Seven Spinal Muscular Atrophy Clinical Trials Currently Active in the United States

It is essential for clinicians to remain informed about emerging clinical trial opportunities and resulting data that may inform therapeutic strategies, affect clinical guidelines, expand treatment options, and ultimately improve patient quality of life.

Today, we are showcasing a selection of the latest clinical trials related to spinal muscular atrophy that are currently active in the United States. All trials are either Phase 3 or 4, and with the exception of the final two, all are currently recruiting in the United States. The following trials are arranged by start date.

Quick View Table of Spinal Muscular Atrophy Clinical Trials

Featured Spinal Muscular Atrophy Clinical Trials

A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (STELLAR-2)

• Sponsor: Biogen
• Goal: In this study, researchers will learn more about the safety and effects of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have already been treated with onasemnogene abeparvovec (OA) after being diagnosed with SMA.
• Interventions: Salanersen, sham procedure
• Primary Outcome Measure: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
• Time Frame: Up to Day 365
• Study Details
• Source

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam (SOLAR)

• Sponsor: Biogen
• Goal: 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
• Intervention: Salanersen
• Primary Outcome Measure: Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Total Score in Treatment-Naïve Cohort
• Time Frame: At month 12
• Study Details
• Source

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA) (STELLAR-1)

• Sponsor: Biogen
• Goal: 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.
• Intervention: Salanersen
• Primary Outcome Measures: Percentage of Participants with 2 Survival Motor Neuron 2 (SMN2) Copies Sitting Without Support (for at Least 10 Seconds); Percentage of Participants with 3 SMN2 Copies Walking Alone (for at Least 5 Steps); Percentage of Participants Attaining and Maintaining World Health Organization (WHO) Motor Milestones.
• Time Frames: At month 12; at month 18; up to day 1825.
• Study Details
• Source

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy (HINALEA 2)

• Sponsor: Hoffman-La Roche
• Goal: 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.
• Intervention: Risdiplam
• Primary Outcome Measure: Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment.
• Time Frame: Baseline, week 72
• Study Details
• Source

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy (HINALEA 1)

• Sponsor: Hoffmann-La Roche
• Goal: 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.
• Intervention: Risdiplam
• Primary Outcome Measure: Change from Baseline in the Raw Score of Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) Gross Motor Score at 72 Weeks of Risdiplam Treatment
• Time Frames: Baseline, week 72
• Study Details
• Source

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab (ONYX)

• Sponsor: Scholar Rock
• Goal: The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
• Intervention: Apitegromab
• Primary Outcome Measure: Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA
• Time Frame: Up to 6 years
• Study Details
• Source

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials (SPECTRUM)

• Sponsor: Novartis Pharmaceuticals
• Goal: 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.
• Intervention: Onasemnogene abeparvovec
• Primary Outcome Measures: Number of participants with treatment-emergent serious adverse events (SAEs); Number of participants with treatment emergent Adverse Events of Special Interest (AESI).
• Time Frames: Up to year 5; Up to year 5.
• Study Details
• Source

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