TRITON-PN is a global Phase 3 randomized, open-label study designed to evaluate the Efficacy and Safety of Nucresiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (hATTR-PN). The study will also evaluate any changes in quality of life experienced by study participants. Nucresiran is a TTR silencer that aims to prevent…
The purpose of this study is to evaluate the safety, tolerability and effectiveness of VX-548 in participants with pain due to Diabetic Peripheral Neuropathy (DPN). To be in this study you must have DPN and be 18 through 80 years of age, inclusive, with presence of bilateral pain in lower extremities due to DPN for at least 1 year.
This is a Phase 3, randomized, double blind, study evaluating efficacy and safety of riliprubart versus intravenous immunoglobulin (IVIg) in participants with chronic inflammatory demyelinating polyneuropathy. Overall the study will last about 109 weeks (2 years). Participants must be 18 years old at the time of signing the informed consent form.
This is a global, multicenter, placebo-controlled, phase 3 study evaluating the efficacy and safety of riliprubart in adult participants with CIDP who are refractory or had an insufficient response to standard of care (SOC) therapies, defined as Ig administered IV (IVIg) or SC (SCIg), or corticosteroids. Treatment duration will be 48 weeks. Participant must…
This program provides family members of individuals with familial ALS the opportunity to contribute to research focused on learning more about why motor neuron degeneration begins and how or why it progresses. This study provides genetic counseling and testing to help participants understand and manage their risk and determine if they want to learn their…
We are doing a study to learn more about the progression of knee osteoarthritis (OA). We are recruiting patients (Men 45-85 years old; Women 55-85 years old) who may or may not have OA of the knee(s). This study involves a screening and enrollment visit with annual visits for 3 years. Study activities include a hip and knee assessment, questionnaires, two…
The purpose of this research is to study whether Discogen's non-invasive, low-intensity pulsed ultrasound treatment can help reduce pain for people with leg pain and low back pain. Eligible participants will be randomized into either a Discogen treatment group or a sham control group. All participants will be asked to come to the Columbia University…
Depression and some antidepressants may be associated with an increased risk of breaking a bone. It is not clear why this could be the case. It is possible that depression and/or certain medications used for the treatment of depression cause bone loss, increase the risk of falls, are associated with lower physical activity, or effects on muscle function.…
Columbia University is currently enrolling in an Ataluren Study from PTC Therapeutics, called PTC-041. We are looking for 5 year old boys, ambulating, confirmed nonsense mutation DMD and on steroids for at least 12 months. If you would like more information about this please reach out to Ameneh Onativia, Columbia Sr. Clinical Research Coordinator II at 212…
The purpose of this study is to Assess Treatments for Painful Diabetic Peripheral Neuropathy (PDPN). To be in this study you must have type II Diabetes Mellitus, PDPN, must be 18 years of age or older, and be able to stop taking some of your current pain medications including pills, creams, patches, marijuana, cannabidiol (CBD) oil, etc. Acetaminophen (…
Precision Medicine Approaches to Renal Osteodystrophy is a study designed to discover new markers of how the kidney affects your bone health. This study will evaluate how your bone is reacting to your kidney problem both before and after you start your bone treatment for hyperparathyroidism or osteoporosis. We are looking for people who are interested in…
We are studying the causes of and potential treatments for pregnancy & lactation-associated osteoporosis (PLO). We know little about the clinical features, causes, and prognosis of PLO. Our goal is to define the clinical characteristics of PLO and better understand similarities/differences among the women. We are looking for women with fractures during/…
Pain is a natural response to injury to let us know there may be damage to our bodies. Pain is the way our body tells us that we must take care of ourselves. Chronic pain is different and lasts for a long time. In some cases, chronic pain remains without any sign of body damage. Today, there is no way for a doctor to measure someone's chronic pain or…
The goal of this study is to learn whether treatment with a safe, commonly used drug called ATENOLOL prevents bone loss in women after menopause. Who Qualifies? Healthy post-menopausal women who are between the ages of 50 and 75. You may not be eligible if you have certain medical conditions. What's Involved? If you are eligible, you will be assigned…
The shoulder joint is the most mobile joint in the human body, steered by a group of muscles and their respective tendons, together called the rotator cuff. A consequence of this mobility is a vulnerability to degradation, or wear and tear, of tendons and inflammation in the surrounding area. Our study aims to create biological samples and data bank to get…
The purpose of this study is to evaluate the efficacy and safety of ISIS TTR Rx given for 65 weeks in patients with Familial Amyloid Polyneuropathy (FAP). The purpose of this study is to determine if ISIS 420915 can help people with mild or moderate FAP. To be in this study you must have FAP and be able to walk without help or walk with using only one cane.
Juvenile fibromyalgia (JFM) is a cause of chronic, whole-body pain. The cause of JFM is unknown. One of the possible causes of JFM is a problem with nerves called "small fiber neuropathy." This is a problem where small nerve fibers in the skin are not working properly and patients can have pain as a result. Recent studies have found evidence of…
International GBS Outcome Study (IGOS) is a study of the Inflammatory Neuropathy Consortium (INC) and Peripheral Nerve Society (PNS) on disease course and outcome in Guillain-Barr syndrome (GBS). Guillian Barre syndrome is an autoimmune disorder affecting the peripheral nervous system, leading to paralysis and potential fatality if it progresses to involve…
The purpose of this study is to look for abnormal genes and gene expression profiles that help determine why a person develops amyotrophic lateral sclerosis (ALS) and related motor neuron diseases (MND) and why their symptoms present and progress with a particular pattern. For up to three years, subjects will be followed every 3 months at the Columbia ALS…
Charcot-Marie-Tooth type 1A (CMT1A) disease is a rare, slowly progressing and debilitating hereditary condition commonly causing weakness of muscles in the feet and lower parts of the legs. There is currently no approved drug to cure CMT1A disease, and the available treatments commonly target the pain and inflammation associated with the disease. In order…
We are doing this study to find out if the research drug known as Rozanolixizumab can have additional benefit for people who are already receiving IVIG( Intravenous Immune Globulin) treatment for CIDP, and to have further safety evaluation of the research drug as well. If you choose to be part of this research study, the following procedures will occur:…
Peripheral Neuropathy is a result of nerve damage, which may cause weakness, numbness, tingling, and pain in your hands, feet, and other areas of your body. Over 20 million Americans are affected by peripheral neuropathy, most commonly caused by diabetes. In approximately 25-50% of all people with peripheral neuropathy, the cause is unknown. These cases…
We are looking for people ages 65 and older to help us evaluate if an investigational drug improves muscle function, which can reduce risk of falls and fractures. To be considered for the study, you: 1. Must complete a pre-screening phone interview to assess your eligibility 2. If you are eligible, you will be invited to attend an in-person appointment at…
Small fiber neuropathy is a painful condition caused by damage to the small nerves in your arms andlegs. This damage causes burning, sharp-stabbing and aching pain, amongst other types of pain. It can be sometimes difficult to know the cause of the damage to the small nerves in your arms and legs. In most cases, treatment for small fiber neuropathy is…
This study is looking at using topiramate as a potential therapy for patients with cryptogenic sensory peripheral neuropathy. Patients who are randomized into the study will return to the clinic for follow up every 16 weeks. The total length of the study is 96 weeks.
The purpose of this study is to see how effective two doses of an investigational new drug called IgPro20 is when given subcutaneously (a shot given into the fat layer between the skin and the muscle) to treat CIDP in patients that still require IVIG and also to look at how safe the IgPro20 is for patients.
The purpose of this research study is to test an investigational drug called NP001. The study will evaluate if NP001 has an effect on slowing the signs and symptoms of your ALS, if there are any side effects, and if there are any medical problems with giving the drug as a solution into your vein (intravenous IV administration). An investigational drug is…
The purpose of this study is to learn more about the bones in children who are short. This study will help to see if the bones of short children who have low levels of growth hormone or children who are short but have normal levels of growth hormone are any different from children of average height. Bones have spongy inner parts and harder outer parts and…
The purpose of this study is to collect information about how patients with FAP respond to the study drug ALN-TTR02 over 18 months. This includes studying any potential improvements in FAP and any side effects that participants may have from the study drug. In addition, the study will examine how the body handles (distributes, break down, eliminates) the…
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves your muscle weakness associated with MG symptoms. A placebo is a medication that looks like the study medication but has no active ingredient. Immunoglobulin is an antibody produced by white blood cells that is used by the immune…
You are being considered to participate in this research study because you have type I or II diabetes with current treatment control and, you are experiencing painful diabetic peripheral neuropathy (DPN) in both lower extremities legs.The specific events that result in painful diabetic peripheral neuropathy are not well understood, but high blood sugar,…
The purpose of this study is to evaluate the efficacy and safety of study drug, fingolimod taken daily compared with a placebo (does not contain active medication) on delaying disability progression in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The study will consist of 3 periods: a Screening Period (lasting for up to 45…
This study is designed to test how well an intravenous human immunoglobulin concentrate, IGIV-C (compared with placebo) improves the ability to taper participant's dose of corticosteroids without causing significant increase in your muscle weakness and worsening MG symptoms. A placebo is a medication that looks like the study medication but has no…
Subjects with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) will be enrolled in the study and Intravenous Immunoglobulin (IVIg) will be given to eligible subjects on the study. CIDP is a chronic inflammatory condition that causes the immune system to attack the nerves. IVIg is one common treatment for CIDP. IVIG is a blood product that is…