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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
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
Secondary ID [1] 0 0
2014-002098-12
Secondary ID [2] 0 0
232SM201
Universal Trial Number (UTN)
Trial acronym
NURTURE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Spinal Muscular Atrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - Nusinersen

Experimental: Nusinersen - Nusinersen administered as an intrathecal injection


Treatment: Drugs: Nusinersen
Solution for intrathecal injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Time to death or respiratory intervention
Timepoint [1] 0 0
Up to Day 2891
Secondary outcome [1] 0 0
Percentage of participants developing clinically manifested spinal muscular atrophy (SMA)
Timepoint [1] 0 0
At 13 and 24 months of age
Secondary outcome [2] 0 0
Percentage of participants alive
Timepoint [2] 0 0
At 13 months, and 2, 3, 4, 5, 6, 7 and 8 years of age
Secondary outcome [3] 0 0
Percentage of participants who attained motor milestones assessed as part of the Hammersmith Infant Neurological Examination (HINE)
Timepoint [3] 0 0
At 13 and 24 months of age
Secondary outcome [4] 0 0
Percentage of participants who attained motor milestones as assessed by World Health Organization (WHO) criteria
Timepoint [4] 0 0
Up to Day 2891
Secondary outcome [5] 0 0
Change from Baseline in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) motor function scale
Timepoint [5] 0 0
Up to Day 2891
Secondary outcome [6] 0 0
Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)
Timepoint [6] 0 0
Up to Day 2891
Secondary outcome [7] 0 0
Change from Baseline in weight for age/length
Timepoint [7] 0 0
Up to Day 2891
Secondary outcome [8] 0 0
Change from Baseline in head circumference
Timepoint [8] 0 0
Up to Day 2891
Secondary outcome [9] 0 0
Change from Baseline in chest circumference ratio
Timepoint [9] 0 0
Up to Day 2891
Secondary outcome [10] 0 0
Change from Baseline in head to chest circumference ratio
Timepoint [10] 0 0
Up to Day 2891
Secondary outcome [11] 0 0
Change from Baseline in arm circumference ratio
Timepoint [11] 0 0
Up to Day 2891
Secondary outcome [12] 0 0
Incidence of adverse events (AEs) and/or serious adverse events (SAEs)
Timepoint [12] 0 0
Up to Day 2891
Secondary outcome [13] 0 0
Change from Baseline in clinical laboratory parameters
Timepoint [13] 0 0
Up to Day 2891
Secondary outcome [14] 0 0
Change from Baseline in electrocardiograms (ECGs)
Timepoint [14] 0 0
Up to Day 2891
Secondary outcome [15] 0 0
Change from Baseline in vital signs
Timepoint [15] 0 0
Up to Day 2891
Secondary outcome [16] 0 0
Change from Baseline in neurological examinations
Timepoint [16] 0 0
Up to Day 2891
Secondary outcome [17] 0 0
Cerebrospinal fluid (CSF) and plasma Nusinersen concentrations
Timepoint [17] 0 0
Up to Day 2801

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.

Key
Minimum age
6 Weeks
Maximum age
6 Weeks
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Prevention
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/05/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2024
Actual
Sample size
Target
Accrual to date
Final
25
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [2] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
Utah
Country [10] 0 0
United States of America
State/province [10] 0 0
Washington
Country [11] 0 0
Argentina
State/province [11] 0 0
Ciudad Autonoma Buenos Aires
Country [12] 0 0
Canada
State/province [12] 0 0
Alberta
Country [13] 0 0
Germany
State/province [13] 0 0
Baden-Württemberg
Country [14] 0 0
Israel
State/province [14] 0 0
Tel Aviv
Country [15] 0 0
Italy
State/province [15] 0 0
Lazio
Country [16] 0 0
Italy
State/province [16] 0 0
Milano
Country [17] 0 0
Qatar
State/province [17] 0 0
Doha
Country [18] 0 0
Taiwan
State/province [18] 0 0
Kaohsiung
Country [19] 0 0
Taiwan
State/province [19] 0 0
Taipei
Country [20] 0 0
Turkey
State/province [20] 0 0
Ankara
Country [21] 0 0
Turkey
State/province [21] 0 0
Istanbul
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Greater London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Biogen
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Biogen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
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