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Clinical Trials/EUCTR2014-002098-12-IT
EUCTR2014-002098-12-IT
Active, not recruiting
Not Applicable

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 - NURTURE

Biogen Idec Research Limited0 sites25 target enrollmentMarch 24, 2015
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ConditionsSpinal Muscular Atrophy (SMA)MedDRA version: 17.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Overview

Phase
Not Applicable
Intervention
Not specified
Conditions
Spinal Muscular Atrophy (SMA)
Sponsor
Biogen Idec Research Limited
Enrollment
25
Status
Active, not recruiting
Last Updated
10 years ago
OverviewInvestigatorsEligibility CriteriaOutcomesSimilar Trials

Overview

Brief Summary

No summary available.

Registry
who.int
Start Date
March 24, 2015
End Date
TBD
Last Updated
10 years ago
Study Type
Interventional clinical trial of medicinal product
Sex
All

Investigators

Eligibility Criteria

Inclusion Criteria

  • •\- 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\).
  • •\- 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.
  • •Are the trial subjects under 18? yes
  • •Number of subjects for this age range: 25
  • •F.1\.2 Adults (18\-64 years) no
  • •F.1\.2\.1 Number of subjects for this age range

Exclusion Criteria

  • •\- 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 dosing 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.

Outcomes

Primary Outcomes

Not specified

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