VYVGART Hytrulo in stiff person syndrome
Official title: The Efficacy of Efgartigimod PH20 SC (VYVGART Hytrulo ) in Patients With Stiff Person Syndrome (SPS): A Double-blind, Placebo-controlled, Cross-over Trial
Run by Thomas Jefferson University · NCT07860021 · Data updated 2026-11-12
This trial tests VYVGART Hytrulo versus placebo in adults with stiff person syndrome who have not fully benefited from IVIg or rituximab.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Ages | 25 Years to 80 Years |
| Sex | All |
| Enrollment | 26 (estimated) |
| Sponsor | Thomas Jefferson University Academic / other |
| Drugs / interventions | rituximab |
| Locations | 1 site (Philadelphia, Pennsylvania) |
| Status | Recruiting |
| Started | September 30, 2026 |
| Main results expected | December 31, 2027 |
| Study ends | July 31, 2028 |
| Registry record updated | October 6, 2026 |
| Trial ID | NCT07860021 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults aged 25-80 with typical stiff person syndrome, who have partial or poor response to IVIg or rituximab and have measurable stiffness and walking impairment, and who are antibody-positive.
✕Probably not a fit
Patients with minimal disease burden or unresponsive to all therapies may not derive meaningful benefit.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a randomized, multi-phase interventional study evaluating VYVGART Hytrulo in adults with stiff person syndrome who have partial or no response to standard therapies. Participants continue their existing symptomatic medications while receiving either Hytrulo or a placebo, with monitoring for changes in stiffness, muscle spasms, and walking ability. The study aims to determine whether Hytrulo can improve axial stiffness and reduce spasms compared with placebo. Eligible patients are GAD-antibody or glycine-receptor antibody-positive and meet criteria reflecting significant disease burden. Sites are located at Thomas Jefferson University in Philadelphia, PA, under the lead sponsor Thomas Jefferson University.
Why it matters
Potential benefit: If successful, participants could experience reduced stiffness and fewer painful spasms, improving mobility and daily functioning.
How similar studies have gone: Previous controlled studies and clinical experience suggest variability in response to related therapies, making this evaluation of Hytrulo in SPS novel and informative.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * 26 patients age 25-80 with typical SPS as per Dalakas's diagnostic criteria (1,2) poorly or partially responding to IVIg or rituximab with declining benefits, exhibiting significant symptomatology of stiffness, painful muscle spasms, and impaired walking ability will be selected. All enrolled patients will be GAD-ab or Glycin-receptor-antibody-positive and will continue receiving the non-immunosuppressive symptomatic drugs, such as diazepam, gabapentin, or baclofen. * All selected patients will be among the large number we currently follow in our outpatient clinic or among those being continuously referred for a second opinion or better therapies when unresponsive to current drugs. * All patients should be able to provide informed consent and willing to travel for scheduled protocol visits. Exclusion Criteria: * Patients will be excluded if: a) have minimal disease, and less than 1 stiffness index; b) have very severe disease unresponsive to all therapies or non-ambulatory; and c) have paraneoplastic SPS.
Where it's running
-
Thomas Jefferson University
Philadelphia, Pennsylvania, United States Recruiting
Study contacts
- Study coordinator: Marinos Dalakas, M.D., PhD, FAAN
- Email: marinos.dalakas@jefferson.edu
- Phone: 215-955-7865
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.