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 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 mNIS7 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 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 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.

This trial investigates the treatment of adults with Chronic Inflammatory Demyelinating Polyneuropathy CIDP. It compares the effects of empasiprubart and intravenous immunoglobulin IVIg to evaluate which treatment may better reduce symptoms and improve function in people with CIDP. The study is a Phase 3, randomized, double-blind trial designed to assess both efficacy and safety of these treatments over an extended period. Participants are randomly assigned in Part A to receive either empasiprubart with a placebo resembling IVIg or IVIg with a placebo resembling empasiprubart for 24 weeks 6 months. After Part A, all participants enter Part B, where they receive empasiprubart for an additional 96 weeks 24 months. During Part B, those previously receiving empasiprubart continue with it, and those initially on IVIg switch to empasiprubart. Treatments are administered by intravenous infusion using a double-dummy design to maintain blinding. Throughout the study, participants undergo regular assessments of their disability, strength, grip, and quality of life using various scales such as aINCAT, I-RODS, MRC-SS, and others. Safety is monitored by tracking adverse events and antibody formation against empasiprubart. The primary outcome is the reduction of at least one point in the aINCAT score at week 24. Total participation lasts up to 120 weeks, including both treatment periods, with ongoing evaluations to understand the long-term effects of empasiprubart.

Researchers are evaluating the efficacy and safety of empasiprubart in adults with Chronic Inflammatory Demyelinating Polyneuropathy CIDP. This Phase 3, randomized, double-blinded, placebo-controlled study compares empasiprubart to placebo to better understand its impact on CIDP symptoms and disease progression. The study has two parts Part A lasts 24 weeks 6 months, where participants receive either empasiprubart or placebo via intravenous infusion. After Part A, all participants enter Part B for 96 weeks 24 months during which everyone receives empasiprubart. Participants who received empasiprubart in Part A will receive a placebo dose once during Part B to maintain the study blind. Participants will have regular assessments including measurements of disability using the adjusted inflammatory neuropathy cause and treatment aINCAT score, grip strength, and other neurological and quality of life scales. Safety is monitored throughout the study. The total participation period spans up to 120 weeks, with evaluations at multiple time points to track changes from baseline and any adverse events.

This research aims to evaluate the safety of efgartigimod or efgartigimod PH20 SC exposure in pregnant women and breastfeeding mothers across multiple countries. It focuses on monitoring maternal, fetal, and infant outcomes related to these exposures, including rates of major congenital malformations. The study collects background data from similar populations for comparison. Participants include women who were exposed to efgartigimod or efgartigimod PH20 SC within 25 days before conception, during pregnancy, or exclusively during breastfeeding. Women who are no longer pregnant at enrollment but had prior exposure are included, as well as those currently pregnant or breastfeeding. During the study, researchers will observe pregnancy outcomes, congenital malformations, fetal growth issues, maternal complications and infections, and serious illnesses in newborns and infants. These outcomes will be tracked for up to 10 years, providing long-term safety data. Participants provide consent and are monitored through routine data collection without assigned treatments.

Leprosy is a chronic infectious disease caused by Mycobacterium leprae or Mycobacterium lepromatosis, mainly affecting the peripheral nerves and leading to neurological damage, disability, and deformities if not diagnosed and treated early. This research evaluates the diagnostic and prognostic accuracy of combining clinical evaluation, peripheral nerve ultrasonography, and molecular detection techniques using ultrasound-guided perineural subcutaneous aspirate. The goal is to improve early detection and monitoring of leprosy compared with traditional methods like bacilloscopy and biopsy. Participants with suspected leprosy will undergo clinical exams, high-resolution ultrasound of the ulnar nerves, and an ultrasound-guided subcutaneous aspirate near the nerve. This aspirate is tested using real-time PCR and RT-PCR to detect DNA and RNA of Mycobacterium leprae, which may help identify viable bacteria. The study involves assessments at diagnosis and after one year of treatment to compare findings and evaluate the new diagnostic methods accuracy. Throughout the one-year study, participants will have clinical and imaging evaluations, molecular testing of aspirated material, and monitoring of nerve changes. Researchers will measure the diagnostic accuracy of the molecular tests and correlate ultrasound findings with molecular results. This approach aims to develop improved, less invasive diagnostic workflows that can be used in public health settings to reduce disease transmission and long-term disability.

Transthyretin amyloidosis ATTR is a condition where the transthyretin protein breaks down and forms amyloid plaques that build up in organs, causing damage. There are two types wild-type ATTR, which occurs with aging, and variant ATTR caused by inherited defective TTR genes. This study focuses on adults who have inherited a pathogenic TTR gene variant but do not yet show symptoms, aiming to see if early treatment can prevent or delay disease development. The trial is a Phase 3, randomized, double-blind, placebo-controlled study evaluating prevention of ATTR cardiomyopathy and polyneuropathy. Participants will be randomly assigned to receive either acoramidis, a drug designed to stabilize the TTR protein and prevent amyloid formation, or a matching placebo. The study drug is taken orally twice daily at a dose equivalent to 800 mg acoramidis HCl BID. Participants will be adults aged 18 to 75 years whose age falls within 10 years younger or older than their predicted age of disease onset based on family history or actuarial tables. The trial will last approximately 7 years from randomization or until study end. During the study, participants will be monitored regularly with evaluations to detect the development of ATTR cardiomyopathy or polyneuropathy. Researchers will assess time to disease onset using centrally adjudicated criteria. Safety and adherence will be tracked throughout the trial. This long-term study will help determine if early treatment with acoramidis can delay or prevent symptoms in asymptomatic carriers of pathogenic TTR variants.