Clinical Trials

Overview

OAV101

OAV-101 IT is a version of Zolgensma that is administered intrathecally, or via injection into the spinal canal, rather than into the bloodstream.

It is an investigational gene therapy developed by Novartis Gene Therapies.

Method of Action

Like Zolgensma, a vector is used to deliver a working copy of the SMN1 gene into cells (but it does not integrate into the genome), allowing motor neurons and other cells to produce functional SMN protein.

Trials of OAV101

Trial Name
STRENGTH
Age:
2 to 17 years old.
Aim:
To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam.
Status:
Completed
Locations:
Australia, Belgium, Canada, France, Italy, Japan, Netherlands, Spain.
Identifier:
NCT05386680
Trial Name
STEER
Age:
2 to 17 years old.
Aim:
To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment naive patients with Type 2 SMA.
Status:
Completed
Locations:
Brasil, China, Denmark, India, Malaysia, Mexico, Saudi Arabia, Singapore, South Africa, Taiwan, Thailand, USA, Vietnam
Identifier:
NCT05089656
Trial Name
SPECTRUM
Age:
Children and adults. Participants must have participated in an OAV101 (IV or IT) clinical trial.
Aim:
To assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial.
Status:
Recruiting
Locations:
Australia, Belgium, Canada, China, Denmark, France, Italy, Japan, Malaysia, Netherlands, Saudi Arabia, Singapore, Spain, Taiwan, Thailand, UK, USA, Vietnam.
Identifier:
NCT05335876
Trial Name
STRONG
Age:
6 months to 5 years 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:
Terminated
Locations:
USA
Identifier:
NCT03381729

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