IVIG in the Treatment of Autoimmune Small Fiber Neuropathy With TS-HDS or FGFR-3 Antibodies
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|ClinicalTrials.gov Identifier: NCT04153422|
Recruitment Status : Suspended (Recruitment not started at Loyola, PI leaving Loyola, PI may resume at next affiliating instituation.)
First Posted : November 6, 2019
Last Update Posted : February 12, 2021
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.
|Condition or disease||Intervention/treatment||Phase|
|Small Fiber Neuropathy Autoimmune Small Fiber Neuropathy Inflammatory Polyneuropathy Immune-Mediated Neuropathy||Drug: Gammagard IVIG Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||This is a randomized, double-blind, placebo-controlled design with 10 patients in the treatment arm and 10 in the placebo arm. Patients in the treatment arm will receive 2g/kg Gammagard IVIG initially (1g/kg dose on Day 1 and 1g/kg dose on Day 2) and then 1g/kg maintenance infusions for 5 additional months (six months total). Patients in the placebo arm will receive 0.9% NaCl during the initial infusion and at maintenance infusions for 5 additional months (six months total).|
|Masking:||Double (Participant, Investigator)|
|Masking Description:||This is a double-blind trial. The participant and the investigator will be blinded.|
|Official Title:||Intravenous Immunoglobulin (IVIG) in the Treatment of Autoimmune Small Fiber Neuropathy Due to TS-HDS and FGFR-3 Antibodies: a Double Blinded Placebo-controlled Phase II Trial|
|Estimated Study Start Date :||January 1, 2022|
|Estimated Primary Completion Date :||September 1, 2023|
|Estimated Study Completion Date :||September 1, 2024|
Experimental: Treatment (IVIG)
Patients in the treatment arm will receive 2g/kg Gammagard IVIG initially (1g/kg dose on Day 1 and 1g/kg dose on Day 2) and then 1g/kg maintenance infusions for 5 additional months (six months total).
Drug: Gammagard IVIG
Immune Globulin Infusion 10% (Human)
Placebo Comparator: Placebo
Patients in the placebo arm will receive 0.9% NaCl during the initial infusion and at maintenance infusions for 5 additional months (six months total).
0.9% NaCl prepared as the calculated dose equivalent volume to IVIG.
- quantified change in intraepidermal nerve fiber density (IENFD) [ Time Frame: Week 24 ]3mm skin punch biopsy
- Change in visual analogue pain scale responses [ Time Frame: baseline and Week 28 ]Self-reported pain intensity on a scale of 0-10 using the Wong-Baker FACES Pain Rating Scale, with 0 being no pain and 10 being pain as bad as can be
- Change in Small Fiber Neuropathy-Rasch Overall Disability Scale (SFN-RODS) score [ Time Frame: baseline and Week 28 ]The SFN-RODS is a 32-item scale measuring disability in daily activities. Scores range from 0 - 64, with a lower score correlating with worse disease
- Change in Small Fiber Neuropathy-Symptom Inventory Questionnaire (SFN-SIQ) score [ Time Frame: baseline and Week 28 ]The SFN-SIQ is a validated 13-item scale measuring various SFN and autonomic symptoms. Scores range from 0 -39, with a higher score correlating with more severe disease.
- Change in Small Fiber Neuropathy-Screening List (SFN-SL) score [ Time Frame: baseline and Week 28 ]The SFN-SL is a validated 21 item scale measuring frequency and severity of SFN and autonomic symptoms. Scores range from 0 - 84, with higher scores reflecting increased disease severity.
- Change in Utah Early Neuropathy Scale (UENS) examination scores [ Time Frame: baseline and Week 28 ]The UENS is a validated physical exam score from 0-42 points to look for small fiber neuropathy. It includes measures of sensation, reflexes, and strength in both lower extremities. A higher score indicates increased impairment.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04153422
|United States, Illinois|
|Northwest Community Healthcare|
|Arlington Heights, Illinois, United States, 60005|
|Principal Investigator:||Lawrence Zeidman, MD, FAAN||Loyola University Chicago|