Clinical Trials

Overview

Onasemnogene abeparvovec-xioi (Zolgensma™)

Novartis Gene Therapies

Onasemnogene abeparvovec-xioi, previously AVXS-101 ( Zolgensma™) is a gene therapy developed by Novartis Gene Therapies (previously AveXis), designed to address the genetic root cause of SMA by replacing the function of the missing or faulty SMN1 gene.

Administered during a single, intravenous (IV) infusion, Zolgensma delivers a new working copy of the SMN1 gene into a patient’s cells, halting disease progression.

Method of Action

Zolgensma™ is made up of a new, working copy of a human SMN gene that is placed inside a vector. This vector transports the new, working SMN gene to the motor neuron cells...

Zolgensma™ treatment

In Focus

The Vector

The vector that delivers the SMN gene is made from a virus called adeno-associated virus 9, or AAV9. This type of virus is harmless. To make the vector, the DNA of the virus is removed and the new SMN gene inserted in its place. Vectors are used because they can travel throughout the body and deliver the new, working gene to the cells where it is needed.

When the new gene reaches its destination, it is ready to tell the motor neurons to start making SMN protein. This happens throughout the body, with many vectors delivering a new, working copy of the SMN gene to motor neurons.

Motor neurons now make sufficient SMN protein to survive, function, and be maintained.

More about Zolgensma™

Trials of onasemnogene abeparvovec-xioi/ AVXS-101

Trial Name
STEER
Age:
2 to 17 years old
Aim:
To evaluate the efficacy and safety of intrathecal OAV101 in Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years old, treatment naive, sitting, and never ambulatory
Status:
Not yet recruiting
Locations:
Identifier:
NCT05089656
Trial Name
SMART
Age:
Up to 17 years old
Aim:
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 in people with SMA with bi-allelic mutations in the SMN1 gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.
Status:
Recruiting
Locations:
Belgium, France, Portugal, Australia & Canada.
Identifier:
NCT04089566
Trial Name
START
Age:
Child, Adult, Older Adult
Aim:
A long term follow-up safety study of people in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering AVXS-101
Status:
Active, not recruiting
Locations:
USA
Identifier:
NCT03421977
Trial Name
STRONG
Age:
Children6 to 60 months old
Aim:
To evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with SMA with 3 copies of SMN2 and deletion of SMN1.
Status:
Suspended
Locations:
USA
Identifier:
NCT03381729
Trial Name
STR1VE
Age:
Up to 180 days old
Aim:
Phase 3, open-label, single-arm, single-dose gene replacement therapy clinical trial for infants with SMA Type 1 with one or two SMN2 copies delivering AVXS-101 by intravenous infusion
Status:
Completed
Locations:
USA
Identifier:
NCT03306277
Trial Name
SPRINT
Age:
Up to 42 days
Aim:
A global study of a single, one-time dose of AVXS-101 delivered to infants with genetically diagnosed and pre-symptomatic SMA with multiple copies of SMN2
Status:
Completed
Locations:
Belgium, Germany,Italy, Spain, UK, Israel, Australia, Canada, Japan, Korea, Taiwan, USA
Identifier:
NCT03505099

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