A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
NCT ID: NCT02193074
Last Updated: 2021-02-17
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
TERMINATED
PHASE3
122 participants
INTERVENTIONAL
2014-08-19
2016-11-21
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
NCT02386553
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
NCT02292537
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
NCT02462759
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)
NCT02052791
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
NCT02594124
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
In August 2016, sponsorship of the trial was transferred to Biogen.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
nusinersen
nusinersen
Administered by intrathecal (IT) injection as specified in the treatment arm.
Sham procedure
Sham procedure
Small needle prick on the lower back at the location where the IT injection is normally made
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
nusinersen
Administered by intrathecal (IT) injection as specified in the treatment arm.
Sham procedure
Small needle prick on the lower back at the location where the IT injection is normally made
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
Exclusion Criteria
* 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
210 Days
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Biogen
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Medical Director
Role: STUDY_DIRECTOR
Biogen
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
UCLA Medical Center
Los Angeles, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours Children's Hospital
Orlando, Florida, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University School of Medicine
St Louis, Missouri, United States
Columbia University Medical Center
New York, New York, United States
Duke Children's Hospital
Durham, North Carolina, United States
Doernbecher Children's Hospital
Portland, Oregon, United States
Children's Hospital of Philadelphia - Neurology
Philadelphia, Pennsylvania, United States
UT Southwestern Medical Center/Children's Medical Center Dallas
Dallas, Texas, United States
Primary Children's Medical Center (University of Utah)
Salt Lake City, Utah, United States
Sydney Children's Hospital
Sydney, New South Wales, Australia
Royal Children's Hospital, Children's Neuroscience Centre
Parkville, Victoria, Australia
Hôpital Universitaire des Enfants Reine FABIOLA (HUDERF)
Brussels, , Belgium
British Columbia Children's Hospital/UBC
Vancouver, British Columbia, Canada
Hospital for Sick Children
Toronto, Ontario, Canada
Institut de Myologie
Paris, , France
Universitatsklinikum Essen
Essen, , Germany
Universtatsklinikum Freiburg, Zentrum fur Kinder-und Jugendmedizin , Abteilung Neuropadiatrie und Muskelerkrankungen
Freiburg im Breisgau, , Germany
Istituto Giannina Gaslini, Centro Traslazionale di Miologia e Patologie Neurodegenerative
Genova, , Italy
Pediatric Neurology Unit, Catholic University
Rome, , Italy
Hyogo College of Medicine
Nishinomiya, Hyōgo, Japan
Tokyo Women's Medical University
Tokyo, , Japan
Seoul National University Hospital
Seoul, , South Korea
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Universitario La Paz, Pediatric Neurology Department
Madrid, , Spain
University of Gothenburg, The Queen Silvia Children's Hospital
Gothenburg, , Sweden
Hacettepe Children's Hospital
Ankara, , Turkey (Türkiye)
UCL Institute of Child Health/Great Ormond Street
London, , United Kingdom
MRC Centre for Neuromuscular Diseases at Newcastle, Institute of Genetic Medicine Newcastle University
Newcastle, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Darras BT, Farrar MA, Mercuri E, Finkel RS, Foster R, Hughes SG, Bhan I, Farwell W, Gheuens S. An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials. CNS Drugs. 2019 Sep;33(9):919-932. doi: 10.1007/s40263-019-00656-w.
Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guerin A, Pivneva I, Wu EQ, Arjunji R, Feltner D, Sproule DM. Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1. Adv Ther. 2019 May;36(5):1164-1176. doi: 10.1007/s12325-019-00923-8. Epub 2019 Mar 16.
Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J, Chiriboga CA, Saito K, Servais L, Tizzano E, Topaloglu H, Tulinius M, Montes J, Glanzman AM, Bishop K, Zhong ZJ, Gheuens S, Bennett CF, Schneider E, Farwell W, De Vivo DC; ENDEAR Study Group. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752.
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.
Related Links
Access external resources that provide additional context or updates about the study.
Cure SMA
Muscular Dystrophy Association
National Organization for Rare Diseases
Clinical Study Report (CSR) Synopsis - a results summary
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2013-004422-29
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ISIS 396443-CS3B
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.