Status:
COMPLETED
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
Lead Sponsor:
Biogen
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
2-12 years
Phase:
PHASE3
Brief Summary
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secon...
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
- Parent or guardian has signed informed consent and, if indicated per participant's age and institutional guidelines, participant has signed informed assent
- Be medically diagnosed with Spinal Muscular Atrophy (SMA)
- Have onset of clinical signs and symptoms consistent with SMA at greater than 6 months of age
- Be able to sit independently, but has never had the ability to walk independently
- Have Motor Function Score (Hammersmith Functional Motor Scale - Expanded) greater than or equal to 10 and less than or equal to 54 at Screening
- Be able to complete all study procedures, measurements and visits and parent or guardian and subject has adequately supportive psychosocial circumstances, in the opinion of the Investigator
- Have an estimated life expectancy of greater than 2 years from Screening, in the opinion of the Investigator
- Meet age-appropriate institutional criteria for use of anesthesia and sedation, if use is planned for study procedures
- For subjects who have reached reproductive maturity, satisfy study contraceptive requirements
- Key
Exclusion
- Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for greater than 6 hours during a 24 hour period, at Screening
- Medical necessity for a gastric feeding tube, where the majority of feeds are given by this route, as assessed by the Site Investigator
- Severe contractures or severe scoliosis evident on X-ray examination at Screening
- Hospitalization for surgery (i.e., scoliosis surgery, other surgery), pulmonary event, or nutritional support within 2 months of Screening or planned during the duration of the study
- 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, including tumors, or abnormalities by magnetic resonance imaging (MRI) or computed tomography (CT) that would interfere with the LP procedures or cerebrospinal fluid (CSF) circulation
- Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
- History of bacterial meningitis
- Dosing with IONIS-SMN Rx in any previous clinical study
- Prior injury (e.g., upper or lower limb fracture) or surgical procedure which impacts the subject's ability to perform any of the outcome measure testing required in the protocol and from which the subject has not fully recovered or achieved a stable baseline
- 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 subject unsuitable for inclusion
- Treatment with another investigational drug (e.g., oral albuterol or salbutamol, riluzole, carnitine, creatine, sodium phenylbutyrate, et.c), biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer. Treatment with valproate or hydroxyurea within 3-months of Screening. Any history of gene therapy, antisense oligonucleotide therapy, or cell transplantation.
- Ongoing medical condition that according to the Site Investigator 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 compromise the ability of the subject to undergo study procedures.
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Key Trial Info
Start Date :
November 24 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 20 2017
Estimated Enrollment :
126 Patients enrolled
Trial Details
Trial ID
NCT02292537
Start Date
November 24 2014
End Date
February 20 2017
Last Update
February 17 2021
Active Locations (24)
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1
UCLA Clinical and Translational Research Center
Los Angeles, California, United States, 90095
2
Lucile Packard Children's Hospital at Stanford
Palo Alto, California, United States, 94304
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
4
Nemours Children's Hospital
Orlando, Florida, United States, 32827