- 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:
- Active, not recruiting
- Locations:
- Denmark, China, India, Malaysia, Mexico, Saudi Arabia, Singapore, South Africa, Taiwan, Thailand, USA, Vietnam
- Identifier:
- NCT05089656
Clinical Trials
Overview
OAV101
OAV-101 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 genetically engineered and harmless adeno-associated virus serotype 9 (AAV9) 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.
Trial of OAV101
- Trial Name
- SPECTRUM
- Status:
- Recruiting
- Aim:
- To assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial.
- Participation criteria:
- Participants must have participated in an OAV101 clinical trial.
- Locations:
- Australia, Belgium, Canada, China, Denmark, France, Italy, Japan, Malaysia, Singapore, Spain, Taiwan, Thailand, UK, USA
- Identifier:
- NCT05335876