Summary

Location
at UCLA
Dates
study started
estimated completion

Description

Summary

The primary objective is to evaluate the 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.

Official Title

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443

Details

This study was initiated and the protocol was registered by Ionis Pharmaceuticals, Inc. In August 2016, Biogen assumed responsibility for this study.

Keywords

Spinal Muscular Atrophy SMA SMN SMNRx ISIS-SMNRx ISIS 396443 SHINE IONIS-SMNRx IONIS-SMN Rx Spinraza nusinersen Muscular Atrophy Muscular Atrophy, Spinal Atrophy

Eligibility

You can join if…

  • Signed informed consent of parent or guardian and signed informed assent of participant, if indicated per participant's age and institutional guidelines.
  • Completion of the index study in accordance with the study protocol or as a result of Sponsor decision (e.g., early termination of the index study) within the preceding 16 weeks

You CAN'T join if...

  • Have any condition or worsening condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study
  • 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 participant unsuitable for participation in the study
  • Participant's parent or legal guardian is not willing or able to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study
  • Treatment with another investigational agent, biological agent, or device within one month of Screening, or 5 half-lives of study agent, whichever is longer

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

Locations

  • Research site
    Los Angeles California 90095 United States
  • Research site
    Palo Alto California 94305 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Biogen
Links
Cure SMA Muscular Dystrophy Association National Organization for Rare Diseases
ID
NCT02594124
Phase
Phase 3
Study Type
Interventional
Last Updated