IVIG (Panzyga) treatment for autoimmune small fiber neuropathy with TS-HDS, FGFR3, or Plexin D1 antibodies

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 2 interventional trial enrolls adults with biopsy-confirmed small fiber neuropathy and positive TS-HDS, FGFR3, or Plexin D1 antibodies to receive IVIG (Panzyga) or placebo. Participants will undergo baseline and 6-month follow-up skin punch biopsies to measure intraepidermal nerve fiber density (IENFD), with the primary outcome being change in IENFD after six months of treatment. Key entry requirements include age 18 or older, reduced IENFD on recent biopsy, elevated specified autoantibodies measured at the Washington University Neuromuscular Laboratory, and minimum pain and neurologic symptom scores. Panzyga is FDA-approved for other immune indications but is not approved for small fiber neuropathy, and this trial aims to generate controlled data on its effects in this antibody-positive subgroup.

Why it matters

Who can join: the full rules

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

Where it's running

• Endeavor Health
  Glenview, Illinois, United States Recruiting

Study contacts

• Principal investigator: Lawrence Zeidman, MD, FAAN — Endeavor Health
• Study coordinator: Anna Pham
• Email: anna.pham@endeavorhealth.org
• Phone: 8475034344

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.