Popular Trials
Behavioural Intervention
Exergaming for Spinal Muscular Atrophy
This trial will test if playing video games at home for 8 weeks can help children and youth with spinal muscular atrophy feel more satisfied with their daily activities compared to their usual care.
SMN2 Splicing Modifier
Long-Term Risdiplam for Spinal Muscular Atrophy
This trial will study the long-term safety and effectiveness of the drug risdiplam in patients with spinal muscular atrophy. The trial will last for up to 5 years, and patients will be followed even if they discontinue the drug.
Monoclonal Antibodies
RO7204239 + Risdiplam for Spinal Muscular Atrophy
This trial tests the safety and effectiveness of combining two treatments, RO7204239 and risdiplam, for patients with spinal muscular atrophy (SMA). Risdiplam helps the body produce a protein needed for muscle movement, while RO7204239 allows muscles to grow bigger and stronger. The study includes SMA patients to see if this combination improves their muscle function and overall health.
Popular Filters
Trials for SMA Patients
Monoclonal Antibodies
Long-Term Apitegromab for Spinal Muscular Atrophy
This trial is testing a medication called Apitegromab to see if it is safe and effective for people with certain types of spinal muscular atrophy (SMA). It focuses on patients who have already participated in previous research. The goal is to see if the medication can help improve their muscle function over time.
Trials for SMN1 Positive Patients
SMN2 Splicing Modifier
Risdiplam for Presymptomatic Spinal Muscular Atrophy
This trial is testing a medicine called risdiplam, which is taken by mouth. It aims to help infants who have a genetic diagnosis of spinal muscular atrophy (SMA) but are not yet showing symptoms. The medicine works by helping the body make more of a protein that muscles need to stay healthy. The goal is to see if early treatment can prevent or lessen the severity of SMA.
Small Molecule
Risdiplam for Spinal Muscular Atrophy
This trial is for SMA patients who have been treated with nusinersen and are now crossover to 36 months of risdiplam monotherapy. The trial will track the most informative outcomes from the nusinersen trial, while adding the Box and Block Test as an additional measure of upper limb endurance and function.
Spinal Cord Stimulation
Spinal Cord Stimulation for Spinal Muscular Atrophy
This trial tests if spinal cord stimulation (SCS) can improve leg movement in people with Type 3 or 4 Spinal Muscular Atrophy (SMA) who can stand but have motor deficits. SCS uses electrical pulses to help the spinal cord communicate better with the brain, potentially improving muscle control and strength. Spinal cord stimulation (SCS) has been shown to improve motor performance in various conditions, including certain types of muscle weakness.
Trials for SMN2 Positive Patients
Antisense Oligonucleotide
Nusinersen for SMA
This trial is evaluating nusinersen as a treatment for SMA in patients who have previously received onasemnogene abeparvovec. The objectives are to assess the safety and efficacy of nusinersen in this population.
Small Molecule
Risdiplam for Spinal Muscular Atrophy
This trial is for SMA patients who have been treated with nusinersen and are now crossover to 36 months of risdiplam monotherapy. The trial will track the most informative outcomes from the nusinersen trial, while adding the Box and Block Test as an additional measure of upper limb endurance and function.
Apitegromab for Spinal Muscular Atrophy
This trial is testing a new drug, apitegromab, as an adjunctive therapy to nusinersen or risdiplam, for patients with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA). The trial will confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.
Phase 3 Trials
Monoclonal Antibodies
Long-Term Apitegromab for Spinal Muscular Atrophy
This trial is testing a medication called Apitegromab to see if it is safe and effective for people with certain types of spinal muscular atrophy (SMA). It focuses on patients who have already participated in previous research. The goal is to see if the medication can help improve their muscle function over time.
Gene Therapy
OAV101 for Spinal Muscular Atrophy
This trial is testing a new treatment for people with a severe form of spinal muscular atrophy. The treatment will be given directly into the spinal fluid and will be evaluated for safety, effectiveness, and side effects over 15 months.
Antisense Oligonucleotide
Nusinersen for Spinal Muscular Atrophy
This trial is for participants with Spinal Muscular Atrophy who have previously participated in another study. They will be given nusinersen, which is a drug meant to help with the disease. The study is to see if the drug is safe and effective long-term.
Taldefgrobep Alfa for Spinal Muscular Atrophy
This trial will test if taldefgrobep alfa can help people with SMA who are already on other treatments. The drug works by blocking a protein that stops muscle growth, potentially making muscles stronger and improving movement. Taldefgrobep alfa has shown promise in increasing muscle mass and function in SMA patients.
Apitegromab for Spinal Muscular Atrophy
This trial is testing a new drug, apitegromab, as an adjunctive therapy to nusinersen or risdiplam, for patients with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA). The trial will confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.
Trials With No Placebo
Monoclonal Antibodies
Long-Term Apitegromab for Spinal Muscular Atrophy
This trial is testing a medication called Apitegromab to see if it is safe and effective for people with certain types of spinal muscular atrophy (SMA). It focuses on patients who have already participated in previous research. The goal is to see if the medication can help improve their muscle function over time.
Antisense Oligonucleotide
Nusinersen for Spinal Muscular Atrophy
This trial is for participants with Spinal Muscular Atrophy who have previously participated in another study. They will be given nusinersen, which is a drug meant to help with the disease. The study is to see if the drug is safe and effective long-term.
Frequently Asked Questions
Introduction to spinal muscular atrophy
What are the top hospitals conducting spinal muscular atrophy research?
When it comes to advancing the treatment options for spinal muscular atrophy (SMA), several hospitals are leading the way in clinical trials. In Aurora, Colorado, Children's Hospital Colorado is actively involved in nine ongoing SMA trials and has conducted a total of 17 trials dedicated to this rare genetic condition. Since their first recorded trial in 2012, they have been committed to finding innovative solutions for patients with SMA. Similarly, Ann & Robert H. Lurie Children's Hospital of Chicago has eight ongoing trials and a history of nine completed studies since initiating their first SMA trial in 2012.
Boston Children's Hospital, located in the heart of Boston's medical hub, is also making strides with seven active SMA trials and an impressive track record of conducting 17 previous studies since their inception into researching this condition back in 2012. Meanwhile, on the East Coast at Children's Hospital of The King's Daughters in Norfolk, Virginia researchers are currently engaged with six active SMA clinical trials while having contributed to eight earlier investigations following its pioneering efforts only three years ago starting from 2018.
Heading westward brings us to Stanford University Medical Center situated in Palo Alto where five active spinal muscular atrophy trials are underway alongside six earlier-held experiments that began as recently as four years ago duringthe yearperiodof2016.Organizations such as these play a crucial role by bringing together talented medical professionals who work tirelessly towards improving the lives of individuals affected by spinal muscular atrophy through cutting-edge research.
These hospitals exemplify dedication and progress when it comes to understanding and treating spinal muscular atrophy. Through rigorous clinical testing within diverse populations across different locations nationwide; discoveries made here can pave new avenues for breakthrough treatments ensuring brighter future not just for those impacted by SMA but potentially other related neurological conditions too
Which are the best cities for spinal muscular atrophy clinical trials?
When it comes to spinal muscular atrophy clinical trials, several cities have emerged as top destinations for research and development. New york leads the pack with 18 active trials focused on studying treatments like Risdiplam, Nusinersen, and Treatment Period. Boston closely follows with 15 ongoing studies examining interventions such as Nusinersen and Risdiplam. Chicago and Palo Alto each offer promising options with 12 and 11 active trials respectively, exploring treatments like Apitegromab, Nusinersen, Risdiplam, and more. Additionally, Aurora in Colorado also showcases its commitment to SMA research through its participation in 11 active trials investigating approaches including Nusinersen treatment. These cities provide individuals affected by spinal muscular atrophy access to cutting-edge clinical trials that hold hope for improved outcomes and quality of life.
Which are the top treatments for spinal muscular atrophy being explored in clinical trials?
Spinal muscular atrophy (SMA) clinical trials are exploring several promising treatments to combat this debilitating condition. Leading the pack is risdiplam, currently under investigation in seven active trials for SMA. Since its introduction in 2016, it has accumulated a total of 11 clinical trials dedicated to treating this disease. Another frontrunner is nusinersen, which is being studied in five ongoing SMA trials and has been listed since 2012 with a total of 11 clinical trials so far. Lastly, apitegromab shows potential with one active trial and two all-time SMA studies since its recent listing in 2022. These developments hold promise for patients affected by SMA and offer hope for improved treatment options moving forward.
What are the most recent clinical trials for spinal muscular atrophy?
Exciting developments are unfolding in the realm of spinal muscular atrophy (SMA), as recent clinical trials offer potential breakthroughs for patients. Notably, cohort 1 of a Phase 2 trial has become available, shedding light on new treatment avenues. Additionally, a Phase 3 trial focusing on the treatment period showcases promising advancements in SMA management. OAV-101 and risdiplam, both in Phase 3 trials, provide further hope for improved outcomes and quality of life for individuals with SMA. Furthermore, taldefgrobep alfa's Phase 3 trial results have generated significant interest within the medical community. These recent studies hold promise to transform SMA care and bring renewed optimism to those affected by this condition.
What spinal muscular atrophy clinical trials were recently completed?
Recently, two clinical trials have concluded, offering new insights and potential treatments for spinal muscular atrophy (SMA). In September 2021, Novartis Pharmaceuticals completed a trial evaluating the efficacy of OAV101. This milestone highlights ongoing efforts to combat SMA. Additionally, in February 2021, Hoffmann-La Roche wrapped up its risdiplam trial. These advancements hold promise for patients affected by this debilitating condition and exemplify the dedication of researchers in their quest to improve SMA treatment options.