Validation of Cutaneous Nerve Demyelination in Diagnosis and Treatment of CIDP

NCT05614128 | Terminated

• 1 other identifier: VR64081
• Study Type: observational
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this observational study is to learn about chronic inflammatory demyelinating polyneuropathy. The main question the investigators would like to answer is 1) can skin biopsy identify demyelination better than nerve conduction studies (electrical tests of the nerves)? and 2) how do nerves improve after treatment in CIDP? Participants will be asked to undergo skin biopsy of the finger at baseline and at 3 months and 6 months after treatment with IVIG (which is the FDA approved treatment for CIDP).

Conditions

CIDP

Outcome Measures

Primary Outcomes (1)

• Percentage of cutaneous nerves with segmental demyelination

  Number of nerve fibers with demyelination visualized under microscopy will be calculated per biopsy.

  6 months

Interventions

IVIg OTHER

Patients will be treated with IVIg as recommended by expert guidelines for treatment of CIDP under care of their primary neurologist. This will not be provided by the study.

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Newly diagnosed patients with CIDP prior or within 12 months of treatment with IVIg.

You may qualify if:

• Adults (age ≥ 18 years) with definite or probable CIDP according to the EFNS/PNS criteria may enter the trial within 12 months of treatment onset or treatment naïve patients. Written informed consent is obtained by the local investigator before entry into the study.
• \. IVIg treatment of 0.8 up to 1.2 g/kg per 30 days will be allowed up to 6 months. It is expected some patients may be on variable treatment regimens, and as along as they are not significantly declining, efforts will be made to continue treatment regimens already instituted.
• \. Prednisone in doses up to 20 mg /day will be allowed as long as dose has been stable for 90 days and is not being escalated or tapered during study.
• \. Previous plasma exchange will be allowed as long as it is not continued during the study.

You may not qualify if:

• \. Any other disease that may cause neurological symptoms and signs or that may interfere with treatment or outcome assessments.
• \. Severe conditions that may interfere with an evaluation of the study product or satisfactory conduct of the study such as current malignancy or history of allogeneic bone marrow/stem cell transplant, cardiac insufficiency (New York Heart Association Classes III/IV), cardiomyopathy, cardiac arrhythmia requiring treatment, unstable or advanced ischemic heart disease, congestive heart failure or severe hypertension, chronic kidney disease deemed too severe to safely use IVIg, known hyperprolinemia, known bleeding disorders, severe skin disease at the planned injection sites or biopsy site, alcohol, drug or medication abuse.
• \. History of keloids or other reactions to local anesthetic making skin biopsies unsafe.
• \. Patients with the following laboratory results:
• Positive result at screening on any of the following viral markers: human immunodeficiency virus-1 or 2, or hepatitis B or C virus.
• Abnormal laboratory parameters: creatinine greater than 1.5 times the upper limit of normal (ULN), blood urea nitrogen greater than three times the ULN if the increase is related to potential kidney disease, or hemoglobin less than 10 g/dL
• b. Abnormal laboratory parameters: creatinine greater than 1.5 times the upper limit of normal (ULN), blood urea nitrogen greater than three times the ULN if the increase is related to potential kidney disease, or hemoglobin less than 10 g/dL 6. Fulfilling the following general criteria: inability to comply with study procedures and treatment regimen; mental condition rendering the patient unable to understand the nature, scope, and possible consequences of the study; pregnancy or nursing mother; intention to become pregnant during the course of the study; female patients of childbearing potential either not using or not willing to use a medically reliable method of contraception for the entire duration of the study or not sexually abstinent for the entire duration of the study or not surgically sterile; participation in another clinical study or use of another investigational medicinal product within the same time period of the study.
• \. Additional medications and treatments other than prednisone and IVIg for treatment of CIDP such as azathioprine, mycophenolate, rituximab and ongoing plasma exchange as this will either confound or interfere with effects of IVIg.

Sponsors & Collaborators

• Vanderbilt University Medical Center: lead
• CSL Behring: collaborator

Study Sites (1)

Peltier Amanda

Nashville, Tennessee, 37232, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

2 mm punch skin biopsies will be obtained at the index finger on the palm side of the non-dominant hand.

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Interventions

Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Wende Fedder, Rn

  Vanderbilt University Medical Center

  STUDY DIRECTOR

• Diana Davis, RN

  Vanderbilt University Medical Center

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: October 31, 2022
• First Posted: November 14, 2022
• Study Start: February 28, 2023
• Primary Completion: September 19, 2024
• Study Completion: September 19, 2024
• Last Updated: June 5, 2025

Record last verified: 2025-06

Locations

US (1)