Efgartigimod for Stiff Person Syndrome (ESPS)

Phase 2

Not yet recruiting

• Conditions: Stiff-Person Syndrome
• Interventions: Biological: Efgartigimod

• Registration Number: NCT06528392
• Lead Sponsor: University of South Florida

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-07
• Study First Submitted: 2024-07-25
• Study First Submitted QC: 2024-07-25
• Last Update Submitted: 2024-07-25
• Study First Posted: 2024-07-30
• Last Update Posted: 2024-07-30
• Study Start: 2024-08-01
• Primary Completion: 2026-06-30
• Study Completion: 2027-06-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 10

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 days after the last dose of IP.

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Exclusion Criteria

1. 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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Distribution of Stiffness Index (DSI)	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.
heightened sensitivity score (HSS)	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 Outcome Measures

Name	Time	Method
Proportion of patients with SPS who are treatment responders	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)
Modified Rankin Scale	From enrollment to end of treatment at week 13
The duration of time patients maintain responder status	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)

Trial Locations

Locations (1)

University of South Florida, Carol and Frank Morsani Center for Advanced Healthcare; 🇺🇸 Tampa, Florida, United States