biological

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

Primary outcomes

  1. Distribution of Stiffness Index (DSI)

    Time frame: From enrollment to the end of treatment at week 13

    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.

  2. heightened sensitivity score (HSS)

    Time frame: From enrollment to end of treatment at week 13

    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.

Secondary outcomes

  1. Proportion of patients with SPS who are treatment responders

    Time frame: enrollment to end of 13 weeks

    Proportion of patients with SPS who are treatment responders, defined as: >= 2 points reduction in the distribution of stiffness index (DSI) AND >= 1 point reduction in heightened sensitivity score (HSS)

  2. The duration of time patients maintain responder status

    Time frame: from enrollment to end of treatment at week 13

    A responder is defined as one having >= 2 points reduction in the distribution of stiffness index (DSI) AND >= 1 point reduction in heightened sensitivity score (HSS)

  3. Modified Rankin Scale

    Time frame: From enrollment to end of treatment at week 13

Study contacts

Contact information is provided by the study sponsor or research team.

Jessica D Shaw, MPH

CONTACT

[email protected]

813-974-9413

Tuan H Vu, MD

CONTACT

[email protected]

813-974-9413

Sponsors and collaborators

Lead sponsor

University of South Florida

Other

Registry information

Acronym: ESPS

Important dates

Study start
2025
Primary completion
2026
Study completion
2027
First posted
Jul 30, 2024
Registry last updated
Apr 11, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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