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Polyneuropathies clinical trials

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NCT ID: NCT06377033 Not yet recruiting - Parkinson Disease Clinical Trials

Using the EHR to Advance Genomic Medicine Across a Diverse Health System

Start date: May 1, 2024
Phase: N/A
Study type: Interventional

Given the expansion of indications for genetic testing and our understanding of conditions for which the results change medical management, it is imperative to consider novel ways to deliver care beyond the traditional genetic counseling visit, which are both amenable to large-scale implementation and sustainable. The investigators propose an entirely new approach for the implementation of genomic medicine, supported by the leadership of Penn Medicine, investigating the use of non-geneticist clinician and patient nudges in the delivery of genomic medicine through a pragmatic randomized clinical trial, addressing NHGRI priorities. Our application is highly conceptually and technically innovative, building upon expertise and infrastructure already in place. Innovative qualities of our proposal include: 1) Cutting edge EHR infrastructure already built to support genomic medicine (e.g., partnering with multiple commercial genetic testing laboratories for direct test ordering and results reporting in the EHR); 2) Automated EHR-based direct ordering or referring by specialist clinicians (i.e., use of replicable modules that enable specialist clinicians to order genetic testing through Epic Smartsets, including all needed components, such as populated gene lists, smartphrases, genetic testing, informational websites and acknowledgement e-forms for patient signature); 3) EHR algorithms for accurate patient identification (i.e., electronic phenotype algorithms to identify eligible patients, none of which currently have phenotype algorithms present in PheKB; 4) Behavioral economics-informed implementation science methods: This trial will be the first to evaluate implementation strategies informed by behavioral economics, directed at clinicians and/or patients, for increasing the use of genetic testing; further it will be the first study in this area to test two forms of defaults as a potential local adaptation to facilitate implementation (ordering vs. referring); and 5) Dissemination: In addition to standard dissemination modalities,PheKB95, GitHub and Epic Community Library, the investigators propose to disseminate via AnVIL (NHGRI's Genomic Data Science Analysis, Visualization, and Informatics Lab-Space). Our results will represent an entirely new paradigm for the provision of genomic medicine for patients in whom the results of genetic testing change medical management.

NCT ID: NCT06360289 Not yet recruiting - Clinical trials for Hereditary Amyloidosis, Transthyretin-Related

Observational Study of Neurofilament Light Chain (NfL) as a Biomarker in Asymptomatic Carriers of the Transthyretin (TTR) Variants and Patients With Hereditary Transthyretin-mediated (hATTR) Amyloidosis With Polyneuropathy

Start date: April 2024
Phase:
Study type: Observational

This is a single-center observational study evaluating the potential value of NfL as a biomarker for diagnosis, detection of disease onset, monitoring of disease progression, and treatment response in asymptomatic carriers of TTR variants and symptomatic hATTR amyloidosis patients with polyneuropathy.

NCT ID: NCT06290141 Not yet recruiting - Clinical trials for Chronic Inflammatory Demyelinating Polyneuropathy

A Study to Test the Efficacy and Safety of Riliprubart Against the Usual Treatment of Intravenous Immunoglobulin (IVIg) in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

VITALIZE
Start date: April 18, 2024
Phase: Phase 3
Study type: Interventional

The purpose of the study is to evaluate efficacy of riliprubart compared to IVIg in adult participants with CIDP who are receiving maintenance treatment with IVIg. The study duration will be for a maximum of 109 weeks including screening, treatment phases, and follow-up.

NCT ID: NCT06033508 Not yet recruiting - Type 2 Diabetes Clinical Trials

Effects of Whole-body Vibration Therapy on Type 2 Diabetic Patients With and Without Polyneuropathy

Start date: February 1, 2024
Phase: N/A
Study type: Interventional

The aim of this study is to show how whole body vibration training practiced for 12 weeks affects the levels of inflammatory biomarkers such as C reactive protein (CRP) and, interleukin-6 (IL-6), in Type 2 diabetic patients (T2DM) with and without peripheral neuropathy.

NCT ID: NCT05940922 Not yet recruiting - Clinical trials for Guillain-Barre Syndrome

RWE-based Treatment Patterns and Outcomes in CIDP

Start date: August 17, 2023
Phase:
Study type: Observational

To describe the demographics, clinical characteristics, treatment patterns and clinical outcomes of chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain-Barre Syndrome (GBS), and heredofamilial amyloidosis (hATTR) adult patients at a single U.K. centre.

NCT ID: NCT05939037 Not yet recruiting - Clinical trials for Monoclonal Gammopathy of Uncertain Significance

Zanubrutinib Treatment in Patients With IgM Monoclonal Gammopathy and Antri-MAG Related Polyneuropathy

MAGNAZ
Start date: October 1, 2023
Phase: Phase 2
Study type: Interventional

The 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.

NCT ID: NCT05219383 Not yet recruiting - Clinical trials for Chronic Inflammatory Demyelinating Polyneuropathy

Clinical and Electrophysiological Patterns of Chronic Dysimmune Polyneuropathy

Start date: January 2023
Phase:
Study type: Observational

Chronic dysimmune neuropathies (CDN) are a heterogenous group of acquired inflammatory demyelinating neuropathies including chronic inflammatory demyelinating polyneuropathies (CIDP), Lewis-Sumner Syndrome (LSS), multifocal motor neuropathy (MMN) and other rare entities. Despite their relatively low prevalence, CDN lead to substantial costs for patients and society. CDN are usually misdiagnosed due to progressive nature of the disease with little known data regarding disease activity and treatment response

NCT ID: NCT05116111 Not yet recruiting - Clinical trials for Distal Symmetric Polyneuropathy (DSPN)

A Study To Evaluate the Safety, Tolerance, Pharmacokinetics/Pharmacodynamics and Efficacy of SYHA1402 Tablets For the Treatment of Patients With Diabetic Peripheral Neuropathy in China

Start date: December 31, 2021
Phase: Phase 2
Study type: Interventional

This study aims to evaluate the safety, tolerance, pharmacokinetics/pharmacodynamics and efficacy of SYHA1402 tablets for the treatment of patients with diabetic peripheral neuropathy (DPN) in China.

NCT ID: NCT04985734 Not yet recruiting - Heart Failure Clinical Trials

Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) in Patients With Idiopathic Peripheral Neuropathy

Start date: October 1, 2021
Phase: N/A
Study type: Interventional

The goal of the present study is to determine the occurrence of wild-type and hereditary transthyretin amyloidosis cardiomyopathy among patients with the diagnosis of idiopathic peripheral neuropathy in the setting of a state-of-the-art diagnostic work-up; the investigators believe that the identification of patients with ATTR-CM in this setting can contribute to the early diagnosis of a largely underrecognized condition and, therefore, offer conditions to timely initiation of appropriate therapy with impact on prognosis of patients.

NCT ID: NCT04978623 Not yet recruiting - Clinical trials for Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Role of High Frequency Ultrasound in Demyelinating Polyneuropathies

Start date: January 1, 2022
Phase:
Study type: Observational

The use of neuroimaging in the evaluation of neuromuscular pathologies is a promising and rapidly expandinf field. Among the different imaging techniques employed, nerve ultrasound is of particular interest given it's non invasivity, lack of side effects, accesibility and low cost. This exam provides complementary data to electroneuromyography (ENMG), enabeling the physician to visualise the morphological correspondents of electrophysiological abnormalities with a spatial resolution at least as good as MRI. Chronic Inflammatory Demyelinating Polyradiculoneuritis (CIDP), Multifocal Motor Neuropathy with Conduction Blocks (MMNBC), Lewis and Sumner syndrome (L-S syndrome) and anti-MAG antibody-associated neuropathies (anti-MAG) are among the main chronic inflammatory neuropathies of autoimmune etiology. The diagnosis of these diseases is based on clinical, paraclinical and ENMG examinations. Several recent studies have highlighted the valuable role of ultrasound not only in the diagnosis but also for follow up of patients afflicted by this group of diseases. However, the current ultrasound imaging techniques does not allow for a detailed study of the internal structure of the nerves. The developement of technologies with improved resolution contribute to further the knowledge in this innovative field and to better understand the pathophysiological mechanisms responsible for initiation and progression of inflammatory nerve disorders.In the Department of Ultrasound at the Nice University Hospital in France the team have at its disposal a high frequency untrasound that the team use to explore the peripheral nervous system. The scientific hypothesis of this pilot study rests on the premise that demyelinationg neuropathies are a heterogenous group where different types present with different morphological characteristics not only at the level of the nerve as a whole, but also at the level of the nerve fascicles and of the peri- and intra-neural vascular structures. In this respect, high frequency ultrasound allows us to visualise different segments of affected nerves and to obtain morphological details which the team then compare with existing ultrasound data in the literature (high frequency ultrasound: 10-20 MHz). Thanks to this non-invasive and painless technique, the nerve can be rapidly visualised along its entire length in static and dynamic mode. In addition, high frequency ultrasound will give us access to complementary data on the morphology of the soft tissues and the peri- and intra-neural vascular structures which may be useful in shedding light on the underlying pathophysiologycal processes. Another important aspect is the opportunity to make electromyographic and clinical correlations The main parameter The team will study in ultrasound will be hypertrophy, corresponding to an increase in the cross-sectional area of the nerve. the team will also conduct analysis on other less codified parameters, such as epineural and endoneural vascularisation (using the Doppler mode), as well as nerve fascicles anatomy (size, organisation). Identification of different characteristics may be of diagnostic interest in cases where the clinical, paraclinical and ENMG examinations do not allow us to make a diagnostic and therapeutic decision, but it can also prove useful in the follow-up of patients and response to treatment. This is a descriptive, pilot, mono-centric, multiparametric and retrospective analysis study on patients followed in our centre with a diagnosis of CIDP, NMMBC, L-S syndrom and anti-MAG