Using skin biopsies to improve diagnosis and treatment of CIDP

Validation of Cutaneous Nerve Demyelination in Diagnosis and Treatment of CIDP

This study is testing if skin biopsies can help doctors diagnose and monitor treatment for people with chronic inflammatory demyelinating polyneuropathy (CIDP) better than traditional nerve tests.

Quick facts

• Study type: Observational
• Enrollment: 10 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Vanderbilt University Medical Center (Academic / other)
• Drugs / interventions: rituximab, Prednisone
• Locations: 1 site (Nashville, Tennessee)
• Trial ID: NCT05614128 on ClinicalTrials.gov

What this trial studies

This observational study aims to evaluate the effectiveness of skin biopsies in diagnosing chronic inflammatory demyelinating polyneuropathy (CIDP) compared to traditional nerve conduction studies. Participants, who are newly diagnosed or within six months of diagnosis, will undergo skin biopsies at baseline, three months, and six months after treatment with IVIG, an FDA-approved therapy for CIDP. The study seeks to identify demyelination in nerves and assess how nerve function improves with treatment. By comparing biopsy results with nerve conduction studies, the researchers hope to enhance diagnostic accuracy and treatment monitoring for CIDP.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older who have been diagnosed with definite or probable CIDP within the last 12 months.

Not a fit: Patients with other causes of polyneuropathy or those with conditions such as diabetes mellitus or multifocal motor neuropathy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to more accurate diagnosis and better monitoring of treatment response in patients with CIDP.

How similar studies have performed: While the approach of using skin biopsies for diagnosing CIDP is innovative, similar studies have not been widely reported, indicating this may be a novel approach.

Eligibility criteria

Inclusion Criteria:

* 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.

  2. 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.

  3. 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.

  4. Previous plasma exchange will be allowed as long as it is not continued during the study.

Exclusion Criteria:

* 1. Other causes of polyneuropathy, multifocal motor neuropathy, diabetes mellitus, alcohol, family history of neuropathy, monoclonal gammopathy or malignancy, history of drug or toxin exposure, which could reasonably cause neuropathy. Coexistent monoclonal gammopathy will also be used as an exclusion as these patients may not have the same response to IVIg.

  2. Any other disease that may cause neurological symptoms and signs or that may interfere with treatment or outcome assessments.

  3. 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.

  4. History of keloids or other reactions to local anesthetic making skin biopsies unsafe.

  5. Patients with the following laboratory results:
  1. Positive result at screening on any of the following viral markers: human immunodeficiency virus-1 or 2, or hepatitis B or C virus.
  2. 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.

  7. 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.

Where this trial is running

Nashville, Tennessee

• Peltier Amanda — Nashville, Tennessee, United States (Recruiting)

Study contacts

• Study coordinator: Amanda C Peltier

Conditions CIDP