Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Biogen
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc.
In August 2016, sponsorship of the trial was transferred to Biogen.
Age
2–14
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Documented Survival Motor Neuron1 (SMN1) homozygous gene deletion
Clinical signs attributable to Spinal Muscular Atrophy (SMA)
Able to complete all study procedures, measurements and visits and parent/participant has adequately supportive psychosocial circumstances, in the opinion of the investigator
Estimated life expectancy \> 2 years from Screening
Meets age-appropriate institutional criteria for use of anesthesia/sedation, if use is planned for study procedure
You may not be if
Respiratory insufficiency defined by the need for invasive or non-invasive ventilation during a 24 hour period
Presence of a gastric feeding tube
Previous scoliosis surgery or scoliosis surgery planned during the duration of the study that would interfere with the lumbar puncture (LP) injection procedure
Hospitalization for surgery or pulmonary event within the last 2 months or planned during the study
Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy
History of brain or spinal cord disease that would interfere with LP procedures or cerebrospinal fluid (CSF) circulation
Presence of an implanted shunt for the draining of CSF or an implanted Central Nervous System (CNS) catheter
History of bacterial meningitis
Clinically significant abnormalities in hematology or clinical chemistry parameters
Treatment with another investigational drug, biological agent, or device within 1-month of Screening or 5 half-lives of study agent whichever is longer. Any history of gene therapy or cell transplantation
Ongoing medical condition that would interfere with the conduct and assessments of the study. Examples are medical disability (e.g. wasting or cachexia, severe anemia, etc.) that would interfere with the assessment of safety or would compromised the ability of the participant to undergo study procedures
Clareo Health | An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
