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Clinical Trials/DRKS00011547
DRKS00011547
Completed
N/A

Expanded Access Program to provide Nusinersen to Patients with infantile-onset spinal muscular atrophy (SMA)

Klinik für Neuropädiatrie und MuskelerkrankungenZentrum für Kinder- und JugendmedizinUniversitätsklinikum Freiburg0 sites61 target enrollmentJanuary 11, 2017
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ConditionsG12.0Infantile spinal muscular atrophy, type I [Werdnig-Hoffman]

Overview

Phase
N/A
Intervention
Not specified
Conditions
G12.0
Sponsor
Klinik für Neuropädiatrie und MuskelerkrankungenZentrum für Kinder- und JugendmedizinUniversitätsklinikum Freiburg
Enrollment
61
Status
Completed
Last Updated
last year
OverviewInvestigatorsEligibility CriteriaOutcomesSimilar Trials

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
January 11, 2017
End Date
July 18, 2020
Last Updated
last year
Study Type
Observational
Sex
All

Investigators

Sponsor
Klinik für Neuropädiatrie und MuskelerkrankungenZentrum für Kinder- und JugendmedizinUniversitätsklinikum Freiburg

Eligibility Criteria

Inclusion Criteria

  • •Patients must meet all of the following criteria to be eligible:
  • •\- 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
  • •\- Treatment with Nusinersen within the EAP
  • •\- Informed consent for the study

Exclusion Criteria

  • •\- Patient qualifies 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.

Outcomes

Primary Outcomes

Not specified

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