Efgartigimod for Stiff Person Syndrome (ESPS) (NCT06528392) | Clinical Trial Compass
Not Yet RecruitingPhase 2
Efgartigimod for Stiff Person Syndrome (ESPS)
United States10 participantsStarted 2025-04-01
Plain-language 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.
Who can participate
Age range
18 Years – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Distribution of Stiffness Index (DSI)
Timeframe: From enrollment to the end of treatment at week 13
2
heightened sensitivity score (HSS)
Timeframe: From enrollment to end of treatment at week 13