A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies

Last updated: September 27, 2024
Sponsor: Biogen
Overall Status: Completed

Phase

3

Condition

Myasthenia Gravis (Chronic Weakness)

Spinal Muscular Atrophy

Muscular Dystrophy

Treatment

nusinersen

Clinical Study ID

NCT02594124
ISIS 396443-CS11
2015-001870-16
  • All Genders

Study Summary

The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Signed informed consent of parent or guardian and signed informed assent ofparticipant, if indicated per participant's age and institutional guidelines.

  • Completion of the index study in accordance with the study protocol or as a resultof Sponsor decision (e.g., early termination of the index study) within thepreceding 16 weeks

Exclusion

Key Exclusion Criteria:

  • Have any condition or worsening condition which in the opinion of the Investigatorwould make the participant unsuitable for enrollment, or could interfere with theparticipant participating in or completing the study

  • Clinically significant abnormalities in hematology or clinical chemistry parametersor electrocardiogram (ECG), as assessed by the Site Investigator, at the Screeningvisit that would render the participant unsuitable for participation in the study

  • Participant's parent or legal guardian is not willing or able to meet standard ofcare guidelines (including vaccinations and respiratory syncytial virus prophylaxisif available), nor provide nutritional and respiratory support throughout the study

  • Treatment with another investigational agent, biological agent, or device within onemonth of Screening, or 5 half-lives of study agent, whichever is longer

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 292
Treatment Group(s): 1
Primary Treatment: nusinersen
Phase: 3
Study Start date:
November 04, 2015
Estimated Completion Date:
August 21, 2023

Study Description

This study was initiated and the protocol was registered by Ionis Pharmaceuticals, Inc.

In August 2016, Biogen assumed responsibility for this study.

Connect with a study center

  • Research site

    Sydney, New South Wales 2031
    Australia

    Site Not Available

  • Sydney Children's Hospital Clinical Research Centre

    Sydney, New South Wales 2031
    Australia

    Site Not Available

  • Research site

    Parkville, Victoria 3052
    Australia

    Site Not Available

  • Royal Children's Hospital

    Parkville, Victoria 3052
    Australia

    Site Not Available

  • Research site

    Brussels, 1020
    Belgium

    Site Not Available

  • Universitair Kinderziekenhuis Koningin Fabiola

    Brussels, 1020
    Belgium

    Site Not Available

  • BC Children's Hospital / UBC

    Vancouver, British Columbia V6H 3V4
    Canada

    Site Not Available

  • Research site

    Vancouver, British Columbia V6H 3V4
    Canada

    Site Not Available

  • Children's Health Research Institute

    Brussel, Ontario N6A 5W9
    Canada

    Site Not Available

  • Research site

    London, Ontario N6A 5W9
    Canada

    Site Not Available

  • Hospital for Sick Children

    Toronto, Ontario M5G 0A4
    Canada

    Site Not Available

  • McGill University Health Centre

    Montreal, Quebec H4A 3J1
    Canada

    Site Not Available

  • Research site

    Montreal, Quebec H4A 3J1
    Canada

    Site Not Available

  • Armand Trousseau Hospital, I-Motion

    Paris, Paris 9 75012
    France

    Site Not Available

  • Research site

    Paris, 75012
    France

    Site Not Available

  • LMU-Campus Innenstadt

    Muenchen, Bayern 80337
    Germany

    Site Not Available

  • Research site

    Munich, Bayern 80337
    Germany

    Site Not Available

  • Research site

    Essen, 45147
    Germany

    Site Not Available

  • Universitatsklinikum Essen

    Essen, 45147
    Germany

    Site Not Available

  • Research site

    Freiburg, 79106
    Germany

    Site Not Available

  • Universitaetsklinikum Freiburg

    Freiburg, 79106
    Germany

    Site Not Available

  • Research site

    Hong Kong, Hong Kong SAR 999077
    Hong Kong

    Site Not Available

  • The University of Hong Kong

    Hong Kong, Hong Kong SAR 999077
    Hong Kong

    Site Not Available

  • Pediatric Neurology Unit, Catholic University

    Essen, 00168
    Italy

    Site Not Available

  • Istituto Giannina Gaslini, Centro Traslazionale di Miologia

    Genova, 16147
    Italy

    Site Not Available

  • Research site

    Genova, 16147
    Italy

    Site Not Available

  • Department of Neuroscience, Università di Messina, AOU Polic

    Messina, 98125
    Italy

    Site Not Available

  • Research site

    Messina, 98125
    Italy

    Site Not Available

  • Research site

    Milan, 20162
    Italy

    Site Not Available

  • Research site

    Rome, 00168
    Italy

    Site Not Available

  • Aichi Children's Health and Medical Center

    Obu, Aichi 474-0038
    Japan

    Site Not Available

  • Research site

    Obu, Aichi 474-0038
    Japan

    Site Not Available

  • Hyogo College of Medicine

    Nishinomiya, Hyogo 663-8501
    Japan

    Site Not Available

  • Research site

    Nishinomiya, Hyogo 663-8501
    Japan

    Site Not Available

  • Hyogo College of Medicine

    Nishinomya-shi, Hyogo
    Japan

    Site Not Available

  • Research site

    Shinjuku-ku, Tokyo 162-8666
    Japan

    Site Not Available

  • Tokyo Women's Medical University

    Shinjuku-ku, Tokyo 162-8666
    Japan

    Site Not Available

  • Kumamoto University Hospital

    Kumamoto, 860-8556
    Japan

    Site Not Available

  • Research site

    Kumamoto, 860-8556
    Japan

    Site Not Available

  • Miyagi Prefectural Children Hospital

    Miyagi, 989-3126
    Japan

    Site Not Available

  • Research site

    Miyazaki, 989-3126
    Japan

    Site Not Available

  • University of Miyazaki Hospital

    Miyazaki, 889-1692
    Japan

    Site Not Available

  • Research site

    Seoul, Korea 3080
    Korea, Republic of

    Site Not Available

  • Seoul National University Hospital

    Seoul, Korea 3080
    Korea, Republic of

    Site Not Available

  • Hospital Sant Joan de Deu

    Barcelona, 08950
    Spain

    Site Not Available

  • Research site

    Barcelona, 08950
    Spain

    Site Not Available

  • Hospital Universitario Vall de Hebron

    Hebron, 08035
    Spain

    Site Not Available

  • Research site

    Hebron, 08035
    Spain

    Site Not Available

  • Hospital Universitario La Paz

    Madrid, 28046
    Spain

    Site Not Available

  • Research site

    Madrid, 28046
    Spain

    Site Not Available

  • Research site

    Gothenburg, SE 416 86
    Sweden

    Site Not Available

  • The Queen Silvia Children's Hospital

    Gothenburg, SE 416 86
    Sweden

    Site Not Available

  • Kaohsiung Medical University Hospital

    Kaohsiung City, 807
    Taiwan

    Site Not Available

  • Research site

    Ankara, 6100
    Turkey

    Site Not Available

  • Uníversity of Hacettepe

    Ankara, 6100
    Turkey

    Site Not Available

  • Marmara University Pendik Training and Research Hospital

    Istanbul, 34662
    Turkey

    Site Not Available

  • Research site

    Istanbul, 34662
    Turkey

    Site Not Available

  • MRC Centre for Neuromuscular Diseases at Newcastle

    Newcastle, Northumberland NE1 3BZ
    United Kingdom

    Site Not Available

  • Research site

    London, WC1N 1EH
    United Kingdom

    Site Not Available

  • UCL Institute of Child Health

    London, WC1N 1EH
    United Kingdom

    Site Not Available

  • Research site

    Newcastle, NE1 3BZ
    United Kingdom

    Site Not Available

  • David Geffen School of Medicine at UCLA

    Los Angeles, California 90095-8344
    United States

    Site Not Available

  • Research site

    Los Angeles, California 90095
    United States

    Site Not Available

  • Research site

    Palo Alto, California 94305
    United States

    Site Not Available

  • Stanford University School of Medicine

    Palo Alto, California 94305
    United States

    Site Not Available

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Research site

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Connecticut Children's Medical Center

    Hartford, Connecticut 06106
    United States

    Site Not Available

  • Research site

    Hartford, Connecticut 06106
    United States

    Site Not Available

  • Nemours Children's Clinic

    Orlando, Florida 32827
    United States

    Site Not Available

  • Research site

    Orlando, Florida 32827
    United States

    Site Not Available

  • Research site

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    New York, Illinois 60611
    United States

    Site Not Available

  • Research Site

    Baltimore, Maryland 21287
    United States

    Site Not Available

  • The Johns Hopkins Hospital

    Baltimore, Maryland 21287
    United States

    Site Not Available

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Research site

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Research site

    Minneapolis, Minnesota 55101
    United States

    Site Not Available

  • Gillette Children's Specialty Healthcare

    Saint Paul, Minnesota 55101
    United States

    Site Not Available

  • Research site

    Saint Paul, Minnesota 55101
    United States

    Site Not Available

  • Research site

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Washington University School of Medicine

    Saint Louis, Missouri 63110
    United States

    Site Not Available

  • Washington University School of Medicine

    St. Louis, Missouri 63110
    United States

    Site Not Available

  • Columbia University Medical Center

    New York, New York 10032
    United States

    Site Not Available

  • Research site

    New York, New York 10032
    United States

    Site Not Available

  • Duke University School of Medicine

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Research site

    Durham, North Carolina 27710
    United States

    Site Not Available

  • Duke University School of Medicine

    Miyagi, North Carolina 27710
    United States

    Site Not Available

  • Oregon Health Sciences University

    Durham, Oregon 97239
    United States

    Site Not Available

  • Research site

    Portland, Oregon 97239
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • Research site

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • Children's Medical Center

    Dallas, Texas 75235
    United States

    Site Not Available

  • Research site

    Dallas, Texas 75235
    United States

    Site Not Available

  • University of Utah

    Obu, Aichi, Utah 84112
    United States

    Site Not Available

  • Research site

    Salt Lake City, Utah 84132
    United States

    Site Not Available

  • Research Site

    Seattle, Washington 98105
    United States

    Site Not Available

  • Seattle Children's Hospital

    Seattle, Washington 98105
    United States

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.