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Why We Started Power

We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

Bask
Bask GillCEO at Power
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    Long-term Follow-up with Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy · Info for Participants · Phase Phase 3 Clinical Trial 2025 | Power | Power

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      Why We Started Power

      We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

      Bask
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      31 Spinal Muscular Atrophy Trials Near You

      Power is an online platform that helps thousands of Spinal Muscular Atrophy patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

      Learn More About Power
      No Placebo
      Highly Paid
      Stay on Current Meds
      Pivotal Trials (Near Approval)
      Breakthrough Medication

      High-Dose Nusinersen for Spinal Muscular Atrophy

      Columbus, Ohio
      In this study, researchers will learn more about the use of a higher dose of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study will focus on teenagers and adults who are unable to walk on their own and who have previously taken another drug for SMA called risdiplam. The main goal of this study is to learn about the effect of high dose (HD) nusinersen on muscle and movement ability (motor function) in SMA. The main question that researchers want to answer is: \- How do the scores of a movement test called the Revised Upper Limb Module change from the start of treatment? The Revised Upper Limb Module is a test used to measure a participant's ability to do specific tasks that involve their shoulders, arms, wrist, elbows, and hands. It measures the changes in their abilities over time. Researchers will also learn more about the safety of HD nusinersen. They will check participants for adverse events and changes in vital signs, heart tests, and laboratory tests including blood and urine tests. The study will be done as follows: * Participants will be screened to check if they can join the study. * After screening, participants will enter the Core Treatment period. * At the start of the Core Treatment period, they will receive 2 "loading" doses of nusinersen. These are 50 mg doses of nusinersen given 2 weeks apart. * Afterwards, they will continue to receive "maintenance" doses of nusinersen once every 4 months. These doses will be 28 mg. * The Core Treatment period will last about 2 years, with a follow-up visit 4 months after the last dose. * Participants who complete the Core Treatment period will have the option to continue receiving 28 mg of nusinersen in the Long-Term Extension (LTE) period for about 2 years. There will also be a follow-up visit 4 months after the last dose. * Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back. * In total, participants will have up to 18 study visits. They will also be called by researchers after each dose of nusinersen. * Participants will stay in the study for about 4.5 years if they complete both the Core Treatment and LTE periods.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:15 - 50

      Key Eligibility Criteria

      Disqualifiers:Major Illness, Active Infection, Others
      Must Be Taking:Risdiplam

      45 Participants Needed

      Long-term Follow-up with Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

      Columbus, Ohio
      This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Parent Unwilling To Participate

      85 Participants Needed

      NMD670 for Spinal Muscular Atrophy

      Columbus, Ohio
      The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2

      Key Eligibility Criteria

      Disqualifiers:Scoliosis, Neuromuscular Diseases, Others

      54 Participants Needed

      Taldefgrobep Alfa for Spinal Muscular Atrophy

      Columbus, Ohio
      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.
      Stay on current meds
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:4 - 21

      Key Eligibility Criteria

      Disqualifiers:Respiratory Insufficiency, Spinal Fusion, Others
      Must Be Taking:Nusinersen, Risdiplam, Onasemnogene

      269 Participants Needed

      Gene Therapy for Spinal Muscular Atrophy

      Columbus, Ohio
      This trial uses a harmless virus to deliver a healthy gene directly into the spinal fluid of patients with diseases caused by IGHMBP2 gene defects. The virus helps correct the genetic issues by bringing the healthy gene to the affected cells. This approach has been shown to improve conditions in similar diseases in animals.
      No Placebo Group

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 1, 2
      Age:2 - 14

      Key Eligibility Criteria

      Disqualifiers:HIV, Hepatitis B/C, Autoimmune, Others
      Must Not Be Taking:Immune Modulatory Therapy

      10 Participants Needed

      Long-Term Apitegromab for Spinal Muscular Atrophy

      Columbus, Ohio
      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.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:2+

      Key Eligibility Criteria

      Disqualifiers:Unstable Nutrition, Ventilator Use, Others
      Must Not Be Taking:SMN-targeted Therapies, Apitegromab

      238 Participants Needed

      Long-Term Risdiplam for Spinal Muscular Atrophy

      Cincinnati, Ohio
      A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for the duration of the study or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments. An optional sub study will assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. Approximately 39 participants from the main study are planned to be enrolled in the sub study.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 4
      Age:2+

      Key Eligibility Criteria

      Disqualifiers:Hypersensitivity To Risdiplam, Schizophrenia, Bipolar, Others
      Must Be Taking:Risdiplam

      402 Participants Needed

      Risdiplam for Spinal Muscular Atrophy

      Ann Arbor, Michigan
      This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:< 19

      Key Eligibility Criteria

      Disqualifiers:SMA Type 0, Blood Pressure Abnormalities, Others
      Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers, MATE Substrates

      10 Participants Needed

      Spinal Cord Stimulation for Spinal Muscular Atrophy

      Pittsburgh, Pennsylvania
      Spinal cord stimulation (SCS) has shown remarkable efficacy in restoring motor function in people with spinal cord injury by recruiting afferent input to enhance the responsiveness of spared neural circuits to residual cortical inputs. This pilot will test if SCS can show evidence to improve motor deficits in people with Type 2, 3, or 4 spinal muscular atrophy (SMA). The investigators will enroll up to six subjects with Type 2, 3, or 4 SMA aged 16 or older that show quantifiable motor deficits of the upper body. The investigators will then implant the subjects with percutaneous, linear spinal leads near the cervical spinal cord for a period of up to 29 days. Although these leads are not optimized for motor function but rather for their clinically approved indication of treating pain, the investigators believe they provide a safe technology enabling our team to perform scientific measurement necessary to evaluate potential for effects of SCS in motor paralysis with SMA. After the end of the study, the leads will be explanted.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:16 - 65

      Key Eligibility Criteria

      Disqualifiers:Spinal Deformation, Joint Contractures, Others
      Must Be Taking:SMN Inducing Therapies

      3 Participants Needed

      Spinal Cord Stimulation for Spinal Muscular Atrophy

      Pittsburgh, Pennsylvania
      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.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:16 - 64

      Key Eligibility Criteria

      Disqualifiers:Spinal Deformation, Joint Contractures, Others
      Must Not Be Taking:Anticoagulants, Anti-spasticity, Anti-seizure

      3 Participants Needed

      Implantable Brain-Computer Interface for Paralysis

      Pittsburgh, Pennsylvania
      The Synchron motor neuroprosthesis (MNP) is intended to be used in subjects with severe motor impairment, unresponsive to medical or rehabilitative therapy and a persistent functioning motor cortex. The purpose of this research is to evaluate safety and feasibility. The MNP is a type of implantable brain computer interface which bypasses dysfunctional motor neurons. The device is designed to restore the transmission of neural signal from the cerebral cortex utilized for neuromuscular control of digital devices, resulting in a successful execution of non-mechanical digital commands.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:21 - 75

      Key Eligibility Criteria

      Disqualifiers:Immunosuppression, Anesthesia Unsuitability, Allergies, Others

      6 Participants Needed

      Nusinersen for Spinal Muscular Atrophy

      London, Ontario
      In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study is an extension study and will enroll only those participants who have completed treatment in the parent study, 232SM203. The main goal of the study is to learn about the long-term safety of nusinersen. The main questions researchers want to answer are: * How many participants have adverse events and serious adverse events during the study? * How do the results of electrocardiograms (ECGs), vital signs, and laboratory tests including blood and urine tests change after treatment? * How many participants have a low platelet count after treatment? * How many participants had a change in the time it took for their heart to recharge between beats after treatment? * How does each participant's height and other measures of growth change after treatment? * How much do the results of neurological exams that check movement, reflexes, and brain function change after treatment? Researchers will also learn about the effect of nusinersen on mobility using various tests. They will study body movements, reflexes, balance, and coordination. They will also record if participants need help with breathing. The 232SM302 study will be done as follows: * Participants will be screened to check if they can join the study. * Participants will receive their 1st dose of nusinersen in this study about 4 months after their final dose in the parent study. * Each participant will receive nusinersen once every 4 months during the treatment period. * Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back. * The treatment period will last for up to 64 months (1921 days). * There will be a follow-up safety period that lasts from 4 to 8 weeks. * In total, participants will have up to 19 study visits. Participants will stay in the study for close to 6 years.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:2+

      Key Eligibility Criteria

      Disqualifiers:Other Investigational Therapies, Others
      Must Be Taking:Nusinersen

      115 Participants Needed

      Risdiplam for Presymptomatic Spinal Muscular Atrophy

      London, Ontario
      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.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2
      Age:1 - 6

      Key Eligibility Criteria

      Disqualifiers:Investigational Drug Use, Significant Syndromes, Others
      Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers

      26 Participants Needed

      Risdiplam for Spinal Muscular Atrophy

      Grand Rapids, Michigan
      This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 4
      Age:3 - 24

      Key Eligibility Criteria

      Disqualifiers:Invasive Ventilation, Feeding Tube, Others
      Must Not Be Taking:Antisense Oligonucleotides, Splicing Modifiers

      28 Participants Needed

      Risdiplam for Spinal Muscular Atrophy

      Grand Rapids, Michigan
      This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 4
      Age:3 - 24

      Key Eligibility Criteria

      Disqualifiers:Invasive Ventilation, Tracheostomy, Feeding Tube, Others
      Must Not Be Taking:Antisense Oligonucleotides, Splicing Modifiers

      28 Participants Needed

      Nusinersen for Spinal Muscular Atrophy

      Grand Rapids, Michigan
      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 objective 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 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. * 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.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1
      Age:3+

      Key Eligibility Criteria

      Disqualifiers:Other Trials, Nusinersen Naive, Pregnant, Others
      Must Be Taking:Nusinersen

      58 Participants Needed

      Powered Wheelchair Standing Devices for Developmental Delay

      Grand Rapids, Michigan
      For children who use a power wheelchair, a powered wheelchair standing device (PWSD) may be considered for daily use. A PWSD allows a child to electronically move between sitting and standing and can be driven in either position. Existing published PWSD research in pediatrics is limited to boys with Duchenne muscular dystrophy (DMD).(1, 2) While these studies provide some insights into PWSD use in boys with DMD, they do not reflect PWSD use in children with other conditions. The purpose of this exploratory study is to determine the feasibility of a research protocol exploring use of a PWSD in children who have neurodevelopmental conditions other than DMD.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:5 - 17

      Key Eligibility Criteria

      Disqualifiers:Duchenne Muscular Dystrophy, Others

      4 Participants Needed

      OAV101 for Spinal Muscular Atrophy

      Chicago, Illinois
      To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment naive patients with Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to \< 18 years of age over a 15 month trial duration.
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3
      Age:2 - 17

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      127 Participants Needed

      Nusinersen for SMA

      Chicago, Illinois
      In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study will focus on children under the age of 3 who were previously treated with the gene therapy onasemnogene abeparvovec but are still facing health challenges related to their disease. The main goal of the study is to learn about the effect nusinersen has on muscle and movement ability (motor function). The main question researchers want to answer is: \- What score do participants have on the HINE Section 2 Motor Milestones test after treatment? The Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones is an assessment that tests movements in different positions. This includes grasping, kicking, head control, rolling, sitting, crawling, standing, and walking. Researchers will use a group of tests to study body movements, reflexes, balance, and coordination. They will also record if participants need help with breathing. Researchers will also learn more about the safety of nusinersen. They will check participants for adverse events and changes in vital signs, heart tests, and laboratory tests including blood and urine tests. The study will be done as follows: * Participants will be screened to check if they can join the study. * Each participant will receive 4 initial doses of 12 mg of nusinersen on Days 1, 15, 29, and 64 of the Treatment Period. Then, they will receive 12 mg doses once every 4 months. * The total number of doses of nusinersen will be 9. * Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back. * The treatment period will last for up to 95 weeks (close to 2 years). * There will be a follow-up safety period that lasts about 4 months. * In total, participants will have up to 14 study visits. Participants will stay in the study for up to 115 weeks.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 4
      Age:2 - 36

      Key Eligibility Criteria

      Disqualifiers:Prior Nusinersen, Severe AEs, Others
      Must Not Be Taking:Investigational Drugs, SMN2 Modifiers

      46 Participants Needed

      Nusinersen for Spinal Muscular Atrophy

      Chicago, Illinois
      The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Age:6 - 6

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      25 Participants Needed

      Why Other Patients Applied

      "My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."

      HZ
      Arthritis PatientAge: 78

      "I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."

      AG
      Paralysis PatientAge: 50

      "I've tried several different SSRIs over the past 23 years with no luck. Some of these new treatments seem interesting... haven't tried anything like them before. I really hope that one could work."

      ZS
      Depression PatientAge: 51

      "I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."

      FF
      ADHD PatientAge: 31

      "As a healthy volunteer, I like to participate in as many trials as I'm able to. It's a good way to help research and earn money."

      IZ
      Healthy Volunteer PatientAge: 38
      Match to a Spinal Muscular Atrophy Trial

      Apitegromab for Spinal Muscular Atrophy

      Winston-Salem, North Carolina
      This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score \<55.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2
      Age:< 2

      Key Eligibility Criteria

      Disqualifiers:Severe Scoliosis, Severe Contractures, Others
      Must Be Taking:SMN1-targeted, SMN2-targeted

      52 Participants Needed

      RO7204239 + Risdiplam for Spinal Muscular Atrophy

      Toronto, Ontario
      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.
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2, 3
      Age:2 - 25

      Key Eligibility Criteria

      Disqualifiers:Recent Surgery, Major Illness, Hereditary Fructose Intolerance, Others
      Must Be Taking:Risdiplam

      259 Participants Needed

      Exergaming for Spinal Muscular Atrophy

      Toronto, Ontario
      This is a multisite randomized controlled registry-based trial to evaluate the efficacy of an 8-week home-based exergaming intervention as compared to usual care on occupational satisfaction of children and youth (8-18 years old age) with spinal muscular atrophy (SMA).
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:8 - 18

      Key Eligibility Criteria

      Disqualifiers:Severe Scoliosis, Contractures, Others
      Must Be Taking:Disease-modifying Therapy

      22 Participants Needed

      Risdiplam for Spinal Muscular Atrophy

      Strasburg, Pennsylvania
      Risdiplam Exchange (RISE) is a study of spinal muscular atrophy (SMA) patients who crossover to 36 months of open-label risdiplam monotherapy following a comparable period of nusinersen treatment. The schedule of assessments (SOAs) carry over seamlessly for the cohort from studies done while treated with nusinersen and continue to track the most informative outcomes from that trial (e.g. nine hole peg test and grip strength), while adding the Box and Block Test (BBT) as an additional measure of upper limb endurance and function.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 4
      Age:2 - 35

      Key Eligibility Criteria

      Disqualifiers:SMN Gene Therapy, Neuromuscular Disorder, Others
      Must Be Taking:Nusinersen

      10 Participants Needed

      Neurosleeve for Arm Weakness/Paralysis

      Wilmington, Delaware
      This trial is testing the NuroSleeve, a wearable device that helps people with weak or paralyzed arms move them using electrical signals. It is aimed at those who haven't fully recovered with standard therapies. The device detects small muscle movements and uses them to activate the brace and stimulate muscles, helping with arm movement. Electrical stimulation has been explored in various contexts, including improving arm and hand function in individuals with spinal cord injuries and post-stroke patients.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Unphased
      Age:4+

      Key Eligibility Criteria

      Disqualifiers:Visual Impairment, Orthopedic Conditions, Others
      Must Not Be Taking:Bupropion

      20 Participants Needed

      OAV101 for Spinal Muscular Atrophy

      Norfolk, Virginia
      This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3
      Age:0 - 100

      175 Participants Needed

      Motor Skill and Strength Training for Spinal Muscular Atrophy

      New York, New York
      This single-arm pilot study will assess the feasibility and preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength training (FST) in children with SMA who are receiving disease-modifying therapies. Participants will attend one 6-hour HABIT-ILE + FST session each weekend (Saturday or Sunday) for 15 weeks, for a total of 90 hours of training. Feasibility will be evaluated through questionnaires, and effectiveness through standardized motor function assessments.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Unphased
      Age:5 - 17

      Key Eligibility Criteria

      Disqualifiers:Orthopedic Surgery, New Treatments, Others
      Must Be Taking:Disease-modifying Therapies

      5 Participants Needed

      Risdiplam for Spinal Muscular Atrophy

      New York, New York
      This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Age:6 - 60

      Key Eligibility Criteria

      Disqualifiers:Not Listed

      174 Participants Needed

      BIIB115 for Spinal Muscular Atrophy

      Ontario
      In this study, researchers will learn about a study drug called BIIB115 in healthy adult male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA. The main objective of the study is to learn about the safety of BIIB115 and how participants respond to different doses of BIIB115. The main question researchers want to answer is: • How many participants have adverse events and serious adverse events during the study? Adverse events are unwanted health problems that may or may not be caused by the study drug. Researchers will also learn about how the body processes BIIB115. They will do this by measuring the levels of BIIB115 in both the blood and the cerebrospinal fluid, also known as the CSF. This is the fluid around the brain and spinal cord. The study will be split into 2 parts - Part A and Part B. During Part A: * After screening, healthy volunteers will be randomly placed into 1 of 4 groups to receive either BIIB115 or a placebo. A placebo looks like the study drug but contains no real medicine. * Participants will receive a single dose of either BIIB115 or the placebo as an injection directly into the spinal canal on Day 1. * Neither the researchers nor the participants will know if the participants will receive BIIB115 or the placebo. * The Part A treatment and follow-up period will last for 13 months. * Participants will have up to 6 clinic visits and 4 phone calls. During Part B: * After screening, children with SMA will be placed into 1 of 2 groups to receive BIIB115. * The doses of each group will be decided based on the results of Part A. * Both researchers and participants will know they are receiving BIIB115. * Participants will first receive 2 total doses of BIIB115 given at 2 different times. * The Part B treatment and follow-up period will last for 24 months. * Participants will have up to 14 clinic visits and 6 phone calls. Part B Long-Term Extension: * After completing the 25 months in Part B, participants may move onto the long-term extension (LTE). * They will receive 5 more doses of BIIB115 at different times. * The Part B LTE treatment and follow-up will last for 60 months. * Participants will have up to 12 more clinic visits and 19 phone calls. In both Part A and Part B, participants will stay in the clinic for 24 hours after each dose so that researchers can check on their health. This 24-hour stay will not be required for the Part B LTE period.

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1
      Age:6 - 55

      Key Eligibility Criteria

      Disqualifiers:Liver Injury, Thrombotic Microangiopathy, Others
      Must Be Taking:Onasemnogene Abeparvovec

      62 Participants Needed

      Salanersen for Spinal Muscular Atrophy

      Flower Mound, Texas
      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 babies who have been diagnosed with SMA through genetic testing but have not yet started showing signs or symptoms. Most people with SMA have changes in a gene called survival motor neuron 1, also known as SMN1. These changes lower the amount of SMN protein in their bodies. Without enough of this protein, motor neurons and muscles cannot work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen works by helping the SMN2 gene to make more SMN protein. In this study, participants will have either 2 SMN2 copies or 3 SMN2 copies. The higher the copy number, the less severe the participant's SMA is. The main goal of this study is to see if starting salanersen before signs or symptoms appear can prevent signs or symptoms of SMA or make them less severe. Researchers will use different tests to learn if motor symptoms are changing, including the World Health Organization (WHO) motor milestones. The main questions researchers want to answer in this study are: * How many participants with 2 copies of the SMN2 gene can sit without support at 12 months? * How many participants with 3 copies of the SMN2 gene can walk alone at 18 months? Researchers will also learn more about: * The effects on participants' motor symptoms and how many new movement milestones participants achieve. * How many participants stay free of SMA symptoms * How much salanersen gets into the fluid surrounding the brain and spinal cord. * How much salanersen gets into the blood. * 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. This study will be done as follows: * First, participants will be screened to check if they can join the study. The screening period will be up to 28 days. * This is an "open label" study. This is a study in which the participants, study doctor, and site staff know which study drug participants are receiving. In this study, all participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord. * There will be 2 parts in this study. During Part 1, participants will receive 2 doses of salanersen, about 12 months apart from each other. Part 1 will last up to 25 months. * During Part 2, participants will continue to receive salanersen. They will receive up to 3 doses, 12 months apart from each other. Part 2 will last up to 36 months. * During Part 1, participants will have up to 11 clinic visits and up to 3 phone calls. During Part 2, participants will have up to 7 clinic visits and up to 12 phone calls.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Not Yet Recruiting
      Trial Phase:Phase 3
      Age:0 - 42

      Key Eligibility Criteria

      Disqualifiers:SMA Symptoms, Areflexia, Hypoxemia, Others
      Must Not Be Taking:SMA Therapies, Myostatin Inhibitors

      30 Participants Needed

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      Frequently Asked Questions

      How much do Spinal Muscular Atrophy clinical trials pay?

      Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

      How do Spinal Muscular Atrophy clinical trials work?

      After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Spinal Muscular Atrophy trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Spinal Muscular Atrophy is 12 months.

      How do I participate in a study as a "healthy volunteer"?

      Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

      What does the "phase" of a clinical trial mean?

      The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

      Do I need to be insured to participate in a Spinal Muscular Atrophy medical study?

      Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

      What are the newest Spinal Muscular Atrophy clinical trials?

      Most recently, we added Salanersen for Spinal Muscular Atrophy, Motor Skill and Strength Training for Spinal Muscular Atrophy and Apitegromab for Spinal Muscular Atrophy to the Power online platform.