Clinical trial NCT06528392: Efgartigimod for Stiff Person Syndrome (ESPS)
Official title: Efgartigimod for Stiff Person Syndrome (ESPS)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Stiff-Person Syndrome
Keywords: Stiff-Person Syndrome
Efgartigimod
Lead sponsor: University of South Florida
Sponsor class: OTHER
First posted: 2024-07-30
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This is an exploratory, single arm treatment, non-inferiority trial.
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

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.

Primary outcomes:
measure: Distribution of Stiffness Index (DSI)
timeFrame: From enrollment to the end of treatment at week 13
measure: heightened sensitivity score (HSS)
timeFrame: From enrollment to end of treatment at week 13
Secondary outcomes:
measure: Proportion of patients with SPS who are treatment responders
timeFrame: enrollment to end of 13 weeks
measure: The duration of time patients maintain responder status
timeFrame: from enrollment to end of treatment at week 13
measure: Modified Rankin Scale
timeFrame: From enrollment to end of treatment at week 13
Other outcomes:
Locations:
city: Tampa
country: United States
facility: University of South Florida, Carol and Frank Morsani Center for Advanced Healthcare
state: Florida
Study officials:
affiliation: University of South Florida
name: Tuan H Vu, MD
role: PRINCIPAL_INVESTIGATOR
References: