NCT02865109

Brief Summary

To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
3 countries

7 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 10, 2016

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 12, 2016

Completed
Last Updated

April 5, 2021

Status Verified

March 1, 2021

First QC Date

August 10, 2016

Last Update Submit

April 1, 2021

Conditions

Infantile-onset Spinal Muscular Atrophy

Keywords

Spinal Muscular AtrophySMASMNSMNRxISIS-SMNRxISIS-SMN RxISIS 396443ISIS-396443ENDEARIONIS-SMNRxIONIS-SMN RxEMBRACENURTUREIONISBIOGENSHINENusinersen

Interventions

NusinersenDRUG

Administered by intrathecal injection

Also known as: ISIS 396443, BIIB058, Spinraza

Eligibility Criteria

Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Genetic documentation of 5q SMA homozygous gene deletion, homozygous mutation, or compound heterozygote.
  • Onset of clinical signs and symptoms at ≤ 6 months (180 days) of age, consistent with infantile onset, Type I SMA
  • Patient whose care in the opinion of the treating physician meets, and is expected to continue to meet, the guidelines set out in the 2007 Consensus Statement for Standard of Care in SMA

You may not qualify if:

  • Patient is qualified to participate in an ongoing clinical trial with nusinersen
  • Participation in a prior nusinersen study
  • Previous exposure to nusinersen
  • History of brain or spinal cord disease that would interfere with the LP procedures or CSF circulation
  • Presence of implanted shunt for the drainage of CSF or implanted CNS catheter
  • Previous or current participation in a clinical trial with an investigational gene therapy for SMA
  • Participation in a study with an investigational therapy for SMA within 6 months or five half-lives of the investigational drug, whichever is the longer, prior to the first dose of nusinersen.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Hospital Pablo Tobon Uribe

Medellín, 050036, Colombia

Location

Auckland City Hospital

Grafton, Auckland, 1023, New Zealand

Location

Auckland District Health Board ADHB

Grafton, Auckland, 1023, New Zealand

Location

Erciyes University Hospital

Kayseri, Anatolia, 38000, Turkey (Türkiye)

Location

Hacettepe University

Ankara, Central Anatolia, 06100, Turkey (Türkiye)

Location

Marmara Uni. Research & Educational Hospital

Kadıköy, Istanbul, 34722, Turkey (Türkiye)

Location

Medipol University Hospital

Istanbul, Marmara, 34214, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

nusinersen

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Medical Director

    Biogen

    STUDY DIRECTOR

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 10, 2016

First Posted

August 12, 2016

Last Updated

April 5, 2021

Record last verified: 2021-03

Locations

CO(1)NZ(2)TU(4)