Isis Begins Recruiting for Phase III Trial

Isis is currently recruiting participants for Endear, a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA). The goal of this Phase III trial is to assess the clinical efficacy and safety of ISIS-SMNRx administered intrathecally (through an injection in the lower back, similar to an epidural) in patients with SMA. This study is being conducted by Isis Pharmaceuticals, Inc.

ISIS-SMNRx, an investigational medication being evaluated in these studies, is an antisense oligonucleotide drug designed to increase the amount of normal SMN protein in patients with SMA. Researchers believe that if the babies produce more of this protein, they will show improvement in symptoms of SMA and possibly survive longer or have a longer time until they need a ventilator to help them breathe. To date, it has been studied in children and infants with SMA and was generally well-tolerated.

From 2003 to 2006, Cure SMA provided the seed funding needed to begin investigation into this therapeutic approach.

Several Cure SMA grants totaling over $500,000 were given to Dr. Ravi Singh at U Massachusetts, to help develop the intellectual property needed to investigate antisense oligonucleotides for SMA. The intellectual property generated with our funding was then licensed to Isis Pharmaceuticals to create ISIS-SMNRx.

To be considered for the infantile-onset SMA study, the infants must be ≤7 months (210 days) of age at screening with onset of SMA signs and symptoms at ≤6 months of age, with SMN2 copy number of 2. The study subjects must live within 9 hours ground travel of an investigative site.

For futher information:

Trial Sites

Sites in California, Florida, Illinois, Massachusetts, and New York have begun recruiting. All other sites are not yet recruiting.

United States, California

UCLA Medical Center
Los Angeles, California, United States, 90095
Contact: Katrina Nguyen – 310.825.3264, [email protected]
Principal Investigator: Perry Shieh, MD

Stanford University Medical Center
Stanford, California, United States, 94305
Contact: Shirley Paulose, MBBS, MS – 650.724.3792, [email protected]
Principal Investigator: John Day, MD

United States, Colorado

Children’s Hospital Colorado
Aurora, Colorado, United States, 80045
Contact: Melissa Gibbons – 720.777.3697, [email protected]
Principal Investigator: Julie Parsons, MD

United States, Florida

Nemours Children’s Hospital
Orlando, Florida, United States, 32827
Contact: Dawn Cook, RN, MSN, CCRC – 407.202.2962, [email protected]
Principal Investigator: Richard Finkel, MD

United States, Illinois

Ann and Robert H. Lurie Children’s Hospital of Chicago
Chicago, Illinois, United States, 60611
Contact: Lauren Webb – 312.227.4483, [email protected]
Principal Investigator: Nancy L. Kuntz, MD

United States, Massachusetts

Boston Children’s Hospital
Boston, Massachusetts, United States, 02115
Contact: Heather Szelag – 617.355.2752, [email protected]
Principal Investigator: Basil T. Darras, MD

United States, Missouri

St. Louis Children’s Hospital
St. Louis, Missouri, United States, 63110
Contact: Pallavi Anand – 314.362.2490, [email protected]
Principal Investigator: Anne Connolly, MD

United States, New York

Columbia University Medical Center
New York, New York, United States, 10032
Contact: Rosangel Cruz, MA, BS – 212.305.1336, [email protected]
Principal Investigator: Claudia Chiriboga, MD

United States, North Carolina

Duke Children’s Hospital
Durham, North Carolina, United States, 27710
Principal Investigator: Loren Del Mar Pena, MD, PhD

United States, Oregon

Oregon Health and Science University
Portland, Oregon, United States, 97239
Contact: Bryn McCarthy – 503.418.8297, [email protected]
Principal Investigator: Erika Finanger, MD

United States, Pennsylvania

Children’s Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Contact: Michele Toms, RN – 215.590.7727, [email protected]
Principal Investigator: Gihan Tennekoon, MD

United States, Texas

UT Southwestern Medical Center/Children’s Medical Center Dallas
Dallas, Texas, United States, 75235
Contact: Stephanie Trest – 214.456.3686, [email protected]
Principal Investigator: Susan Iannaccone, MD

Australia, New South Wales

Sydney Children’s Hospital 
Sydney, New South Wales, Australia, 2031
Contact: Nisha Berthon-Jones – +61 2 9382 1757, [email protected]
Principal Investigator: Hugo Sampaio, MD

Australia, Victoria

Royal Children’s Hospital, Children’s Neuroscience Centre
Parkville, Victoria, Australia, 3052
Contact: Daniella Villano – +61 2 9345 4633, [email protected]
Principal Investigator: Monique Ryan, MD

Canada, British Columbia

British Columbia Children’s Hospital/UBC
Vancouver, British Columbia, Canada, V6H 3V4
Contact: Vesna Popovska, MD – 604.875.2345 x5157, [email protected]
Principal Investigator: Kathryn Selby, MD

Canada, Ontario

Hospital for Sick Children
Toronto, Ontario, Canada, M5G 0A4
Contact: Lynn MacMillan, RN, CNN(c), MSCN – 416.813.7355, [email protected]
Principal Investigator: Jiri Vajsar, MD

Canada, Quebec

The Research Institut of the McGill University Health Centre
Montreal, Quebec, Canada, H3H 1P3
Contact: Valerie Gagne, TM – 514.934.1934 x23534, [email protected]
Principal Investigator: Maryam Oskoui, MD


Groupe Hospitalier Pitie-Salpetriere
Paris, France
Contact: Laurent Servais, MD – +33 1 42 16 58 70, [email protected]
Principal Investigator: Laurent Servais, MD


Universitatsklinikum Essen 
Essen, Germany
Principal Investigator: Ulrike Schara, MD

Universtatsklinikum Freiburg, Zentrum fur Kinder-und Jugendmedizin
Freiburg, Germany
Principal Investigator: Jan Kirschner, MD

Hong Kong

The University of Hong Kong, Queen Mary Hospital, Department of Paediatrics and Adolescent Medicine
Hong Kong, Hong Kong SAR, Hong Kong
Contact: Amanda Mok – +852 22554538, [email protected]
Principal Investigator: Sophelia Chan, MD


Istituto Giannina Gaslini, Centro Traslazionale di Miologia e Patologie Neurodegenerative
Genova, Italy
Principal Investigator: Claudio Bruno, MD

Pediatric Neurology Unit, Catholic University
Rome, Italy
Contact: Marika Pane, MD – 0039 0630155340, [email protected]
Principal Investigator: Eugenio Mercuri, MD


Tokyo Women’s Medical University
Tokyo, Tokyo Metropolitan Prefecture, Japan
Contact: Aiko Umeno – 81.3.3353.8111, [email protected]
Principal Investigator: Kayoko Saito, MD

Korea, Republic of

Seoul National University Hospital
Seoul, Korea, Republic of
Contact: Jisu Hong – +82 2 2072 1652, [email protected]
Principal Investigator: Jong-Hee Chae, MD


Hospital Valle Hebron
Barcelona, Spain
Contact: Eduardo Tizzano, MD – +34 (0) 935537369, [email protected]
Principal Investigator: Eduardo Tizzano, MD

Hospital Universitario La Paz, Pediatric Neurology Department
Madrid, Spain
Contact: Samuel P Pascual, MD – +34 (0) 91-7277388, [email protected]
Principal Investigator: Pascual Samuel Pascual, MD


University of Gothenburg, The Queen Silvia Children’s Hospital
Gothenburg, Sweden
Contact: Anna-Lena Gustafsson – +46-31-3436069, [email protected]
Principal Investigator: Mar Tulinius, MD


Kaohsiung Medical University Hospital
Kaohsiung City, Taiwan
Contact: Yun-Hui Chou – +886 972 977 320, [email protected]
Principal Investigator: Yuh-Jyh Jong, MD

United Kingdom

UCL Institute of Child Health/Great Ormond Street
London, United Kingdom, WC1N 1EH
Contact: Isabelle Wilson – +44 (0) 20 7905 2151 x2151, [email protected]
Principal Investigator: Francesco Muntoni, MD

MRC Centre for Neuromuscular Diseases at Newcastle, Institute of Genetic Medicine Newcastle University
Newcastle, United Kingdom, NE1 3BZ
Contact: Becky Davis – +44 (0) 191 241 8649, [email protected]
Principal Investigator: Straub Volker, MD

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