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



Status:Recruiting
Conditions:Neurology, Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:Any
Updated:4/21/2016
Start Date:July 2014
End Date:July 2017

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A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy

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

This is a Phase 3 multicenter, double-blind, randomized, sham-procedure controlled study of
IONIS-SMN Rx in patients with infantile-onset SMA. Subjects will be randomized 2:1 to
receive IONIS-SMN Rx or a sham procedure control, respectively.

Inclusion Criteria:

- Be 7 months (210 days) of age or younger at screening

- Be born (gestational age) between 37 and 42 weeks

- Be medically diagnosed with spinal muscular atrophy (SMA)

- Have SMN2 Copy # of 2

- Body weight equal to or greater than 3rd percentile for age using appropriate
country-specific guidelines

- Be able to follow all study procedures

- Reside within 9 hours ground travel each way, for the duration of the study

- Meet additional study-related criteria

Exclusion Criteria:

- Hypoxemia (O2 saturation awake less than 96% or O2 saturation asleep less than 96%,
without ventilation support) during screening evaluation

- Meet additional study-related criteria
We found this trial at
14
sites
Durham, North Carolina 27710
Principal Investigator: Loren Del Mar Pena, MD, PhD
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Gihan Tennekoon, MD
Phone: 267-426-7161
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Principal Investigator: Erika Finanger, MD
Phone: 503-418-8297
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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Portland, OR
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Principal Investigator: Julie Parsons, MD
Phone: 720-777-1319
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Aurora, CO
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Basil T. Darras, MD
Phone: 617-355-2752
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Boston, MA
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Dallas, Texas 75235
Principal Investigator: Susan Iannaccone, MD
Phone: 214-456-4426
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Dallas, TX
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757 Westwood Plaza
Los Angeles, California 90024
(310) 825-9111
Principal Investigator: Perry Shieh, MD
Phone: 310-825-3264
UCLA Medical Center Founded in 1955, UCLA Medical Center became Ronald Reagan UCLA Medical Center...
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Los Angeles, CA
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630 W 168th St
New York, New York
212-305-2862
Principal Investigator: Claudia Chiriboga, MD
Phone: 212-305-1336
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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New York, NY
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13535 Nemours Parkway
Orlando, Florida 32827
(407) 567-4000
Principal Investigator: Richard Finkel, MD
Phone: 407-567-6201
Nemours Children's Hospital Nemours Children's Hospital in Orlando brings pediatric specialty care never before offered...
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Orlando, FL
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Salt Lake City, Utah 84132
Principal Investigator: Nicholas E Johnson, MD
Phone: 801-585-9055
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St. Louis, Missouri 63108
Principal Investigator: Anne Connolly, MD
Phone: 314-362-2490
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St. Louis, MO
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300 Pasteur Dr
Stanford, California 94305
(650) 723-4000
Principal Investigator: John Day, MD
Phone: 650-724-3792
Stanford Univ Med Ctr The Medical Center is uniquely advantaged by its location on the...
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Sydney, New South Wales
Principal Investigator: Hugo Sampaio, MD
Phone: +61 2 9382 1757
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