A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec

study id #: NCT04488133

condition: Muscular Atrophy, Spinal

status: Recruiting

The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atrophy (SMA) who previously received onasemnogene abeparvovec.

The secondary objectives of this study are to evaluate the safety and tolerability; and clinical outcomes following treatment with nusinersen in participants with SMA who previously received onasemnogene abeparvovec.

intervention: Nusinersen

results: https://clinicaltrials.gov/ct2/show/results/NCT04488133

last updated: February 04, 2022

start date: January 7, 2021

estimated completion: September 4, 2024

last updated: December 6, 2021

phase of development: Phase 4

size / enrollment: 60

primary outcomes:

• Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score
  Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp (0 to 3), ability to kick in supine position (0 to 4), head control (0 to 2), rolling (0 to 3), sitting (0 to 4), crawling (0 to 4), standing (0 to 3), and walking (0 to 3). Total HINE score is the sum of points from each item and can range from 0 to 26, with higher scores depicting better level of ability.
• Up to Day 778
  

secondary outcomes:

• Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
  Up to Day 778
• Number of Participants with Change from Baseline in Clinical Laboratory Parameters
  Up to Day 778
• Number of Participants with Change from Baseline in Electrocardiograms (ECGs)
  Up to Day 778
• Number of Participants with Change from Baseline in Vital Signs
  Up to Day 778
• Number of Participants who Achieved Motor Milestones as Assessed by World Health Organization (WHO) Criteria
  Up to Day 778
• Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Score
  Up to Day 778
• Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
  Up to Day 778
• Change from Baseline in Revised Upper Limb Module (RULM) Score
  Up to Day 778
• Time to Death or Permanent Ventilation
  Up to Day 778

inclusion criteria:

exclusion criteria: Criteria:

Prior exposure to nusinersen
Ongoing severe or serious AEs related to onasemnogene abeparvovec

Note: Other protocol defined Inclusion/

sponsor: Biogen

contacts: US Biogen Clinical Trial Center, 866-633-4636, clinicaltrials@biogen.com

investigators: Medical Director,Biogen

trial center locations: United States,Israel,Italy,Spain

• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  Andres Nascimento, anascimento@sjdhospitalbarcelona.org
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  Riccardo Masson, +39 022394 2218, riccardo.masson@istituto-besta.it
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  Ignacio Pascual, 0034 917277388, ipascual@salud.madrid.org
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  312-227-3019, itejani@luriechildrens.org
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  314-362-2490, anandp@wustl.edu
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  501-364-3122, bryanar@archildrens.org
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  503-418-8297, mccarbry@ohsu.edu
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  617-312-8318, KSWOBODA@mgh.harvard.edu
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  630157062, mariarosaria.vizzino@policlinicogemelli.it
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  650-725-4341, neuromuscularresearch@stanford.edu
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  Heather Wendorf, 651-325-2314, hwendorf@gillettechildrens.com
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  678-883-6897, info@rarediseaseresearch.com
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  720-777-3293, Hannah.johnson@childrenscolorado.org
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  757-668-6417, cara.headrick@chkd.org
  
  
• A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
  Shelby Murdock, 801-858-9717, Shelby.murdock@hsc.utah.edu