Health TrialsbyHealthPanel

Efgartigimod for Stiff Person Syndrome (ESPS)

ConditionStiff-Person Syndrome
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-80
SponsorUniversity of South Florida

About this trial

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

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

None

Trial design

Design model

Single group

Treatments tested in this trial

  • Efgartigimod

    Biological/Vaccine

    Efgartigiomd-hyaluroidase (EFG) is a neonatal Fc receptor (FcRn) inhibitor, which blocks the recycling of Immunoglobulin G (IgG), including pathogenic IgG

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: subcutaneous Efgartigimod-hyaluronidase (EFG)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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
2

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

1

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
2

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
3

Modified Rankin Scale

Time frame
From enrollment to end of treatment at week 13

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of South Florida

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: