IVIG in the Treatment of Autoimmune Small Fiber Neuropathy With TS-HDS or FGFR-3 Antibodies

• Status: Suspended
• Trial end date: 2024-09-01
• Target enrollment: 0
• Participant gender: All

Summary

This study will enroll patients with small fiber neuropathy (SFN). The study will look at an intravenous immunoglobulin (IVIG) called Gammagard. Gammagard is approved by the FDA as a therapy for certain diseases that result from an impaired immune system and as a maintenance therapy to improve sensation and strength in patients with Chronic Immune Demyelinating Polyneuropathy (CIDP) and muscle strength in adults with Multifocal Motor Neuropathy (MMN). It has not been approved by the FDA for use in this condition.

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Lawrence Zeidman; Loyola University
• Collaborator: Takeda
• Treatments: Antibodies; gamma-Globulins; Immunoglobulins; Immunoglobulins, Intravenous; Rho(D) Immune Globulin

Criteria

Inclusion Criteria:

1. Patients ≥ age 18

2. Patient with clinical and biopsy evidence of pure small fiber neuropathy 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.

3. Patients must have elevated titers of autoantibodies to TS-HDS-IgM or FGFR3-IgG, measured by the Washington University Neuromuscular Laboratory (St Louis).

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

Exclusion Criteria:

1. Any other known cause for small fiber neuropathy other than the presence of the elevated titers of auto-antibodies.

2. Patients with generalized, severe musculoskeletal conditions other than SFN that prevent a sufficient assessment of the patient by the physician.

3. Underlying severe heart, kidney, liver disease, or HIV infection,

4. 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.

5. Known IgA deficiency with antibodies to IgA,

6. History of hypersensitivity, anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products, or any component of Gammagard,

7. Known blood hyperviscosity, or other hypercoagulable states,

8. Use of IgG products within six months prior to enrollment,

9. Patients with a history of drug or alcohol abuse within the past five years prior to enrollment,

10. Patients unable or unwilling to understand or comply with the study protocol