Biogen

In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in individuals with SMA who are between the ages of 15 and 60 years old. In most people living with SMA, changes to or a lack of a gene called survival motor neuron 1 (SMN1) - often referred to as gene mutations or variants - affect how this gene works. As a result, their bodies produce less SMN protein. Without enough of this protein, motor neurons and muscles cannot work properly. There is a similar gene called SMN2 that produces SMN protein, but it usually does not produce enough SMN protein on its own to make up for the changes in the SMN1 gene. Salanersen is a drug designed to help the SMN2 gene to make more working SMN protein. In this study, there will be 2 groups of participants: a group who has never received treatment for SMA before joining this study, and a group who has been treated with risdiplam, an approved drug for SMA . Those participants must not have received any other SMA treatments before and will need to stop their risdiplam treatment for the duration of the study. The main goal of this study is to learn more about how salanersen affects the participants' motor function. Researchers will use different tests and questionnaires to learn if motor function is changing over the study duration. The main question researchers want to answer in this study is: • For the group who has never been treated for SMA, how much do scores on the HFMSE movement test change at 12 months compared to the beginning of the study? The Hammersmith Functional Motor Scale - Expanded (HFMSE) has 33 activities that are scored which include sitting, lying down, walking, jumping, and more. Researchers will also learn more about: * The effects on participants' motor function and how well their nerves and muscles function. * The effects on participants' overall sense of change and how they perform daily activities. * How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug. * How much salanersen gets into the fluid surrounding the brain and spinal cord. * How much salanersen gets into the blood. This study will be done as follows: * First, participants will be screened to check if they can join the study. The screening period may be up to 4 weeks. * This is an "open-label" study. This is a study in which the participants, study doctor, and site staff will know that participants are receiving salanersen. * All participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord. * Participants will receive salanersen once every year for a total of 5 times throughout the study. * Including screening, participants will have 17 study visits and 9 telephone calls during this study, which will last up to 61 months in total.Show more

In this study, researchers will learn more about the use of felzartamab in participants with immunoglobulin A nephropathy (IgAN). IgAN is a kidney disease caused by the buildup of an antibody called IgA in the kidneys over time. In people with IgAN, abnormal IgA and other antibodies form clusters that build up in the small filters of the kidneys, which leads to inflammation and damage. Felzartamab is designed to target certain immune cells that produce these abnormal antibodies. This study will focus on participants who have protein in their urine (proteinuria) as a result of damaged kidneys. The main goal of the study is to learn about the effect felzartamab has on proteinuria. The main question that researchers want to answer is: • How much does the amount of protein in the urine change from the start of the study to Week 36? Researchers will learn about the effect felzartamab has on the kidneys' ability to filter blood. They will also learn more about the safety of felzartamab and how it is processed by the body. The study will be done as follows: * Participants will be screened to check if they can join the study. * Participants will be randomized to receive either felzartamab or a placebo. A placebo looks like the study drug but contains no real medicine. * Neither the researchers nor the participants will know what the participants will receive. * Participants will receive felzartamab or placebo as intravenous (IV) infusions. The treatment period will last 24 weeks. * Afterwards, participants will enter a follow-up period which will last 80 weeks. * In total, participants will have 17 study visits. Participants will stay in the study for about 2 years.Show more

In this study, researchers will learn more about the use of felzartamab in participants with primary membranous nephropathy, also known as PMN. In people with PMN, autoantibodies build up in the glomeruli of the kidney. Antibodies are proteins that help the body fight off infection. An autoantibody is a type of antibody that mistakenly targets and attacks the body's own tissues. Glomeruli are the filters of the kidney that remove waste and extra fluid from the body. In PMN, the build-up of autoantibodies in the glomeruli causes damage to the kidneys. Kidney damage can lead to too much protein and blood leaking into the urine. High levels of protein in the urine, called proteinuria, are common in people with PMN. Symptoms of PMN can include swelling in the legs and body, tiredness, and high blood pressure. If left untreated, PMN can eventually lead to kidney failure. In this study, researchers will learn more about how a study drug called felzartamab affects people with PMN. Felzartamab is a monoclonal antibody, which means it is an antibody made in a laboratory. Felzartamab can target immune cells that produce autoantibodies, helping to lower their buildup in the kidneys. The main goal of this study is to compare how felzartamab works compared to a drug called tacrolimus. Tacrolimus is another drug given to people with PMN and kidney disease. The main question that researchers want to answer is: * How many participants achieve a complete response after 104 weeks of treatment? * A complete response means that their urine protein levels decrease to a low level and their kidney function remains stable. Researchers will also learn about: * How long it takes before the participants' disease gets worse * How long the participants' urine protein levels stay low * How many participants develop antibodies against felzartamab in the blood? * How many participants achieve a complete response after 76 weeks of treatment * How many participants have medical problems during the study * How felzartamab is processed by the body * How felzartamab affects participants' tiredness and overall physical health The study will be done as follows: * Participants will be screened to check if they can join the study. This may take up to 42 days. * Participants will be randomized to receive either felzartamab as intravenous (IV) infusions or tacrolimus, taken orally as tablets. * If participants have worsening kidney function or worsening proteinuria, or if their PMN relapses, or if they show no signs of improvement in their PMN, they will have a chance to receive rescue treatment. * If a participant stops treatment early, there will be follow-up visits every 12 weeks until they reach Week 104. * In total, participants will have up to 23 study visits. Participants who do not need rescue treatment will stay in the study for up to 104 weeks. Participants who need rescue treatment will stay in the study for up to 156 weeks.Show more

This is a clinical trial examining to impact of aerobic training plus omaveloxolone in FRDA. Thirty individuals with FRDA will be recruited; 20 individuals will be on omaveloxolone treatment whereas the other ten individuals will not. Individuals will undergo baseline assessment including cardiopulmonary exercise testing (CPET), mFARS, gait speed, Timed Up and Go (TUG), Fatigue Severity Scale (FSS), Fatigue Impact Scale (FIS), and 6-minute Walk Test (6MWT). Individuals will then perform 3-months of home aerobic training. Repeat assessments will be conducted at 3- and 6-months.Show more

In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE study may be enrolled in the PIERRE-PK study. The main goal of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are: * What is the highest amount of nusinersen found in the blood after dosing? * How much nusinersen is found in the blood over the first 24 hours after dosing? The PIERRE-PK study will be done as follows: * Participants will be screened to check if they can join the study. The screening period will be up to 30 days for this study and may overlap with the PIERRE study. * Participants will join 1 of 2 groups based on their ongoing nusinersen treatment. One group will be receiving 12 mg doses of nusinersen. The other group will be receiving 28 mg doses of nusinersen. * Participants will first receive a dose of nusinersen by lumbar puncture. * The ThecaFlex DRx system will be implanted after the lumbar puncture, as part of the PIERRE study. * Participants will receive a dose of nusinersen by the ThecaFlex DRx system, as part of the PIERRE study. This dose will be a regular maintenance dose that happens about 4 months after their first dose in this study. * Researchers will take blood samples before and after each dose. The last blood sample will be taken 24 hours after the dose. * The total study duration for each participant in the PIERRE-PK study will be approximately 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.Show more

Spinal muscular atrophy (SMA) is a form of motor neuron disease, most commonly caused by a mutation in the survival motor neuron 1 gene (SMN1) which results in a wide disease spectrum affecting children and adults. It is an autosomal recessive disorder and is therefore caused by inheritance of a mutated gene from each parent. All forms of SMA have an estimated combined incidence of 1 in 6,000 to 1 in 10,000 live births, with a carrier frequency of 1/40 to 1/60. The patient registry aims to facilitate a questionnaire-based research study in order to better characterise and understand the disease in the UK and in Ireland. Entry is via self-registration over a secure internet connection (https://www.sma-registry.org.uk/). Online, patients are asked to read an information sheet about the research project and then indicate their consent to demonstrate willingness to participate. Following online consent, subjects will be entered into the registry. This is an on-going database and all participants are invited to update their information on a biannual basis.Show more

The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA). All enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRx™ System) and will be followed for 12 months after receiving the implant. The 12-month data will be used to assess the primary endpoint support a Pre-Market Approval (PMA) application.Show more

Biogen is a neurobiological company with strong focus on rare neurodegenerative and neurological diseases including spinal muscular atrophy, Friedreich's ataxia, and ALS. The company develops disease-modifying therapies for rare inherited neurological conditions.

Biogen has active programs across 31 rare diseases: Alport Syndrome, Amyotrophic Lateral Sclerosis, Angelman Syndrome, Dermatomyositis, Eosinophilic Granulomatosis with Polyangiitis, Inherited Erythromelalgia, Focal Segmental Glomerulosclerosis, Friedreich Ataxia, Gastrointestinal Stromal Tumor, Giant Cell Arteritis, Graves’ Disease, Hemophilia A, Hemophilia B, Idiopathic Pulmonary Fibrosis, IgA Nephropathy, Immune Thrombocytopenia, KCNT1-Related Epilepsy, Membranous Nephropathy, Mesothelioma, Multiple Sclerosis, Myotonic Dystrophy, Polycystic Kidney Disease, Progressive Supranuclear Palsy, Pulmonary Arterial Hypertension, Retinitis Pigmentosa, Sarcoidosis, Sickle Cell Disease, Sjögren's Syndrome, Spinal Muscular Atrophy, Spinocerebellar Ataxia, Systemic Sclerosis. These span several therapeutic areas. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Biogen is a diversified pharmaceutical company headquartered in Cambridge, United States, founded in 1978. While it operates across multiple therapeutic areas, it maintains a significant rare disease portfolio with 31 active rare disease programs including Alport Syndrome, Amyotrophic Lateral Sclerosis, Angelman Syndrome. Many of its rare disease treatments hold orphan drug designations from the FDA.

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Biogen, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

Biogen was founded in 1978 and is headquartered in Cambridge, United States. The company has been active for 48 years. It trades publicly under the ticker symbol BIIB.