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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02386553
Registration number
NCT02386553
Ethics application status
Date submitted
27/02/2015
Date registered
12/03/2015
Date last updated
16/05/2024
Titles & IDs
Public title
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
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Scientific title
An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
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Secondary ID [1]
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2014-002098-12
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Secondary ID [2]
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232SM201
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Universal Trial Number (UTN)
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Trial acronym
NURTURE
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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: Nusinersen - Nusinersen administered as an intrathecal injection
Treatment: Drugs: Nusinersen
Solution for intrathecal 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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Time to death or respiratory intervention
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Assessment method [1]
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The time will be the age of the participant at the first occurrence of either a respiratory intervention or death. Respiratory intervention is defined as invasive or noninvasive ventilation for =6 hours/day continuously for 7 or more days OR tracheostomy.
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Timepoint [1]
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Up to Day 2891
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Secondary outcome [1]
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Percentage of participants developing clinically manifested spinal muscular atrophy (SMA)
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Assessment method [1]
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Timepoint [1]
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At 13 and 24 months of age
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Secondary outcome [2]
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Percentage of participants alive
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Assessment method [2]
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Timepoint [2]
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At 13 months, and 2, 3, 4, 5, 6, 7 and 8 years of age
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Secondary outcome [3]
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Percentage of participants who attained motor milestones assessed as part of the Hammersmith Infant Neurological Examination (HINE)
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Assessment method [3]
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Timepoint [3]
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At 13 and 24 months of age
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Secondary outcome [4]
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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 [4]
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Timepoint [4]
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Up to Day 2891
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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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Timepoint [5]
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Up to Day 2891
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Secondary outcome [6]
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Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)
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Assessment method [6]
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Timepoint [6]
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Up to Day 2891
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Secondary outcome [7]
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Change from Baseline in weight for age/length
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Assessment method [7]
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Timepoint [7]
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Up to Day 2891
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Secondary outcome [8]
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Change from Baseline in head circumference
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Assessment method [8]
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Timepoint [8]
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Up to Day 2891
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Secondary outcome [9]
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Change from Baseline in chest circumference ratio
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Assessment method [9]
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Timepoint [9]
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Up to Day 2891
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Secondary outcome [10]
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Change from Baseline in head to chest circumference ratio
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Assessment method [10]
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Timepoint [10]
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Up to Day 2891
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Secondary outcome [11]
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Change from Baseline in arm circumference ratio
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Assessment method [11]
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Timepoint [11]
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Up to Day 2891
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Secondary outcome [12]
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Incidence of adverse events (AEs) and/or serious adverse events (SAEs)
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Assessment method [12]
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Timepoint [12]
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Up to Day 2891
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Secondary outcome [13]
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Change from Baseline in clinical laboratory parameters
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Assessment method [13]
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Assessed by the following laboratory tests: Hematology: red blood cells, hemoglobin, hematocrit, platelets, white blood cells, white blood cell differential, Blood chemistry: total protein, albumin, creatinine, cystatin C, creatine phosphokinase, blood urea nitrogen, total bilirubin (direct and indirect), alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, glucose, calcium, phosphorous, chloride, sodium, potassium. Urinalysis: specific gravity, pH, protein, glucose, ketones, bilirubin, red blood cells, white blood cells, epithelial cells, bacteria, casts, crystals
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Timepoint [13]
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Up to Day 2891
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Secondary outcome [14]
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Change from Baseline in electrocardiograms (ECGs)
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Assessment method [14]
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Timepoint [14]
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Up to Day 2891
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Secondary outcome [15]
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Change from Baseline in vital signs
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Assessment method [15]
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Vital sign will be assessed by: temperature, pulse rate, resting systolic and diastolic blood pressure, and respiratory rate.
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Timepoint [15]
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Up to Day 2891
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Secondary outcome [16]
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Change from Baseline in neurological examinations
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Assessment method [16]
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Timepoint [16]
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Up to Day 2891
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Secondary outcome [17]
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Cerebrospinal fluid (CSF) and plasma Nusinersen concentrations
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Assessment method [17]
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Timepoint [17]
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Up to Day 2801
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Eligibility
Key inclusion criteria
Key
* Age = 6 weeks at first dose.
* Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation.
* Genetic documentation of 2 or 3 copies of survival motor neuron 2 (SMN2).
* Ulnar compound muscle action potential (CMAP) = 1 mV at Baseline.
* Gestational age of 37 to 42 weeks for singleton births; gestational age of 34 to 42 weeks for twins.
* Meet additional study related criteria.
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Minimum age
6
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Maximum age
6
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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
* Hypoxemia (oxygen saturation <96% awake or asleep without any supplemental oxygen or respiratory support).
* Any clinical signs or symptoms at Screening or immediately prior to the first dosing (Day 1) that are, in the opinion of the Investigator, strongly suggestive of SMA.
* Clinically significant abnormalities in hematology or clinical chemistry parameters.
* Treatment with an investigational drug given for the treatment of SMA biological agent, or device. Any history of gene therapy, prior antisense oligonucleotide (ASO) treatment, or cell transplantation.
* Meet additional study related criteria.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
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Study design
Purpose of the study
Prevention
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Allocation to intervention
NA
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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
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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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
18/05/2015
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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
31/12/2024
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Actual
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Sample size
Target
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Accrual to date
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Final
25
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Recruitment in Australia
Recruitment state(s)
QLD,VIC
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Recruitment hospital [1]
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Queensland Children's Hospital - South Brisbane
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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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4101 - South Brisbane
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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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State/province [2]
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Colorado
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Country [3]
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United States of America
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State/province [3]
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Florida
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Country [4]
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United States of America
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State/province [4]
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Illinois
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Country [5]
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United States of America
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State/province [5]
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Maryland
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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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Massachusetts
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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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New York
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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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Pennsylvania
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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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Utah
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Country [10]
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United States of America
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State/province [10]
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Washington
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Country [11]
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Argentina
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State/province [11]
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Ciudad Autonoma Buenos Aires
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Country [12]
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Canada
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State/province [12]
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Alberta
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Country [13]
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Germany
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State/province [13]
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Baden-Württemberg
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Country [14]
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Israel
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State/province [14]
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Tel Aviv
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Country [15]
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Italy
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State/province [15]
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Lazio
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Country [16]
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Italy
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State/province [16]
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Milano
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Country [17]
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Qatar
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State/province [17]
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Doha
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Country [18]
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Taiwan
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State/province [18]
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Kaohsiung
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Country [19]
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Taiwan
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State/province [19]
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Taipei
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Country [20]
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Turkey
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State/province [20]
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Ankara
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Country [21]
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Turkey
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State/province [21]
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Istanbul
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Country [22]
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United Kingdom
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State/province [22]
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Greater 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 of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.
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Trial website
https://clinicaltrials.gov/study/NCT02386553
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Trial related presentations / publications
Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, Yamashita M, Rigo F, Hung G, Schneider E, Norris DA, Xia S, Bennett CF, Bishop KM. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016 Dec 17;388(10063):3017-3026. doi: 10.1016/S0140-6736(16)31408-8. Epub 2016 Dec 7.
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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/NCT02386553
Download to PDF