A Study to Assess the Efficacy and Safety of IONIS-SMN Rx in Patients With Later-onset Spinal Muscular Atrophy


This study will test the Clinical Efficacy and Safety of ISIS-SMN Rx in patients with later-onset Spinal Muscular Atrophy.

Study Start Date

November 2014

Estimated Completion Date

June 2017


  • Drug: ISIS-SMN Rx
  • Drug: IONIS-SMN Rx
  • Procedure: Sham procedure


  • Orthopedics: Spine
  • Physician Assistant: Orthopedics

MeSH Terms

  • Atrophy
  • Spinal
  • Spinal Muscular Atrophy

Study ID

Isis Pharmaceuticals -- ISIS 396443-CS4



Trial ID


Study Type


Trial Phase

Phase 3

Enrollment Quota



Isis Pharmaceuticals

Inclusion Criteria

  • Parent or guardian has signed informed consent and, if indicated per subject's age and institutional guidelines, subject 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 2 to 12 years of age at screening
  • 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

Exclusion Criteria

  • 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 MRI or CT that would interfere with the LP procedures or CSF circulation
  • Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter
  • History of bacterial meningitis
  • Dosing with ISIS-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 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.




2 Years to 12 Years

Accepts Healthy Volunteers


Study Locations and Contact Information (11)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles None None None
Columbia University Medical Center - New York, New York 181.2 miles None None None
Childrens Hospital of Philadelphia - Philadelphia, Pennsylvania 270.8 miles None None None
Ann and Robert H Lurie Childrens Hospital of Chicago - Chicago, Illinois 847.9 miles None None None
St Louis Childrens Hospital - St. Louis, Missouri 1,039.5 miles None None None
Nemours Childrens Hospital - Orlando, Florida 1,122.7 miles None None None
UT Southwestern Medical CenterChildrens Medical Center Dallas - Dallas, Texas 1,551.5 miles None None None
Childrens Hospital Colorado - Aurora, Colorado 1,759.8 miles None None None
Oregon Health and Science University - Portland, Oregon 2,538.3 miles None None None
UCLA Medical Center - Los Angeles, California 2,605.2 miles None None None
Stanford University Medical Center - Stanford, California 2,694.5 miles None None None

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