Summary

Location
at UC Davis UCLA
Dates
study started
completion around
Principal Investigator
by Francy Shu (ucla)

Description

Summary

The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.

Official Title

An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Keywords

Spinal Muscular Atrophy, NURTURE, Muscular Atrophy, Atrophy, Nusinersen

Eligibility

You can join if…

  • Age ≤ 6 weeks at first dose.
  • Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation.
  • Genetic documentation of 2 or 3 copies of survival motor neuron 2 (SMN2).
  • Ulnar compound muscle action potential (CMAP) ≥ 1 mV at Baseline.
  • Gestational age of 37 to 42 weeks for singleton births; gestational age of 34 to 42 weeks for twins.
  • Meet additional study related criteria.

You CAN'T join if...

  • Hypoxemia (oxygen saturation <96% awake or asleep without any supplemental oxygen or respiratory support).
  • Any clinical signs or symptoms at Screening or immediately prior to the first dosing (Day 1) that are, in the opinion of the Investigator, strongly suggestive of SMA.
  • Clinically significant abnormalities in hematology or clinical chemistry parameters.
  • Treatment with an investigational drug given for the treatment of SMA biological agent, or device. Any history of gene therapy, prior antisense oligonucleotide (ASO) treatment, or cell transplantation.
  • Meet additional study related criteria.

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

Locations

  • David Geffen School of Medicine
    Los Angeles California 90095 United States
  • University of California Davis Health System
    Sacramento California 95817 United States

Lead Scientist at University of California Health

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Biogen
ID
NCT02386553
Phase
Phase 2 research study
Study Type
Interventional
Participants
About 25 people participating
Last Updated