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Trial details
Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen
Current status:
Approved
|
Date registered:
09/01/2023
Trial version(s)
Current: 08/09/2022
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2022105117
Protocol number
232SM302
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Prospective
Date of registration in national regulatory agency
18/10/2022
Primary sponsor
Biogen MA Inc.
Primary sponsor: Country of origin
United States of America
Public title
Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen
Acronym
Scientific 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
Acronym
Brief summary of the study: English
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).
Brief summary of the study: Arabic
الهدف الأساسي من هذه الدراسة هو تقييم السلامة على المدى الطويل وقابلية تحمل nusinersen المعطى داخل القراب بجرعات أعلى للمشاركين المصابين بضمور العضلات الشوكي (SMA) الذين شاركوا سابقًا في دراسة 232SM203 (NCT04089566). الهدف الثانوي لهذه الدراسة هو تقييم الفعالية طويلة المدى لـ nusinersen المعطى داخل القراب بجرعات أعلى للمشاركين مع SMA الذين شاركوا سابقًا في الدراسة 232SM203 (NCT04089566).
Health conditions/problem studied: Specify
Muscular Atrophy, Spinal
Interventions: Specify
Drug: Nusinersen (BIIB058) Administered as specified in the treatment arm
Key inclusion and exclusion criteria: Inclusion criteria
Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the study protocol.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
1
Key inclusion and exclusion criteria: Age maximum
11
Key inclusion and exclusion criteria: Exclusion criteria
- Treatment with another investigational therapy or enrollment in another interventional clinical study after the Day 302 visit in study 232SM203 (NCT04089566). - Treatment with Zolgensma (all participants) after the Day 302 visit of study 232SM203 (NCT04089566). - Treatment with an approved therapy for SMA (other than Zolgensma) that is inconsistent with protocol requirements for allowed or disallowed concomitant therapies. NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Other
Trial scope: Specify scope
Safety, tolerability and efficacy
Study design: Allocation
Non-randomized controlled trial
Study design: Masking
Blinded (masking used)
Study design: Control
N/A
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Parallel
IMP has market authorization
Yes, Worldwide
IMP has market authorization: Specify the countries
USA- Europe
Name of IMP
Nusinersen
Year of authorization
2016
Month of authorization
12
Type of IMP
Others
Type of IMP: Specify
Pharmaceutical
Pharmaceutical class
Antisense oligonucleotide inhibitors
Therapeutic indication
Muscular Atrophy, Spinal
Therapeutic benefit
People with Spinal Muscular Atrophy have an absence of survival of motor neuron (SMN) proteins; nusinersen is an antisense oligonucleotide that increases production of SMN leading to an improvement of their condition.
Biospecimen retention
Samples with DNA**
Biospecimen description
Plasma, urine, and CSF samples will be collected using standardized procedures. Sample volume will be determined by weight of the patient using standardized volume limitations. Biological samples will be analyzed for PK and Biomarker concentrations.
Target sample size
5
Actual enrollment target size
2
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
15/01/2023
Date of study closure: Type
Anticipated
Date of study closure: Date
30/09/2027
Recruitment status
Recruiting
Date of completion
IPD sharing statement plan
Yes
IPD sharing statement description
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on http://clinicalresearch.biogen.com/
Additional data URL
https://vivli.org/
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
ClinicalTrials.gov
NCT04729907
EudraCT
2020-004708-32
Sources of Monetary or Material Support
Name
Biogen
Secondary Sponsors
Name
N/A
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Dr. Hicham
Mansour
Beirut
Lebanon
-
hicham.mansour@gmail.com
SGUMC
Scientific
Medical
Director
Innovation House, 70 Norden Road, Maidenhead SL6 4AY
United Kingdom
-
Clinicaltrials@biogen.com
Biogen Idec Research Limited
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Saint George University Medical Center
Dr. Hicham Mansour
Pediatric Neurology and Metabolic Diseases
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Saint George Hospital University Medical Center
10/02/2022
Dr. Michel Daher
-
+961 1 566780
Countries of Recruitment
Name
Lebanon
United States of America
Brazil
Estonia
Germany
Japan
Taiwan
Canada
Russian Federation
Poland
Spain
Health Conditions or Problems Studied
Condition
Code
Keyword
Spinal Muscular Atrophy
Spinal muscular atrophy and related syndromes (G12)
Muscular Atrophy, Spinal, Atrophy Pathological Conditions, Anatomical Neuromuscular Manifestations, Neurologic Manifestations, Nervous System Diseases, Spinal Cord Diseases, Central Nervous System Diseases, Motor Neuron Disease, Neurodegenerative Diseases, Neuromuscular Diseases
Interventions
Intervention
Description
Keyword
Drug
Nusinersen (BIIB058)
-
Primary Outcomes
Name
Time points
Measure
To evaluate the long-term safety and tolerability of nusinersen
Up to Day 1081
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
To evaluate the long-term safety and tolerability of nusinersen
Up to Day 1081
Change from Baseline in Growth Parameters
To evaluate the long-term safety and tolerability of nusinersen
Up to Day 1081
Number of Participants With Shifts from Baseline in Clinical Laboratory Parameters
To evaluate the long-term safety and tolerability of nusinersen
Up to Day 1081
Number of Participants With Shifts from Baseline in Electrocardiogram (ECG)
To evaluate the long-term safety and tolerability of nusinersen
Up to Day 1081
Number of Participants With Shifts from Baseline in Vital Signs
To evaluate the long-term safety and tolerability of nusinersen
Up to Day 961
Change from Baseline in Activated Partial Thromboplastin Time (aPTT)
To evaluate the long-term safety and tolerability of nusinersen
Up to Day 961
Change from Baseline in Prothrombin Time (PT)
To evaluate the long-term safety and tolerability of nusinersen
Up to Day 961
Change from Baseline in International Normalized Ratio (INR)
Key Secondary Outcomes
Name
Time points
Measure
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Total Number of New World Health Organization (WHO) Motor Milestones
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Number of Participants Who Used Respiratory Support, by Type
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Number of Hours Per Day of Respiratory Support
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Number of Days That Respiratory Support Is Used
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Time to Death (Overall Survival)
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Total Score
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Percentage of HINE Section 2 Motor Milestone Responders
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Percentage of Time Spent on Ventilation
To evaluate the long-term efficacy of nusinersen
Up to Day 1081
Time to Death or Permanent Ventilation
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial