Spinal Muscular Atrophy (SMA)
Clinical Study of Spinal Muscular Atrophy (PNCR/iSMAC study)
Protocol ID: 31140
NCT00443066
PI: Dr. John W. Day
Study coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsors: Spinal Muscular Atrophy Foundation, CureSMA
Purpose: To study the natural history and treatment effects in SMA
Status: Recruiting in clinic
SRK-015-002: Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy (TOPAZ)
Protocol ID: 49533
NCT 03921528
Sponsor: Scholar Rock
PI: Dr. Carolina Tesi Rocha
Coordinator: Tia Lum, NeuromuscularResearch@stanford.edu, 650-725-4341
Purpose: Study the safety and efficacy of the study medication in patients with late onset Spinal Muscular Atrophy
Status: Active, not recruiting
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (SAPPHIRE)
Protocol ID: 64058
NCT05156320
PI: Dr. Carolina Tesi-Rocha
Study coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock
Purpose: Assess the safety and efficacy of apitegromab in children and adults with later-onset SMA currently receiving nusinersen or risdiplam treatment
Status: Recruiting
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX (ONYX)
Protocol ID: 68228
NCT05626855
PI: Dr. Carolina Tesi-Rocha
Study coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock
Purpose: Assess the long term effects of apitegromab in children and adults with late-onset SMA
Status: Recruiting soon by invitation only
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy (DEVOTE)
Protocol ID: 53421
NCT04089566
PI: Dr. John W. Day
Study coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen Pharmaceuticals
Purpose: Assessing the efficacy of higher doses of nusinersen in SMA
Status: Recruiting cohort B (<7 months old)
A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec (RESPOND)
Protocol ID: 58724
NCT04488133
PI: Dr. John W. Day
Study coordinator: Susan Thomas, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen Pharmaceuticals
Purpose: Assess the safety and efficacy of Spinraza treatment after Zolgensma treatment
Status: Recruiting
A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen (ONWARD)
Protocol ID: 59527
NCT04729907
PI: Dr. John W. Day
Study coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen Pharmaceuticals
Purpose: Test the safety and efficacy of higher doses of nusinersen
Status: Recruiting by invitation only
A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam (ASCEND)
Protocol ID: 62798
NCT05067790
PI: Dr. John W. Day
Study coordinator: Emily Lien, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen Pharmaceuticals
Purpose: Assessing the safety and efficacy of a higher dose of nusinersen (Spinraza) in people with SMA who have previously taken risdiplam (Evrysdi)
Status: Recruiting
Nusinersen in Adults with Spinal Muscular Atrophy: An Open Label Study to Assess Efficacy and Exploratory Endpoints (ASE study)
Protocol ID: 55518
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Christina Frater, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Assess the neuromuscular effects of SMA
Status: Recruiting in clinic
CureSMA registry
Protocol ID: 49895
NCT: N/A
PI: Dr. Jacinda Sampson
Study coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: CureSMA
Purpose: Clinical data registry with CureSMA
Status: Recruiting in clinic
Wearable Technology to Assess Gait Function in SMA and DMD
Protocol ID: 55106
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Sally Dunaway Young, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Assessing Wearable Technology to Assess Gait Function in SMA and DMD
Status: Recruiting
Development and Validation for the Adult Test of Neuromuscular Disorders (ATEND), a Functional Motor Outcome Measure
Protocol ID: 31140
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Dr. Tina Duong
Sponsor: Investigator Initiated
Purpose: Development and evaluation of psychometric properties of the ATEND for weaker individuals with neuromuscular disorders
Status: Recruiting in clinic
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Protocol ID: 52085
NCT04042025
PI: Dr. John W. Day
Study coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Novartis
Purpose: Assessing the long term effects of AVXS-101
Status: Active, not recruiting