Efgartigimod for Stiff Person Syndrome (ESPS)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Efgartigimod.
- Who it may be relevant to
- Registry conditions: Stiff-Person Syndrome. Basic parameters: 18 years — 80 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Interventions
- Biological Efgartigimod
Efgartigiomd-hyaluroidase (EFG) is a neonatal Fc receptor (FcRn) inhibitor, which blocks the recycling of Immunoglobulin G (IgG), including pathogenic IgG
Primary outcome measures
- Distribution of Stiffness Index (DSI) [Time frame: From enrollment to the end of treatment at week 13]
- heightened sensitivity score (HSS) [Time frame: From enrollment to end of treatment at week 13]
Secondary outcome measures (3)
- Proportion of patients with SPS who are treatment responders [Time frame: enrollment to end of 13 weeks]
- The duration of time patients maintain responder status [Time frame: from enrollment to end of treatment at week 13]
- Modified Rankin Scale [Time frame: From enrollment to end of treatment at week 13]
Eligibility criteria
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
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 1 center
- University of South Florida, Carol and Frank Morsani Center for Advanced Healthcare — Tampa
Identifiers
NCT: NCT06528392 · STUDY006922