Intravenous immunoglobulin therapy for small fiber neuropathy: a randomised, double-blind, placebo-controlled study on efficacy and safety.

Phase 1

• Conditions: Skin-biopsy proven small fiber neuropathy without an underlying cause; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2015-002624-31-NL
• Lead Sponsor: Maastricht UMC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-11-16
• Last Update Posted: 12 December 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

•18 years or older.
•Skin-biopsy proven idiopathic SFN or idiopathic painful neuropathy with predominantly SFN pattern
•Pain intensity rated = 5 on the PI-NRS (maximum pain) or on the neuropathic pain scale,36,37 question number 1 for at least 12 weeks before the study as declared by each patient to the best of their knowledge; if available, medical records of each patient will be consulted on the reported pain intensity.
•Each subject will receive an information leaflet and an informed consent form. Subjects must give informed consent by signing and dating prior to study entry.
•Eligible patients must be willing to complete all study-related activities and examination required by the protocol

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

•Are unable or unwilling to provide written informed consent.
•Have predominant clinical picture of large nerve fiber involvement (i.e., weakness, loss of vibration sense, hypo-/areflexia).
•Had treatment with IVIg or any other immunomodulatory/immunosuppressive agents (e.g., steroids) within the last 12 weeks prior to the date of informed consent.
•Have an underlying cause of SFN (diabetes, SCN9A/10A/11A mutations, hypothyroidism, renal failure, vitamin B12 deficiency, monoclonal gammopathy, alcohol abuse (more than 5 IU/day), malignancies, drugs that cause neuropathy (e.g. chemotherapy, amiodarone, propafenone)).
•Have a history of anaphylaxis or severe systemic response to immunoglobulin or with a blood product.
•Have cardiac insufficiency (NYHA III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease, or history of congestive heart failure, severe hypertension (diastolic blood pressure >120 mmHg or systolic >170 mmHg).
•Are females who are pregnant, breast-feeding, or if of childbearing potential, or unwilling to practice adequate contraception throughout the study.
•Have known hyperviscosity.
•Have a history of renal insufficiency or high serum creatinine levels (MDRD <30).
•Have known selective IgA deficiency.
•Have conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome).
•Have a known hypercoagulable state.
•Are mentally challenged adult subjects unable to give independent informed consent.
•The use of pain (analgesic/anti-neuropathic) medication is allowed, but only if dosages are remained unchanged for at least 30 days prior to randomization. A change in dosage of these drugs will not be allowed throughout the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of IVIg treatment (4 courses of treatment, 3 weeks apart) compared to placebo by assessing changes on the mean weekly level of peak pain intensity (PI-NRS) compared to baseline scores.;Secondary Objective: Corneal confocal microscopy, pain intensity, pain qualities, and other SFN related complaints, daily and social functioning, as well as quality of life will be assessed.<br>Safety: adverse events, vital signs and laboratory values outside the normal range;Primary end point(s): Comparison of the percentage of responder subjects between the two treatment groups from the first randomization during 12 weeks. A patient withdrawing from the study will be recorded as treatment failure for their randomization arm.;Timepoint(s) of evaluation of this end point: During the complete length of the study (12 weeks)