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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:
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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| subcutaneous Efgartigimod-hyaluronidase (EFG) | Experimental | Participants with GAD65-antibody positive SPS will be given subcutaneous Efgartigimod-hyaluronidase (EFG). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Efgartigimod | Biological | Efgartigiomd-hyaluroidase (EFG) is a neonatal Fc receptor (FcRn) inhibitor, which blocks the recycling of Immunoglobulin G (IgG), including pathogenic IgG |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of patients with SPS who are treatment responders | 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) | enrollment to end of 13 weeks |
| The duration of time patients maintain responder status |
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Inclusion Criteria:
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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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Tuan H Vu, MD | Contact | 813-974-9413 | tvu6@usf.edu | |
| Jessica D Shaw, MPH | Contact | 813-974-9413 | jessshaw@usf.edu |
| Name | Affiliation | Role |
|---|---|---|
| Tuan H Vu, MD | University of South Florida | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of South Florida, Carol and Frank Morsani Center for Advanced Healthcare | Tampa | Florida | 33612 | United States |
IPD will be shared between designated study sites during the trial. After the end of the trial data may be shared but this has yet to be determined.
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| ID | Term |
|---|---|
| D016750 | Stiff-Person Syndrome |
| ID | Term |
|---|---|
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C000718373 | efgartigimod alfa |
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This is an exploratory, single arm treatment, non-inferiority trial.
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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) |
| from enrollment to end of treatment at week 13 |
| Modified Rankin Scale | From enrollment to end of treatment at week 13 |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |