CI

At a glance

ClinicalIndex Comparison Record
Phase 1Completed· 15 enrolled
Drug / intervention
AVXS-101biological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT02122952
NCT02122952Phase 1Completed
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Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101

Novartis Gene Therapies·interventional·Posted Apr 25, 2014·Updated Sep 15, 2022

In Brief

A Phase 1 clinical trial evaluating AVXS-101 for Spinal Muscular Atrophy 1. Completed, enrolled 15 participants across 1 site.

Detailed 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).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy 1
CountriesUnited States
Collaborators--

Timeline

Phase 1CompletedFinished
2014201520162017201820192020202120222023202420252026
First PostedApr 25, 2014
Enrollment StartMay 5, 2014
Primary CompletionDec 15, 2017
TodayJul 2, 2026
Enrollment to primary: 3.6 yearsPosted 12.2 years ago

Interventions

AVXS-101biological

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