Status:
TERMINATED
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
Lead Sponsor:
Biogen
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
Up to 210 years
Phase:
PHASE3
Brief Summary
The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal musc...
Detailed Description
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc.. In August 2016, sponsorship of the trial was transferred to Biogen.
Eligibility Criteria
Inclusion
- Key
- Be born (gestational age) between 37 and 42 weeks
- Be medically diagnosed with spinal muscular atrophy (SMA)
- Have Survival Motor Neuron2 (SMN2) Copy number = 2
- Body weight equal to or greater than 3rd percentile for age using appropriate country-specific guidelines
- Be able to follow all study procedures
- Reside within approximately 9 hours ground-travel distance from a participating study center, for the duration of the study
- Key
Exclusion
- Hypoxemia (oxygen \[O2\] saturation awake less than 96% or O2 saturation asleep less than 96%, without ventilation support) during screening evaluation
- 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 to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Key Trial Info
Start Date :
August 19 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 21 2016
Estimated Enrollment :
122 Patients enrolled
Trial Details
Trial ID
NCT02193074
Start Date
August 19 2014
End Date
November 21 2016
Last Update
February 17 2021
Active Locations (31)
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1
UCLA Medical Center
Los Angeles, California, United States, 90095
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
3
Nemours Children's Hospital
Orlando, Florida, United States, 32827
4
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611