Stiff-Person SyndromePhase 2ESPSNCT06528392
Efgartigimod for Stiff Person Syndrome (ESPS)
A Phase 2 study testing Efgartigimod in people with Stiff-Person Syndrome, run by University of South Florida at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Stiff-Person Syndrome
- What is being tested?
- Efgartigimod
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Apr 2025to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
University of South Florida, Carol and Frank Morsani Center for Advanced Healthcare
Tampa, Florida, 33612
Jessica D Shaw, MPH, 813-974-9413, jessshaw@usf.edu
Tuan H Vu, MD
About this study
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.
- Conditions
- Stiff-Person Syndrome
- Being tested
- Efgartigimod
- Keywords
- Stiff-Person Syndrome, Efgartigimod
- Medical subject headings
- Stiff-Person Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EfgartigimodBiological
Efgartigiomd-hyaluroidase (EFG) is a neonatal Fc receptor (FcRn) inhibitor, which blocks the recycling of Immunoglobulin G (IgG), including pathogenic IgG
| Study group | Role | What they receive |
|---|---|---|
| subcutaneous Efgartigimod-hyaluronidase (EFG) | Experimental | Participants with GAD65-antibody positive SPS will be given subcutaneous Efgartigimod-hyaluronidase (EFG). Receives: Biological: Efgartigimod |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: From enrollment to end of treatment at week 13
Secondary outcomes (3)
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)
Measured: enrollment to end of 13 weeks
The duration of time patients maintain responder status
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)
Measured: from enrollment to end of treatment at week 13
Modified Rankin Scale
Measured: From enrollment to end of treatment at week 13
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2024
- Study start1 Apr 2025 (estimated)
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stiff-Person SyndromeStudies of the same condition, open and under way.
- More trials run by University of South FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is an exploratory, single arm treatment, non-inferiority trial.
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Tuan H Vu, MD, Principal investigator, University of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06528392
- Sponsor's protocol ID
- STUDY006922
- Acronym
- ESPS
- Status last verified
- Apr 2025
- First submitted
- 25 Jul 2024
- Last updated
- 11 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: 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.
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efgartigimod for Stiff Person Syndrome (ESPS)
- ClinicalTrials.gov ID
- NCT06528392
- Acronym / sponsor ID
- ESPS / STUDY006922
- Conditions
- Stiff-Person Syndrome
- Keywords
- Stiff-Person Syndrome, Efgartigimod
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Study start
- 1 Apr 2025 (estimated)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 30 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is an exploratory, single arm treatment, non-inferiority trial.
| Arm | Type | Receives |
|---|---|---|
| subcutaneous Efgartigimod-hyaluronidase (EFG) | Experimental | Biological: EfgartigimodParticipants with GAD65-antibody positive SPS will be given subcutaneous Efgartigimod-hyaluronidase (EFG). |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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).
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EfgartigimodEfgartigiomd-hyaluroidase (EFG) is a neonatal Fc receptor (FcRn) inhibitor, which blocks the recycling of Immunoglobulin G (IgG), including pathogenic IgG | Biological | subcutaneous Efgartigimod-hyaluronidase (EFG) |
Primary outcome
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.
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.
3 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2025.
- Individual participant dataSharing planned: Undecided. 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.
Related trials for Stiff-Person Syndrome
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A Study Investigating Intravenous Human Normal Immune Globulin 10% in Adults With Stiff Person Syndrome
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VYVGART (Hytrulo) in Patients With Stiff Person Syndrome (SPS)
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RecruitingObservational study
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Enrolling by invitationPhase 1
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Tuan H Vu, MD principal investigator
University of South Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Efgartigimod in people with Stiff-Person Syndrome, run by University of South Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 11 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Tuan H Vu, MD (813-974-9413, tvu6@usf.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Distribution of Stiffness Index (DSI) (time frame: From enrollment to the end of treatment at week 13). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Apr 2025.