A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)

Completed · Phase 2

Conditions studied: Spinal Muscular Atrophy

In brief

The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA). The secondary objectives are to examine the safety and tolerability of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA and to examine the cerebral spinal fluid (CSF) and plasma Pharmacokinetics (PK) of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA.

Key facts

Study ID

NCT01839656

Run by

Biogen

People needed

21

Starts

2013-05-08

Expected to finish

2017-08-21

Last updated by the study team

2021-02-17

Who can join

Age: 0 and older, up to 1. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Hypoxemia (O2 saturation awake <96% or O2 saturation asleep <96%, without ventilation support)
• Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the screening period
• History of brain or spinal cord disease that would interfere with the lumbar puncture (LP) procedures, CSF circulation, or safety assessments
• Presence of an implanted shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter
• History of bacterial meningitis
• Clinically significant abnormalities in hematology or clinical chemistry parameters, as assessed by the Site Investigator, at screening that would render the participant unsuitable for inclusion
• Treatment with another investigational drug (e.g., albuterol, riluzole, carnitine, creatine, sodium phenylbutyrate, salbutamol, valproate, hydroxyurea etc), biological agent, or device within 90 days prior to enrollment or anytime during the study. Any history of gene therapy or cell transplantation
• The participants parent(s) or legal guardian(s) is unable to understand the nature, scope, and possible consequences of the study, or does not agree to comply with the protocol defined schedule of assessments
• Ongoing medical condition that according to the Site Investigator would interfere with the conduct and assessments of the study. Examples are medical disability other than SMA that would interfere with the assessment of safety or would compromise the ability of the participant to undergo study procedures
• NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Where it is running

• Stanford University Medical Center — Stanford, California, United States
• Nemours Children's Hospital — Orlando, Florida, United States
• Columbia University Medical Center — New York, New York, United States
• The Hospital for Sick Children (SickKids) — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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