Back to resultsExtension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen (ONWARD)
Primary Purpose
Muscular Atrophy, Spinal
Status
Enrolling by invitation
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Nusinersen
Sponsored by
About this trial
This is an interventional treatment trial for Muscular Atrophy, Spinal
Eligibility Criteria
Key Inclusion Criteria:
- Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the study protocol
Key Exclusion Criteria:
- Treatment with another investigational therapy or enrollment in another interventional clinical study
- Treatment with an approved therapy for SMA after the Day 302 Visit of Study 232SM203 (NCT04089566)
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Sites / Locations
- Stanford University Medical Center
- Children's Hospital Colorado
- Ann & Robert H. Lurie Children's Hospital of Chicago
- The Johns Hopkins Hospital
- Boston Children's Hospital
- St. Jude Children's Research Hospital
- Children's Medical Center
- HC-UFMG - Hospital das Clinicas da Universidade Federal de Minas Gerais
- Hospital de Clínicas de Porto Alegre
- Hospital das Clinicas - FMUSP
- London Health Sciences Centre (LHSC) - Children's Hospital
- Hospital Luis Calvo Mackenna
- Clinica Las Condes
- Clinica MEDS La Dehesa
- Guangzhou Woman and Children's Medical Center
- Hospital Universitario San Ignacio
- Fundacion Hospitalaria San Vicente de Paul
- Tallinn Children's Hospital
- Universitaetsklinikum Freiburg
- Universitaetsklinikum Giessen und Marburg GmbH
- Fondazione Serena Onlus - Centro Clinico Nemo
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Kurume University Hospital
- Hyogo Medical University Hospital
- Tokyo Women's Medical University Hospital
- Saint George University Hospital Medical Center
- Instituto Nacional de Pediatria
- Hospital Infantil de Mexico Federico Gomez
- Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde
- Instytut 'Pomnik - Centrum Zdrowia Dziecka'
- Regional Pediatric Clinical Hospital #1
- Russian Children Neuromuscular Center of Veltischev
- King Fahad Specialist Hospital
- National Guard Health Affairs: King Abdulaziz Medical City
- King Faisal Specialist Hospital & Research Center
- Hospital Universitario La Paz
- Hospital Universitari i Politecnic La Fe
- Kaohsiung Medical University Chung-Ho Memorial Hospital
- National Taiwan University Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
BIIB058 28 mg (Prior Maintenance Dose 28 mg)
BIIB058 50/28 mg (Prior Maintenance Dose 12 mg)
Arm Description
Participants who received maintenance dose of 28 milligram (mg) nusinersen in study 232SM203 (NCT04089566), will receive maintenance dose of 28 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 961.
Participants who received maintenance dose of 12 mg nusinersen in study 232SM203 (NCT04089566), will receive loading dose of 50 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 961.
Outcomes
Primary Outcome Measures
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a birth defect, or is a medically important event.
Change from Baseline in Growth Parameters
Growth parameters will be assessed by measuring body length or height (if feasible and appropriate), ulnar length (all participants), and head circumference, chest circumference, and arm circumference (all participants 3 years of age and younger) in centimeters.
Number of Participants With Shifts from Baseline in Clinical Laboratory Parameters
Number of Participants With Shifts from Baseline in Electrocardiogram (ECG)
Number of Participants With Shifts from Baseline in Vital Signs
Change from Baseline in Activated Partial Thromboplastin Time (aPTT)
Change from Baseline in Prothrombin Time (PT)
Change from Baseline in International Normalized Ratio (INR)
Change from Baseline in Urine Total Protein
Change from Baseline in Neurological Examination Outcomes for Participants ≤2 Years of Age
For participants 2 years of age and younger, the Hammersmith Infant Neurological Exam (HINE) Sections 1 and 3 will be conducted. This standard examination (developed by [Dubowitz and Dubowitz 1981]) is a quantitative scorable method for assessing the neurological development of infants between 2 and 24 months of age. The examination includes assessment of cranial nerve functions, posture, movements, tone, and reflexes. The HINE Section 1 form utilized in ONWARD contains 26 items and the Section 3 form utilized contains 3 items. For HINE Section 1 items, each item is scored 0-3. For HINE Section 3 items, scoring is variable (1-4, 1-5, or 1-6). Higher scores indicate better neurological function.
Number of Participants with Change from Baseline in Neurological Examination Outcomes for Participants >2 Years of Age
For all participants >2 years of age, standard neurological examinations, which include assessments of mental status, level of consciousness, sensory function, motor function, cranial nerve function, and reflexes, will be conducted.
Percentage of Participants With a Postbaseline Platelet Count Below the Lower Limit of Normal on at least 2 Consecutive Measurements
Percentage of Participants With a Postbaseline Corrected QT Interval Using Fridericia's Formula (QTcF) of >500 millisecond (msec) and an Increase from Baseline to Any Postbaseline Timepoint in QTcF of >60 msec
Secondary Outcome Measures
Total Number of New World Health Organization (WHO) Motor Milestones
Number of Participants Who Used Respiratory Support, by Type
Number of Hours Per Day of Respiratory Support
Number of Days That Respiratory Support Is Used
Time to Death (Overall Survival)
Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Total Score
The CHOP INTEND test is designed to evaluate the motor skills of infants with significant motor weakness. It includes 16 items (capturing neck, trunk, and proximal and distal limb strength) structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0-4. The total score ranges from 0-64, with higher scores depicting better response. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones
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. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Percentage of HINE Section 2 Motor Milestone Responders
Section 2 of HINE is used to assess motor milestones of 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 sum of points from each item and can range from 0 to 26, with higher scores depicting better level of ability. HINE section 2 motor milestone responder is participant who demonstrates at least 2-point increase in category of ability to kick or increase to maximal score on that category or 1-point increase in motor milestones category of head control, rolling, sitting, crawling, standing, or walking, and among 7 motor milestone categories (excluding voluntary grasp), participant demonstrates improvement in more categories than worsening. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Percentage of Time Spent on Ventilation
This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Time to Death or Permanent Ventilation
Permanent ventilation is defined as tracheostomy or ≥16 hours of ventilation/day continuously for >21 days in the absence of an acute reversible event. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
The HFMSE is a tool used to assess motor function in children with SMA. The original 20 item Hammersmith Functional Motor Scale (HFMS) was expanded to include 13 additional items to improve sensitivity for the higher functioning ambulant population. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all activities, with a maximum score of 66 with higher scores depicting better ability to perform activities. Participants ≥ 2 years of age (at the time of the study visit) will be evaluated with HFMSE.
Change from Baseline in Revised Upper Limb Module (RULM) Score
The RULM is developed to assess upper limb functional abilities participants with SMA. This test consists of upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function. Participants ≥ 2 years of age (at the time of the study visit) will be evaluated with RULM.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04729907
Brief Title
Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen
Acronym
ONWARD
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
April 19, 2021 (Actual)
Primary Completion Date
May 30, 2026 (Anticipated)
Study Completion Date
May 30, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biogen
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The primary objective of this study is to evaluate the long-term safety and tolerability of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA) who previously participated in study 232SM203 (NCT04089566).
The secondary objective of this study is to evaluate the long-term efficacy of nusinersen administered intrathecally at higher doses to participants with SMA who previously participated in study 232SM203 (NCT04089566).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Atrophy, Spinal
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Non-Randomized
Enrollment
172 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
BIIB058 28 mg (Prior Maintenance Dose 28 mg)
Arm Type
Experimental
Arm Description
Participants who received maintenance dose of 28 milligram (mg) nusinersen in study 232SM203 (NCT04089566), will receive maintenance dose of 28 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 961.
Arm Title
BIIB058 50/28 mg (Prior Maintenance Dose 12 mg)
Arm Type
Experimental
Arm Description
Participants who received maintenance dose of 12 mg nusinersen in study 232SM203 (NCT04089566), will receive loading dose of 50 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 961.
Intervention Type
Drug
Intervention Name(s)
Nusinersen
Other Intervention Name(s)
BIIB058, Spinraza
Intervention Description
Administered as specified in the treatment arm
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Description
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a birth defect, or is a medically important event.
Time Frame
Up to Day 1081
Title
Change from Baseline in Growth Parameters
Description
Growth parameters will be assessed by measuring body length or height (if feasible and appropriate), ulnar length (all participants), and head circumference, chest circumference, and arm circumference (all participants 3 years of age and younger) in centimeters.
Time Frame
Up to Day 1081
Title
Number of Participants With Shifts from Baseline in Clinical Laboratory Parameters
Time Frame
Up to Day 1081
Title
Number of Participants With Shifts from Baseline in Electrocardiogram (ECG)
Time Frame
Up to Day 1081
Title
Number of Participants With Shifts from Baseline in Vital Signs
Time Frame
Up to Day 1081
Title
Change from Baseline in Activated Partial Thromboplastin Time (aPTT)
Time Frame
Up to Day 961
Title
Change from Baseline in Prothrombin Time (PT)
Time Frame
Up to Day 961
Title
Change from Baseline in International Normalized Ratio (INR)
Time Frame
Up to Day 961
Title
Change from Baseline in Urine Total Protein
Time Frame
Up to Day 1081
Title
Change from Baseline in Neurological Examination Outcomes for Participants ≤2 Years of Age
Description
For participants 2 years of age and younger, the Hammersmith Infant Neurological Exam (HINE) Sections 1 and 3 will be conducted. This standard examination (developed by [Dubowitz and Dubowitz 1981]) is a quantitative scorable method for assessing the neurological development of infants between 2 and 24 months of age. The examination includes assessment of cranial nerve functions, posture, movements, tone, and reflexes. The HINE Section 1 form utilized in ONWARD contains 26 items and the Section 3 form utilized contains 3 items. For HINE Section 1 items, each item is scored 0-3. For HINE Section 3 items, scoring is variable (1-4, 1-5, or 1-6). Higher scores indicate better neurological function.
Time Frame
Up to Day 1081
Title
Number of Participants with Change from Baseline in Neurological Examination Outcomes for Participants >2 Years of Age
Description
For all participants >2 years of age, standard neurological examinations, which include assessments of mental status, level of consciousness, sensory function, motor function, cranial nerve function, and reflexes, will be conducted.
Time Frame
Up to Day 1081
Title
Percentage of Participants With a Postbaseline Platelet Count Below the Lower Limit of Normal on at least 2 Consecutive Measurements
Time Frame
Up to Day 1081
Title
Percentage of Participants With a Postbaseline Corrected QT Interval Using Fridericia's Formula (QTcF) of >500 millisecond (msec) and an Increase from Baseline to Any Postbaseline Timepoint in QTcF of >60 msec
Time Frame
Up to Day 1081
Secondary Outcome Measure Information:
Title
Total Number of New World Health Organization (WHO) Motor Milestones
Time Frame
Up to Day 1081
Title
Number of Participants Who Used Respiratory Support, by Type
Time Frame
Up to Day 1081
Title
Number of Hours Per Day of Respiratory Support
Time Frame
Up to Day 1081
Title
Number of Days That Respiratory Support Is Used
Time Frame
Up to Day 1081
Title
Time to Death (Overall Survival)
Time Frame
Up to Day 1081
Title
Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Total Score
Description
The CHOP INTEND test is designed to evaluate the motor skills of infants with significant motor weakness. It includes 16 items (capturing neck, trunk, and proximal and distal limb strength) structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0-4. The total score ranges from 0-64, with higher scores depicting better response. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Time Frame
Up to Day 1081
Title
Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones
Description
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. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Time Frame
Up to Day 1081
Title
Percentage of HINE Section 2 Motor Milestone Responders
Description
Section 2 of HINE is used to assess motor milestones of 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 sum of points from each item and can range from 0 to 26, with higher scores depicting better level of ability. HINE section 2 motor milestone responder is participant who demonstrates at least 2-point increase in category of ability to kick or increase to maximal score on that category or 1-point increase in motor milestones category of head control, rolling, sitting, crawling, standing, or walking, and among 7 motor milestone categories (excluding voluntary grasp), participant demonstrates improvement in more categories than worsening. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Time Frame
Up to Day 1081
Title
Percentage of Time Spent on Ventilation
Description
This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Time Frame
Up to Day 1081
Title
Time to Death or Permanent Ventilation
Description
Permanent ventilation is defined as tracheostomy or ≥16 hours of ventilation/day continuously for >21 days in the absence of an acute reversible event. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Time Frame
Up to Day 1081
Title
Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
Description
The HFMSE is a tool used to assess motor function in children with SMA. The original 20 item Hammersmith Functional Motor Scale (HFMS) was expanded to include 13 additional items to improve sensitivity for the higher functioning ambulant population. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all activities, with a maximum score of 66 with higher scores depicting better ability to perform activities. Participants ≥ 2 years of age (at the time of the study visit) will be evaluated with HFMSE.
Time Frame
Up to Day 1081
Title
Change from Baseline in Revised Upper Limb Module (RULM) Score
Description
The RULM is developed to assess upper limb functional abilities participants with SMA. This test consists of upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function. Participants ≥ 2 years of age (at the time of the study visit) will be evaluated with RULM.
Time Frame
Up to Day 1081
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the study protocol
Key Exclusion Criteria:
Treatment with another investigational therapy or enrollment in another interventional clinical study
Treatment with an approved therapy for SMA after the Day 302 Visit of Study 232SM203 (NCT04089566)
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
Stanford University Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Children's Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611-260
Country
United States
Facility Name
The Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
St. Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
Facility Name
Children's Medical Center
City
Plano
State/Province
Texas
ZIP/Postal Code
75024
Country
United States
Facility Name
HC-UFMG - Hospital das Clinicas da Universidade Federal de Minas Gerais
City
Horizonte
ZIP/Postal Code
30130-100
Country
Brazil
Facility Name
Hospital de Clínicas de Porto Alegre
City
Porto Alegre
ZIP/Postal Code
90035-903
Country
Brazil
Facility Name
Hospital das Clinicas - FMUSP
City
São Paulo
ZIP/Postal Code
5403900
Country
Brazil
Facility Name
London Health Sciences Centre (LHSC) - Children's Hospital
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada
Facility Name
Hospital Luis Calvo Mackenna
City
Santiago
ZIP/Postal Code
7500539
Country
Chile
Facility Name
Clinica Las Condes
City
Santiago
ZIP/Postal Code
7550000
Country
Chile
Facility Name
Clinica MEDS La Dehesa
City
Santiago
ZIP/Postal Code
7691236
Country
Chile
Facility Name
Guangzhou Woman and Children's Medical Center
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510623
Country
China
Facility Name
Hospital Universitario San Ignacio
City
Bogota
ZIP/Postal Code
110231
Country
Colombia
Facility Name
Fundacion Hospitalaria San Vicente de Paul
City
Medellin
ZIP/Postal Code
50010
Country
Colombia
Facility Name
Tallinn Children's Hospital
City
Tallinn
ZIP/Postal Code
13419
Country
Estonia
Facility Name
Universitaetsklinikum Freiburg
City
Freiburg
State/Province
Baden Wuerttemberg
ZIP/Postal Code
79106
Country
Germany
Facility Name
Universitaetsklinikum Giessen und Marburg GmbH
City
Giessen
State/Province
Hessen
ZIP/Postal Code
35392
Country
Germany
Facility Name
Fondazione Serena Onlus - Centro Clinico Nemo
City
Milano
ZIP/Postal Code
20162
Country
Italy
Facility Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
City
Roma
ZIP/Postal Code
168
Country
Italy
Facility Name
Kurume University Hospital
City
Kurume-shi
State/Province
Fukuoka-Ken
ZIP/Postal Code
830-0011
Country
Japan
Facility Name
Hyogo Medical University Hospital
City
Nishinomiya-shi
State/Province
Hyogo-Ken
ZIP/Postal Code
663-8501
Country
Japan
Facility Name
Tokyo Women's Medical University Hospital
City
Shinjuku-ku
State/Province
Tokyo-To
ZIP/Postal Code
162-8666
Country
Japan
Facility Name
Saint George University Hospital Medical Center
City
Beirut
ZIP/Postal Code
11 00 2807
Country
Lebanon
Facility Name
Instituto Nacional de Pediatria
City
Mexico
State/Province
Distrito Federal
ZIP/Postal Code
4530
Country
Mexico
Facility Name
Hospital Infantil de Mexico Federico Gomez
City
Mexico
State/Province
Distrito Federal
ZIP/Postal Code
6720
Country
Mexico
Facility Name
Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde
City
Guadalajara
ZIP/Postal Code
44280
Country
Mexico
Facility Name
Instytut 'Pomnik - Centrum Zdrowia Dziecka'
City
Warszawa
ZIP/Postal Code
04-730
Country
Poland
Facility Name
Regional Pediatric Clinical Hospital #1
City
Ekaterinburg
ZIP/Postal Code
620149
Country
Russian Federation
Facility Name
Russian Children Neuromuscular Center of Veltischev
City
Moskva
ZIP/Postal Code
125412
Country
Russian Federation
Facility Name
King Fahad Specialist Hospital
City
Dammam
ZIP/Postal Code
31444
Country
Saudi Arabia
Facility Name
National Guard Health Affairs: King Abdulaziz Medical City
City
Jeddah
ZIP/Postal Code
21423
Country
Saudi Arabia
Facility Name
King Faisal Specialist Hospital & Research Center
City
Riyadh
ZIP/Postal Code
11211
Country
Saudi Arabia
Facility Name
Hospital Universitario La Paz
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Hospital Universitari i Politecnic La Fe
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Kaohsiung Medical University Chung-Ho Memorial Hospital
City
Kaohsiung
ZIP/Postal Code
807
Country
Taiwan
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
IPD Sharing URL
https://vivli.org/
Links:
URL
https://www.sma-europe.eu/
Description
SMA Europe
URL
https://www.curesma.org/
Description
CureSMA
URL
http://www.mda.org/disease/spinal-muscular-atrophy
Description
Muscular Dystrophy Association
Learn more about this trial
Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen
We'll reach out to this number within 24 hrs