Ongoing Clinical Trials for Bulbospinal Muscular Atrophy
There are currently 3 ongoing clinical trials studying treatments for bulbospinal muscular atrophy, a rare genetic neuromuscular disorder that causes progressive muscle weakness and wasting, primarily affecting males. These trials are testing different medications including clenbuterol, NIDO-361, and nipocalimab, and are being conducted across several European countries. (Also known as: Spinal and Bulbar Muscular Atrophy, SBMA, Kennedy’s disease)
Clinical trial locations
- Czechia
- Denmark
- France
- Germany
- Hungary
- Italy
- Poland
- Spain
Study of Clenbuterol vs Placebo in Adult Patients with Spinal and Bulbar Muscular Atrophy (SBMA)
This study is testing clenbuterol, a medication that belongs to a class of drugs called beta-2 agonists, to see if it can help improve muscle strength and function in patients with SBMA. The trial will last for 48 weeks and will compare the effects of clenbuterol tablets with placebo tablets.
Who can participate: The study is looking for male patients between 18 and 75 years old who have a confirmed genetic diagnosis of SBMA with at least 38 CAG repeats in the AR gene. Participants must have symptoms such as muscle atrophy, limb weakness, or bulbar palsy, and must be able to walk independently, although they can use support devices like canes or walkers. Wheelchairs are not allowed.
Who cannot participate: Female patients cannot join this study, as SBMA primarily affects males. The study also excludes individuals under 18, those with other forms of muscular atrophy not related to SBMA, patients with severe heart conditions, uncontrolled high blood pressure, or severe liver or kidney dysfunction. People currently taking medications that could interact with clenbuterol, those with known allergies to the medication, or individuals participating in other clinical trials are also excluded.
What the study involves: Participants will take either clenbuterol or placebo tablets by mouth, with a maximum daily dose of 40 micrograms for those receiving the active medication. Throughout the study, various measurements will be taken including a six-minute walking test to assess mobility, breathing function tests, muscle strength evaluations, and blood tests to check creatinine levels. Quality of life will also be assessed through questionnaires. The main focus is to determine if clenbuterol can improve the distance patients can walk in six minutes compared to their starting ability.
Study of NIDO-361 for Patients with Spinal and Bulbar Muscular Atrophy (SBMA)
This clinical trial is investigating NIDO-361, a new medication taken as a daily tablet, to see if it can help restore muscle volume in patients with SBMA. The study will last up to 12 months and is designed as a double-blind trial, meaning neither participants nor researchers will know who is receiving the actual medication or placebo.
Who can participate: Male patients between 18 and 70 years old who can walk on their own are eligible for this study. Participants must have a Body Mass Index between 18 and 32 and a confirmed diagnosis of SBMA through DNA genetic testing. They must be able to complete a six-minute walk test and have a score between 25 and 45 on the SBMA Functional Rating Scale, which assesses the severity of the condition. An important requirement is showing evidence of muscle fat replacement on an MRI scan, with at least 10% but no more than 50% muscle fat in affected muscles. If using supplements or vitamins, the dosage must have been stable for at least eight weeks before screening. Sexually active participants who have not been sterilized must agree to use adequate contraception during the study and for 90 days after the last dose.
Who cannot participate: Only male patients can participate in this study. Individuals who do not have SBMA or who are part of a vulnerable population that may need special protection are excluded.
What the study involves: After initial assessments including a six-minute walk test and whole-body MRI, participants will be randomly assigned to receive either NIDO-361 or a placebo tablet once daily. Regular monitoring will include MRI scans to track changes in muscle volume, as well as various physical activity assessments such as the six-minute walk test, grip strength measurements, and timed up and go tests. The study will also monitor for any side effects or adverse events. The main goal is to observe whether NIDO-361 can increase muscle volume and to assess its safety when taken as a daily oral medication.
Study on Nipocalimab for Adults with Active Inflammatory Muscle Diseases
This study is evaluating nipocalimab, a medication given as an infusion directly into the bloodstream, for treating active idiopathic inflammatory myopathies. These are rare conditions that cause muscle inflammation and weakness. The trial will last up to 52 weeks and is expected to conclude by March 2027.
Who can participate: Both male and female participants can join this study. Participants must have active idiopathic inflammatory myopathies and meet specific diagnostic criteria based on 2017 guidelines, established at least 6 weeks before starting treatment. They must also have specific antibodies related to muscle inflammation present in their blood. If currently using low-strength topical treatments for skin lesions, the dosage and frequency must have been stable for at least 4 weeks before starting the study and must remain the same until Week 52.
Who cannot participate: The study excludes people with any other serious health condition that could interfere with the study, those who are pregnant or breastfeeding, and individuals with a history of severe allergic reactions to medications. People using certain medications that might affect study results, those with recent infections requiring treatment, or individuals with a history of drug or alcohol abuse cannot participate. The study also excludes those who have participated in another clinical trial recently, have conditions affecting the immune system, have a history of cancer except for some types of skin cancer, or have blood disorders.
What the study involves: After confirming the diagnosis through initial assessments including antibody testing, participants will receive either nipocalimab or a placebo through intravenous infusion. Throughout the study, regular monitoring will be conducted to evaluate the effectiveness and safety of the treatment, including assessments of symptom improvements and any potential side effects. The primary goal is to achieve at least minimal improvement in the condition by Week 52, with a reduction in the use of oral prednisone or its equivalent. Nipocalimab works at the molecular level by targeting and blocking specific proteins involved in the immune response, helping to reduce inflammation. It is classified as a monoclonal antibody.
Summary
The three ongoing clinical trials for bulbospinal muscular atrophy are testing different therapeutic approaches to address this rare genetic neuromuscular disorder. Two trials are specifically focused on SBMA, testing clenbuterol and NIDO-361, while a third trial is examining nipocalimab for inflammatory muscle diseases, which includes conditions that may overlap with or be related to SBMA.
The trials are concentrated primarily in Europe, with Italy hosting all three studies, making it a central location for this research. Other participating countries include Denmark, Hungary, Germany, Spain, France, Czechia, and Poland, demonstrating a collaborative European effort to find effective treatments for this condition.
Each trial takes a different approach: the clenbuterol study focuses on improving muscle strength and walking ability over 48 weeks, the NIDO-361 trial aims to restore muscle volume over 12 months using MRI imaging to track changes, and the nipocalimab study addresses inflammatory aspects of muscle disease over 52 weeks. All three trials are double-blind, placebo-controlled studies, which represents the gold standard in clinical research.
These trials offer hope for patients with this progressive condition, as they explore multiple potential treatment pathways using different mechanisms of action. The variety of approaches reflects the complexity of the disease and the ongoing efforts to find effective therapies for those affected by bulbospinal muscular atrophy.
