NCT07860021 VYVGART (Hytrulo) in Patients With Stiff Person Syndrome (SPS)
| NCT ID | NCT07860021 |
| Status | Recruiting |
| Phase | Phase 2, Phase 3 |
| Sponsor | Thomas Jefferson University |
| Condition | Stiff Person Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 26 participants |
| Start Date | 2026-09-30 |
| Primary Completion | 2027-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 26 participants in total. It began in 2026-09-30 with a primary completion date of 2027-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
Stiff Person Syndrome (SPS) is a rare autoimmune disorder, characterized by: a) incapacitating diffuse muscle stiffness prominent in the axial, abdominal and thoracolumbar paraspinal muscles leading to hyperlordosis, difficulty turning and bending; b) painful muscle spasms precipitated by external stimuli with difficulty performing routine daily activities; and c) slow freezing gait with frequent falls and need for walking aids leading to disability. SPS is due to impaired inhibitory GABAergic neurotransmission associated with high-titer IgG antibodies against Glutamic Acid Decarboxylase (GAD), the rate-limiting enzyme for the synthesis of the inhibitory gammaaminobutyric acid (GABA). Although SPS patients respond to IVIg based on the PI's controlled study (NEJM 2001), several patients do not improve, or their response is modest and declines over time, as documented in a longitudinal study of 36 patients followed in our Jefferson clinic; because another controlled study we performed with Rituximab was negative and SPS is a progressive disease, new therapies are needed. Efgartigimod is an FcRn inhibitor that enhances the catabolism of circulating IgG; its subcutaneous, self-administered form, HYTRULO, has been effective and FDA-approved in patients with myasthenia gravis or CIDP poorly responding to IVIg. There is evidence that patients with myasthenia who also had SPS, Hytrulo not only helped their myasthenia but the SPS also impressively improved. The present study is an investigator- initiated, double-blind, placebo controlled, cross-over, trial in 26 GAD-positive SPS patients poorly responding to available therapies. Patients will be recruited from the large number of patients seen and referred to the PI who is known for his work in SPS and has conducted the 2 previous controlled trials. Although Hytrulo is approved as selfadministered subcutaneous drug with pre-refilled syringes, in this study will be given by a contracted home infusion company once a week to ensure convenience, safety and validity. Patients will be randomized by the Jefferson Pharmacy to receiving Hytrulo or identical hyaluronidase-containing placebo for 3 months; then they will be crossed-over to placebo/Hytrulo for another 3 months. Efficacy will be based on one primary endpoint, the stiffness index, previously validated by the PI, and on two also validated secondary endpoints of time to walk and excitability index. This design provides the statistical power to objectively assess a 25-50% or greater change and objectively determine if Hytrulo offers meaningful benefits in reducing stiffness and spasms, improving gait and increasing quality of life in SPS patients poorly responding to current therapies.
Eligibility 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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07860021 clinical trial?
This trial is open to participants of all sexes, aged 25 Years or older, up to 80 Years, studying Stiff Person Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07860021 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT07860021 currently recruiting?
Yes, NCT07860021 is actively recruiting participants. Contact the research team at marinos.dalakas@jefferson.edu for enrollment information.
Where is the NCT07860021 trial being conducted?
This trial is being conducted at Philadelphia, United States.
Who is sponsoring the NCT07860021 clinical trial?
NCT07860021 is sponsored by Thomas Jefferson University. The trial plans to enroll 26 participants.