NCT02386553CompletedPhase 2
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
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
Source: ClinicalTrials.gov · Last updated 20 Oct 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 25 (actual)
- First submitted
- 27 Feb 2015
- Start date
- 18 May 2015
- Primary completion date
- 17 Dec 2024
- Lead sponsor
- Biogen
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT02386553
- EudraCT number
- 2014-002098-12
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 25
- From 18 May 2015 to the primary completion date, 17 Dec 2024
- 21 sites in 7 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
- 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.
Other
- Hypoxemia (oxygen saturation \<96% awake or asleep without any supplemental oxygen or respiratory support).
- Clinically significant abnormalities in hematology or clinical chemistry parameters.
- Meet additional study related criteria.
- Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Who the study is looking for
- 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.
Age in the registry: 0 weeks to 6 weeks · Sex: All · Does not accept healthy volunteers
Sites (21)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Royal Children's Hospital Coordinator of this site? Confirm its status | Parkville | Australia | Unknown status |
| Queensland Children's Hospital Coordinator of this site? Confirm its status | South Brisbane | Australia | Unknown status |
| Universitaetsklinikum Freiburg Coordinator of this site? Confirm its status | Freiburg im Breisgau | Germany | Unknown status |
| Fondazione Serena ONLUS centro clinico NEMO Coordinator of this site? Confirm its status | Milan | Italy | Unknown status |
| Ospedale Pediatrico Bambino Gesù Coordinator of this site? Confirm its status | Rome | Italy | Unknown status |
| Hamad General Hospital Coordinator of this site? Confirm its status | Doha | Qatar | Unknown status |
| Kaohsiung Medical University Chung-Ho Memorial Hospital Coordinator of this site? Confirm its status | Kaohsiung City | Taiwan | Unknown status |
| National Taiwan University Hospital Coordinator of this site? Confirm its status | Taipei | Taiwan | Unknown status |
| Children's Hospital Colorado Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| The Johns Hopkins Hospital Coordinator of this site? Confirm its status | Baltimore | United States | Unknown status |
| Massachusetts General Hospital Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Ann & Robert H. Lurie Children's Hospital of Chicago Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| David Geffen School of Medicine Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Columbia University Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Nemours Children's Hospital - Orlando Coordinator of this site? Confirm its status | Orlando | United States | Unknown status |
| The Children's Hospital Of Philadelphia Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| University of California Davis Health System Coordinator of this site? Confirm its status | Sacramento | United States | Unknown status |
| University of Utah Coordinator of this site? Confirm its status | Salt Lake City | United States | Unknown status |
| Seattle Children's Research Institute Coordinator of this site? Confirm its status | Seattle | United States | Unknown status |
| Hacettepe University Medical Faculty Coordinator of this site? Confirm its status | Ankara | Turkey (Türkiye) | Unknown status |
| Yeditepe University Medical School Hospital Coordinator of this site? Confirm its status | Istanbul | Turkey (Türkiye) | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.