Prevalence of Wild-type TTR Cardiac Amyloidosis in Patients With Polyneuropathy of Unknown Cause....
PolyneuropathyAmyloidosisTo investigate to what extent chronic axonal length-dependent polyneuropathy (CAP) and/or small-fiber neuropathy (SFN) is part of early non-cardiac manifestations of wild-type TTR cardiac amyloidosis (wtTTR-CA). Consequently, explore whether this could ultimately lead to faster diagnosis and clinical outcome of wild-type TTR cardiac amyloidosis (wtTTR-CA).
Peripheral Nerve Block in Patients With Painful Diabetic Polyneuropathy
Painful Diabetic NeuropathyNeuropathic Pain1 moreThe purpose of the present study is to evaluate the role of peripheral afferent input for spontaneous pain in painful diabetic polyneuropathy
Baby Detect : Genomic Newborn Screening
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1134 moreNewborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple blood test, rare genetic conditions can be easily detected, and the early start of transformative treatment will help avoid severe disabilities and increase the quality of life. Baby Detect Project is an innovative NBS program using a panel of target sequencing that aims to identify 126 treatable severe early onset genetic diseases at birth caused by 361 genes. The list of diseases has been established in close collaboration with the Paediatricians of the University Hospital in Liege. The investigators use dedicated dried blood spots collected between the first day and 28 days of life of babies, after a consent sign by parents.
Comparison of FEETME® Soles and GAITRITE® Walkway for the Evaluation of Gait Disorders in CIDP....
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)Chronic Inflammatory Demyelinating Polyneuropathies (CIDP) are acquired, autoimmune and inflammatory neuropathies leading mainly to gait disorders in patients. Assessment of gait disorders is a major component of therapeutic management, but evaluation is sometimes difficult due to the fluctuating nature of the symptoms. The principal objective of this study is to compare the walking speed of patients with a reference method (GAITRITE® walkway) and an innovative one (FEETME® connected soles) with the aim of validating the use of theses connected soles in CIDP gait disorders evaluation.
Zanubrutinib Treatment in Patients With IgM Monoclonal Gammopathy and Antri-MAG Related Polyneuropathy...
Monoclonal Gammopathy of Uncertain SignificanceThe objective of this investigator-initiated phase II single-arm open-label clinical trial is to investigate neurological response rate, safety and tolerability of Zanubrutinib 320 mg daily in combination with Rituximab 375 mg/m2 (standard therapy) for the treatment of immunoglobulin M monoclonal gammopathy of unknown significance (IgM MGUS) related polyneuropathy with Myelin Associated Glycoprotein antibodies (anti-MAG). 42 adult patients will be included in two Dutch hospitals (University Medical Center Utrecht and Amsterdam University Medical Center). This trial consists of a 6-month treatment period, after which the hematological response will be evaluated. Adequately responding participants (at least partial response) will be treated for an additional 6 months, after which hematological response will be re-evaluated. Participants with at least a very good partial response will remain on treatment. Non-responding participants will be followed for clinical outcomes only. The total study period per participant will be 36 months.
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyTransthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy1 moreThis is a follow-up study of subjects who received NTLA-2001 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
Electronic Monitoring of Disease Activity in Patients With Chronic Inflammatory Demyelinating Polyneuropathy...
CIDPThis clinical, prospective study aims to evaluate the usefulness of electronically captured wearable data to assess disease activity in CIDP (chronic inflammatory demyelinating polyneuropathy) patients undergoing IVIG treatment. Close clinical monitoring will complement smartwatch-acquired data to shed light on different patient clusters and end-of-dose phenomena.
IVIG in the Treatment of Autoimmune Small Fiber Neuropathy With TS-HDS, FGFR-3, or Plexin D1 Antibodies...
Small Fiber NeuropathyAutoimmune Small Fiber Neuropathy2 moreThis study will enroll patients with small fiber neuropathy (SFN). The study will look at an intravenous immunoglobulin (IVIG) called Panzyga. Panzyga is approved by the FDA as a therapy for Primary humoral immunodeficiency (PI) in patients 2 years of age and older; Chronic immune thrombocytopenia (ITP) in adults and Chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. It has not been approved by the FDA for use in SFN. 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.
IVIG vs SCIG in CIDP
CIDPImmunoglobulin Deficiency1 moreCurrent dosing practices for immunoglobulin G (IgG) may be inadequate in extreme body weight. The current study will evaluate the influence of body composition on intravenous and subcutaneous administration of immunoglobulin G in patients.
Impact Of Sensory Re-Education Paradigm On Sensation And Quality Of Life In Patients Post-Covid...
Post-COVID-19 SyndromeThis study will be conducted to investigate the effect of a sensory re-education paradigm on sensation and proprioception in patients with post covid-19 neuropathy.