MedTrace Logo

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

NCT02122952 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2022-09-15

Study results available
· View outcomes & findings →

Summary

The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1).

Conditions

  • Spinal Muscular Atrophy 1

Interventions

BIOLOGICAL

AVXS-101

Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter

Sponsors & Collaborators

  • Novartis Gene Therapies

    lead INDUSTRY

Principal Investigators

  • Jerry R Mendell, MD · The Research Institute at Nationwide Children's Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-05-05
Primary Completion
2017-12-15
Completion
2017-12-15

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02122952 on ClinicalTrials.gov