NCT04153422 IVIG in the Treatment of Autoimmune Small Fiber Neuropathy With TS-HDS, FGFR-3, or Plexin D1 Antibodies
| NCT ID | NCT04153422 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Endeavor Health |
| Condition | Small Fiber Neuropathy |
| Study Type | INTERVENTIONAL |
| Enrollment | 20 participants |
| Start Date | 2023-12-15 |
| Primary Completion | 2027-08-27 |
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 20 participants in total. It began in 2023-12-15 with a primary completion date of 2027-08-27.
⚠ 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
This study will enroll patients with small fiber neuropathy (SFN). The study will look at an intravenous immunoglobulin (IVIG) called Panzyga. Panzyga is approved by the FDA as a therapy for Primary humoral immunodeficiency (PI) in patients 2 years of age and older; Chronic immune thrombocytopenia (ITP) in adults and Chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. It has not been approved by the FDA for use in SFN. There is mounting evidence that Intravenous Immunoglobulin (IVIG) can cause pain reduction and improve objective nerve fiber densities on skin biopsies in great numbers in SFN patients. The primary outcome is quantified improvement in intraepidermal nerve fiber density (IENFD) on repeat skin punch biopsy after 6 months of IVIG treatment.
Eligibility Criteria
Inclusion Criteria: 1. Patients ≥ age 18 2. Patient with clinical and biopsy evidence of pure small fiber neuropathy (with or without dysautonomia) as evidenced by reduced IENFD on skin biopsy using PGP 9.5 as the immunostain. Biopsy must have been performed within 12 months of study enrollment. If biopsies were not done at CRL, they will be repeated and done at 3 sites (upper and lower thigh, lower calf), to have consistent and equivalent biopsy data with the follow up biopsy done after 6 mos of treatment 3. Patients must have elevated and/or abnormal titers of autoantibodies to TS-HDS-IgM, FGFR3-IgG, or Plexin-D1 measured by the Washington University Neuromuscular Laboratory (St Louis) within 12 mos of enrollment 4. Patients must have a baseline pain score on a visual analogue scale (VAS) of Greater or equal to 4/10 5. Patients must have a baseline Utah Early Neuropathy Scale (UENS) score of Greater or equal to 4/10 6. Small Fiber Neuropathy Screening List (SFNSL) score of 11/84 or greater 7. Non-pregnant, non-lactating female. Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during treatment Exclusion Criteria: 1. Any other known cause for small fiber neuropathy other than the presence of the elevated titers of TS-HDS-IgM, FGFR3-IgG, or Plexin-D1 autoantibodies 2. Patients with generalized, severe musculoskeletal conditions other than SFN that prevent a sufficient assessment of the patient by the physician 3. Electromyography/nerve conduction study (EMG/NCS) evidence of large fiber polyneuropathy, to be confirmed by study PI 4. Underlying severe heart, kidney, liver disease, or HIV infection, (Note: If there is no previous HIV test result documented within the last 5 years, a test may be performed in order to confirm eligibility) 5. Patients with a history of deep vein thrombosis within the last year prior to baseline visit or pulmonary embolism ever; patients with susceptibility to embolism or deep vein thrombosis 6. Known significant IgA deficiency with antibodies to IgA 7. History of hypersensitivity, anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products, or any component of IVIG 10% 8. Known blood hyperviscosity, or other hypercoagulable states 9. Use of IgG products within six months prior to enrollment 10. Patients with a history of drug or alcohol abuse within the past five years prior to enrollment 11. Patients unable to understand or unwilling or unable to comply with the study protocol
Contact & Investigator
Lawrence Zeidman, MD, FAAN
PRINCIPAL INVESTIGATOR
Endeavor Health
Frequently Asked Questions
Who can join the NCT04153422 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Small Fiber Neuropathy. 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 NCT04153422 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 NCT04153422 currently recruiting?
Yes, NCT04153422 is actively recruiting participants. Contact the research team at anna.pham@endeavorhealth.org for enrollment information.
Where is the NCT04153422 trial being conducted?
This trial is being conducted at Glenview, United States.
Who is sponsoring the NCT04153422 clinical trial?
NCT04153422 is sponsored by Endeavor Health. The principal investigator is Lawrence Zeidman, MD, FAAN at Endeavor Health. The trial plans to enroll 20 participants.