Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Researchers are investigating the relationship between amylin, a hormone increased in people with Type-2 diabetes, and peripheral neuropathy symptoms in patients with this condition. The study explores whether the amount of amylin in the upper limbs correlates with the severity of nerve damage symptoms. This observational study aims to better understand how diabetes-related vascular damage affects nerve function and may identify biomarkers for disease severity. Participants will provide blood samples and skin biopsies from the forearm to measure amylin deposition in blood vessels and red blood cells. The study also includes nerve conduction velocity tests and sensory assessments of pain and temperature response in the upper extremities. These tests occur at the time of sample collection and follow-up sensory testing one week later. Throughout the study, participants undergo clinical sensory exams, nerve conduction velocity testing, and pain threshold evaluations using pressure, cold, and heat stimuli. Researchers will compare amylin levels to symptom severity and nerve function. The study collects data at single time points and includes follow-up sensory testing to provide preliminary insights for larger trials. Total participation involves initial testing and follow-up assessments one week later.

Researchers are evaluating Claseprubart (DNTH103) in adults with multifocal motor neuropathy (MMN) to assess its safety, tolerability, pharmacometrics, and effectiveness. This Phase 2 randomized, double-blinded, placebo-controlled study aims to better understand how this drug works for people with MMN and to monitor any treatment-related side effects. The sponsor of this trial is Dianthus Therapeutics. Participants receive either Claseprubart or a placebo. On the first day, they get an intravenous loading dose, followed by subcutaneous doses every two weeks from Week 1 to Week 15. The study includes multiple groups receiving either 300 mg or 600 mg doses of Claseprubart, or placebo, to compare outcomes. This design helps researchers evaluate different doses and their effects. During the study, participants will be closely monitored from baseline up to Week 17 for side effects and treatment response, including grip strength, muscle function scores, disability scales, and quality of life measures. Some assessments continue up to Week 52 in an open-label extension period. Blood samples will be collected to analyze drug concentration and immune responses. Safety and effectiveness data are gathered through various clinical tests and questionnaires, ensuring comprehensive participant evaluation throughout the study.

Researchers are evaluating a new rehabilitation process to assess and relieve chemotherapy-induced peripheral neuropathy in the feet of patients treated for early-stage breast cancer. This study compares two orthopedic supports: a standard treatment using customized insoles and shoes, and a novel silicone orthosis designed as a soft, sock-like device for indoor use. The goal is to understand if the silicone orthosis can reduce symptoms and improve function compared to standard treatment. Participants will be randomly assigned to receive either the customized insoles and shoes or the silicone orthosis along with the standard treatment. The study involves initial assessments including symptom grading, foot health questionnaires, quality of life scales, and 3D foot scanning. Activity levels will be tracked using an activity sensor worn on the leg for two weeks. Participants using the silicone orthosis will keep a diary of device usage. Follow-ups occur 3 to 6 weeks after starting the intervention to evaluate symptom changes and patient experiences. During the study, participants will undergo foot examinations and complete questionnaires about their symptoms, foot health, and quality of life. Mobility and activity patterns will be monitored objectively with activity trackers. Follow-up may be conducted in person, by phone, or digitally, lasting about 45 minutes. The main outcomes include changes in neuropathy symptoms and physical activity after 3 to 6 weeks of using the orthopedic devices. This research aims to improve rehabilitation care for breast cancer survivors experiencing foot neuropathy from chemotherapy.

Researchers are studying the safety and effectiveness of a single dose of nexiguran ziclumeran (NTLA-2001) compared to a placebo in people with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This phase 3, multinational, randomized, double-blind, placebo-controlled trial involves about 60 participants with this nerve disease and genetic condition affecting the peripheral nervous system. Participants will be randomly assigned to receive a single intravenous infusion of either nexiguran ziclumeran 55 mg or a placebo of normal saline. To give everyone a chance to receive the study drug, participants may switch to the other treatment group at either 12 or 18 months, depending on specific study criteria. The study is designed to compare the effects of the drug and placebo over time. During the study, participants will be monitored for nerve function using the Modified Neuropathy Impairment Score +7 (mNIS+7) over 18 months and blood levels of serum transthyretin at 29 days and 18 months. Quality of life, body mass index, and other health measures will also be evaluated. The study includes careful safety monitoring and will last up to 18 months with ongoing assessments to track participants' nerve health and overall well-being.

Researchers are conducting a multicenter, prospective observational study to understand the natural history of Charcot-Marie-Tooth Disease, Type 4J (CMT4J), a rare neurodegenerative and neuromuscular condition confirmed by specific genetic testing. The study will enroll 20 participants of any age who have a molecularly confirmed diagnosis of CMT4J. The purpose is to collect detailed health information and disease progression data over time, without providing any investigational treatments. Participants will follow a uniform protocol with visits scheduled every 12 months plus a 4-week window, for up to 2 years. At each visit, researchers will collect demographic and medical history data, perform physical and neurological exams, standard lab tests, and use several disease outcome measures. Additional assessments may include neuropsychological tests, nerve conduction studies, muscle MRI, pulmonary function tests, and scoliosis x-rays. Early termination visits are planned if participants leave before completing the study. Throughout the study, participants will undergo thorough evaluations to monitor their condition and collect data relevant to CMT4J progression. This includes clinical exams, imaging, lab tests, and questionnaires. Researchers will use this information to study disease patterns and outcomes over the 2-year follow-up period. No investigational products are administered, and visits may be unscheduled if necessary with approval. Participants are expected to comply with study procedures and travel requirements.

Researchers are studying how nerve test results change in people with urothelial carcinoma who are treated with enfortumab vedotin. This observational study focuses on assessing nerve function and patient-reported neuropathy in individuals with metastatic or muscle-invasive urothelial carcinoma. The study is led by Memorial Sloan Kettering Cancer Center and aims to understand chemotherapy-induced peripheral neuropathy linked to this treatment. Participants will undergo non-invasive nerve conduction studies to objectively measure nerve function, especially in sensory fibers affected by neuropathy. They will also provide self-reported information about their neuropathy symptoms. The study includes individuals planned to start systemic therapy with enfortumab vedotin, with or without pembrolizumab. During the study, nerve conduction tests and questionnaires about neuropathy will be collected from baseline up to 9 to 15 weeks. Researchers will monitor changes in nerve function and neuropathy severity over this period. Participants must be able to communicate in English and have a performance status allowing study participation. The study does not involve therapeutic interventions but focuses on observation and assessment of nerve health during treatment.

Researchers are evaluating the safety and effectiveness of NT 201, a type of botulinum toxin A, compared with a placebo in adults who have moderate to severe platysma prominence, which refers to noticeable bands in the neck area. This Phase 3 study aims to understand how well NT 201 works and how safe it is for these participants. The study is sponsored by Merz Aesthetics GmbH and will be conducted in Europe. Participants will be randomly assigned to receive either a single injection of NT 201 or a matching placebo injection in the main study period (MP) on Day 1. After this period, those who received NT 201 can enter an open-label extension period (OLEX) where they will receive another injection of NT 201 on Day 1 of that phase. The study involves a double-blind design, meaning neither the participants nor the researchers know who receives the active drug or placebo during the main period. During the study, participants will be assessed using the Merz Aesthetics Platysma Scale - Dynamic (MAPS-D) to measure improvement in platysma prominence at various time points, including Week 2 and up to Week 17 in the main period. Both investigator and participant assessments will be used to evaluate the degree of improvement. Satisfaction questionnaires and global aesthetic improvement scales will also be used to measure participant satisfaction and aesthetic changes. Safety and efficacy will be closely monitored throughout the study, which lasts through the main and extension periods.

Researchers are evaluating a new device called a real-time pressure controller designed to prevent peripheral neuropathy caused by chemotherapy with albumin paclitaxel in breast cancer patients. This device aims to improve upon existing treatments by precisely controlling pressure that gradually decreases from the fingers or toes to the wrists or limbs. The study addresses the need for a more acceptable and effective method for Chinese patients, who often have poor tolerance for current cold compresses or pressure gloves. The study compares two groups: one using the real-time pressure controller device and one with no intervention. This device applies controlled pressure in a sequential manner to reduce nerve damage caused by paclitaxel chemotherapy. The study is randomized and open-label, with no masking. Its goal is to assess how well this device can reduce chemotherapy-induced peripheral neuropathy symptoms and improve patients' ability to continue treatment. Participants will be monitored through various assessments including changes in tactile sensitivity and the Patient Neurotoxicity Questionnaire to evaluate the presence and severity of neuropathy. The primary outcome will be measured over about one year from the start of the study. Safety and effectiveness will be closely observed by the study team. The trial will include adults aged 18 to 75 years who are suitable for albumin paclitaxel treatment and have good overall health status.

Researchers are evaluating the effectiveness and safety of empasiprubart compared to intravenous immunoglobulin (IVIg) for adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). This Phase 3 randomized, double-blinded, double-dummy study aims to compare these two intravenous treatments over a six-month period and further assess empasiprubart over an extended 24-month follow-up. The study is divided into two parts. In Part A, lasting 24 weeks, participants receive either empasiprubart with a placebo mimicking IVIg or IVIg with a placebo mimicking empasiprubart. After completing Part A, all participants move to Part B, which lasts 96 weeks, where everyone receives empasiprubart. This design allows comparison in the first phase and extended evaluation of empasiprubart in the second phase. Participants will undergo regular assessments throughout both parts, including measurements of muscle strength, disability scores, grip strength, and quality of life. Researchers will monitor changes in clinical scales such as the aINCAT score to evaluate improvements or declines in symptoms. Safety is also closely tracked by recording adverse events and antibody responses. The total participation time spans up to 120 weeks, combining both study parts.

The trial investigates the effectiveness and safety of empasiprubart in adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), including typical and certain variant forms of the condition. Participants must have active disease with some residual disability and may be treatment-naive or currently receiving CIDP treatments. The study is a Phase 3, randomized, double-blinded, placebo-controlled trial sponsored by argenx. During part A, participants are randomly assigned to receive either empasiprubart or a placebo via intravenous infusion for 24 weeks (6 months). In part B, all participants receive empasiprubart for 96 weeks (24 months). Those initially receiving empasiprubart will receive a placebo once in part B to maintain the study's blind. The study evaluates various measures of disability, strength, and quality of life over these periods. Participants will have their disability measured using the adjusted INCAT score at 24 weeks as the primary outcome. Additional assessments include muscle strength, grip strength, walking tests, and patient-reported outcomes. Safety is monitored through adverse event tracking and blood tests for antibodies against empasiprubart. The total participation spans up to 120 weeks, including both parts A and B, with regular visits for treatment and evaluations.