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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02594124
Registration number
NCT02594124
Ethics application status
Date submitted
30/10/2015
Date registered
1/11/2015
Date last updated
6/02/2024
Titles & IDs
Public title
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
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Scientific title
An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443
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Secondary ID [1]
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2015-001870-16
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Secondary ID [2]
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ISIS 396443-CS11
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Universal Trial Number (UTN)
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Trial acronym
SHINE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Spinal Muscular Atrophy
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Neurological
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Other neurological disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional(has expanded access)
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Description of intervention(s) / exposure
Treatment: Drugs - nusinersen
Experimental: Group 1 - Participants transitioned from ISIS 396443-CS3B (NCT02193074)
Experimental: Group 2 - Participants transitioned from ISIS 396443-CS4 (NCT02292537)
Experimental: Group 3 - Participants transitioned from ISIS 396443-CS12 (NCT02052791)
Experimental: Group 4 - Participants transitioned from ISIS 396443-CS3A (NCT01839656)
Experimental: Group 5 - Participants transitioned from 232SM202 (NCT02462759)
Treatment: Drugs: nusinersen
Administered by intrathecal (IT) injection
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of participants experiencing Adverse events (AEs) and/or Serious Adverse Events (SAEs)
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Assessment method [1]
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Timepoint [1]
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Up to Day 1814
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Primary outcome [2]
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Number of participants with clinically significant vital sign abnormalities
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Assessment method [2]
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Timepoint [2]
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Up to Day 1814
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Primary outcome [3]
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Number of participants with clinically significant weight abnormalities
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Assessment method [3]
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Timepoint [3]
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Up to Day 1814
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Primary outcome [4]
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Number of participants with clinically significant neurological examination abnormalities
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Assessment method [4]
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Timepoint [4]
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Up to Day 1814
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Primary outcome [5]
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Number of participants with clinically significant laboratory assessment abnormalities
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Assessment method [5]
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Timepoint [5]
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Up to Day 1814
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Primary outcome [6]
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Number of participants with clinically significant coagulation parameter abnormalities
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Assessment method [6]
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Timepoint [6]
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Up to Day 1814
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Primary outcome [7]
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Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities
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Assessment method [7]
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Timepoint [7]
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Up to Day 1814
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Primary outcome [8]
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Change from Baseline in concomitant medications
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Assessment method [8]
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Timepoint [8]
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Up to Day 1814
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Secondary outcome [1]
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Percentage of participants who attained motor milestones as assessed by World Health Organization (WHO) criteria
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Assessment method [1]
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Timepoint [1]
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Up to Day 1814
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Secondary outcome [2]
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Percentage of participants who attained motor milestones as assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE)
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Assessment method [2]
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Timepoint [2]
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Up to Day 1814
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Secondary outcome [3]
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Time to death or permanent ventilation
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Assessment method [3]
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Timepoint [3]
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Up to Day 1814
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Secondary outcome [4]
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Percentage of participants not requiring permanent ventilation
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Assessment method [4]
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Timepoint [4]
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Up to Day 1814
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Secondary outcome [5]
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Change from Baseline in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) motor function scale
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Assessment method [5]
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CHOP-INTEND tests includes 16 items 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.
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Timepoint [5]
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Up to Day 1814
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Secondary outcome [6]
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Change from Baseline in Hammersmith Functional Motor Scale
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Assessment method [6]
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The HFMSE tests motor function of participants with SMA. The original 20 item Hammersmith Functional Motor Scale was expanded to include 13 additional adapted items from the Gross Motor Function Measure to improve sensitivity for the higher functioning ambulant population.
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Timepoint [6]
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Up to Day 1814
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Secondary outcome [7]
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Change from Baseline in Revised Upper Limb Module (RULM)
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Assessment method [7]
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Timepoint [7]
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Up to Day 1814
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Secondary outcome [8]
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Change from Baseline in 6-Minute Walk Test (6MWT)
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Assessment method [8]
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6MWT: walking up and down a 25 meter track without aids or orthotics as fast as possible for 6 minutes. Lap splits, minute splits and total distance are recorded, in addition to any rests and falls.
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Timepoint [8]
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Up to Day 1814
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Secondary outcome [9]
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Change from Baseline in Compound Muscular Action Potential (CMAP)
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Assessment method [9]
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CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles.
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Timepoint [9]
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Up to Day 1814
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Secondary outcome [10]
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Change from Baseline in body length and/or height (for all participants)
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Assessment method [10]
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Timepoint [10]
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Up to Day 1814
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Secondary outcome [11]
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Change from Baseline in head circumference (for participants up to 36 months of age)
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Assessment method [11]
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Timepoint [11]
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Up to Day 1814
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Secondary outcome [12]
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Change from Baseline in chest circumference (for participants up to 36 months of age)
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Assessment method [12]
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Timepoint [12]
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Up to Day 1814
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Secondary outcome [13]
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Change from Baseline in arm circumference (for participants up to 36 months of age)
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Assessment method [13]
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Timepoint [13]
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Up to Day 1814
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Secondary outcome [14]
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Proportion of CMAP responders
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Assessment method [14]
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Timepoint [14]
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Up to Day 1814
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Secondary outcome [15]
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Number of participants with motor milestones achieved
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Assessment method [15]
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Timepoint [15]
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Up to Day 1814
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Secondary outcome [16]
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Proportion of participants who achieved standing alone
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Assessment method [16]
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Timepoint [16]
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Up to Day 1814
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Secondary outcome [17]
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Proportion of participants who achieved walking with assistance
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Assessment method [17]
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Timepoint [17]
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Up to Day 1814
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Secondary outcome [18]
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Number of participants with serious respiratory events
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Assessment method [18]
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Timepoint [18]
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Up to Day 1814
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Secondary outcome [19]
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Number of participants hospitalized
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Assessment method [19]
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Timepoint [19]
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Up to Day 1814
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Secondary outcome [20]
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Duration of hospitalizations
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Assessment method [20]
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Timepoint [20]
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Up to Day 1814
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Secondary outcome [21]
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Change from Baseline in Cobb-Angle on X-Ray of the thoracolumbar spine
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Assessment method [21]
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Timepoint [21]
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Up to Day 1814
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Secondary outcome [22]
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Change from Baseline in Quality of Life (QOL) Questionnaires
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Assessment method [22]
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Timepoint [22]
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Up to Day 1814
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Secondary outcome [23]
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Number of Disease-related hospitalizations and AEs
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Assessment method [23]
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Timepoint [23]
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Up to Day 1814
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Secondary outcome [24]
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Overall survival rate
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Assessment method [24]
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Timepoint [24]
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Up to Day 1814
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Eligibility
Key inclusion criteria
Key
* Signed informed consent of parent or guardian and signed informed assent of participant, if indicated per participant's age and institutional guidelines.
* Completion of the index study in accordance with the study protocol or as a result of Sponsor decision (e.g., early termination of the index study) within the preceding 16 weeks
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Have any condition or worsening condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study
* Clinically significant abnormalities in hematology or clinical chemistry parameters or electrocardiogram (ECG), as assessed by the Site Investigator, at the Screening visit that would render the participant unsuitable for participation in the study
* Participant's parent or legal guardian is not willing or able to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study
* Treatment with another investigational agent, biological agent, or device within one month of Screening, or 5 half-lives of study agent, whichever is longer
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
4/11/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
21/08/2023
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Sample size
Target
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Accrual to date
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Final
292
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Sydney Children's Hospital Clinical Research Centre - Sydney
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Recruitment hospital [2]
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Royal Children's Hospital - Parkville
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Recruitment postcode(s) [1]
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2031 - Sydney
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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United States of America
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Colorado
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United States of America
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Connecticut
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United States of America
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Florida
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Country [5]
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United States of America
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Illinois
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Country [6]
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United States of America
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State/province [6]
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Maryland
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Country [7]
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United States of America
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State/province [7]
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Massachusetts
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Country [8]
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United States of America
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State/province [8]
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Minnesota
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Country [9]
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United States of America
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State/province [9]
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Missouri
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Country [10]
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United States of America
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New York
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Country [11]
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United States of America
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State/province [11]
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North Carolina
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Country [12]
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United States of America
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State/province [12]
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Oregon
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Country [13]
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United States of America
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Pennsylvania
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Country [14]
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United States of America
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State/province [14]
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Texas
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Country [15]
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United States of America
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State/province [15]
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Utah
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Country [16]
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United States of America
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State/province [16]
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Washington
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Country [17]
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Belgium
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State/province [17]
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Brussels
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Country [18]
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Canada
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State/province [18]
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British Columbia
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Country [19]
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Canada
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State/province [19]
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Ontario
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Country [20]
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Canada
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State/province [20]
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Quebec
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Country [21]
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France
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State/province [21]
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Paris 9
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Country [22]
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Germany
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Bayern
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Country [23]
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Germany
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State/province [23]
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Essen
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Germany
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State/province [24]
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Freiburg
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Country [25]
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Hong Kong
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State/province [25]
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Hong Kong SAR
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Country [26]
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Italy
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State/province [26]
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Essen
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Italy
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Genova
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Italy
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Messina
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Japan
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Aichi
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Japan
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Hyogo
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Japan
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Tokyo
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Japan
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State/province [32]
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Kumamoto
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Japan
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State/province [33]
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Miyagi
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Country [34]
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Japan
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State/province [34]
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Miyazaki
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Country [35]
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Korea, Republic of
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Korea
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Country [36]
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Spain
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Barcelona
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Spain
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Hebron
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Spain
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State/province [38]
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Madrid
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Country [39]
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Sweden
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State/province [39]
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Gothenburg
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Country [40]
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Turkey
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State/province [40]
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Ankara
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Turkey
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State/province [41]
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Istanbul
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Country [42]
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United Kingdom
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State/province [42]
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Northumberland
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Country [43]
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United Kingdom
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State/province [43]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Biogen
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The primary objective is to evaluate the long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.
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Trial website
https://clinicaltrials.gov/study/NCT02594124
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Director
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Address
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Biogen
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT02594124
Download to PDF