Status:

COMPLETED

IVIg for Small Fiber Neuropathy With Autoantibodies TS-HDS and FGFR3

Lead Sponsor:

Beth Israel Deaconess Medical Center

Collaborating Sponsors:

Phoenix Neurological Associates, LTD

Conditions:

Small Fiber Neuropathy

Idiopathic Peripheral Neuropathy

Eligibility:

All Genders

Phase:

PHASE2

Brief Summary

The objective of this study is to develop a rationale for the selective treatment of small fiber neuropathy with immune globulin (IVIG) in the appropriate patients. The investigators hypothesize that...

Detailed Description

Small fiber neuropathies, and mixed small and large fiber neuropathies, have many potential causes including diabetes, vitamin deficiencies, environmental and toxic exposures, HIV, autoimmune and para...

Eligibility Criteria

Inclusion

  • Patient with clinically evident and biopsy proven pure small fiber neuropathy as evidenced by reduced intra-epidermal nerve fiber density seen on skin biopsy using PGP 9.5 as the immunostain.
  • Patients must have a baseline pain score on a VAS scale of Greater or equal to 4/10
  • Patients must have elevated titers of autoantibodies to TS-HDS or FFR3 as measured in Dr Alan Pestronk's lab at Washington University in St Louis.

Exclusion

  • Any other known cause for small fiber neuropathy other than the presence of the elevated titers of auto-antibodies. For example patients with diabetes, HIV, Sjogrens, Vitamin deficiency etc.
  • Patients with generalized, severe musculoskeletal conditions other than SFN that prevent a sufficient assessment of the patient by the physician
  • Cardiac insufficiency (New York Heart Association III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease).
  • Severe liver disease (ALAT 3x \> normal value).
  • Severe kidney disease (creatinine 1.5x \> normal value).
  • Known hepatitis B, hepatitis C or HIV infection.
  • Patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever; patients with susceptibility to embolism or deep vein thrombosis.
  • Body mass index (BMI) ≥40 kg/m2.
  • Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome).
  • Known IgA deficiency with antibodies to IgA.
  • History of hypersensitivity, anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products, or any component of Gamunex.
  • Known blood hyperviscosity, or other hypercoagulable states.
  • Use of IgG products within six months prior to enrolment.
  • Use of other blood or plasma-derived products within three months prior to enrollment.
  • Patients with a history of drug or alcohol abuse within the past five years prior to enrollment.
  • Patients unable or unwilling to understand or comply with the study protocol
  • Participating in another interventional clinical study with investigational treatment within three months prior to enrollment.
  • Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method (such as implants, injectable, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or vasectomized partner) while on study.

Key Trial Info

Start Date :

September 1 2018

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

February 1 2022

Estimated Enrollment :

20 Patients enrolled

Trial Details

Trial ID

NCT03401073

Start Date

September 1 2018

End Date

February 1 2022

Last Update

September 13 2023

Active Locations (1)

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1

Beth Israel Deaconness Medical Center

Boston, Massachusetts, United States, 02215