Efgartigimod for Stiff Person Syndrome (ESPS)

Phase 2Not Yet RecruitingNCT06528392

University of South Florida10 enrolled

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Stiff-Person Syndrome

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

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.

Time frame: 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.

Time frame: From enrollment to end of treatment at week 13

Secondary Outcomes

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)

Time frame: 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)

Time frame: from enrollment to end of treatment at week 13

Modified Rankin Scale