Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

PHASE3CompletedINTERVENTIONAL
Enrollment

127

Participants

Timeline

Start Date

February 1, 2022

Primary Completion Date

November 12, 2024

Study Completion Date

April 29, 2025

Conditions
Type 2 Spinal Muscular Atrophy
Interventions
GENETIC

OAV101

Gene therapy

PROCEDURE

Sham control

The sham procedure will consist of a small needle prick on the lower back at the location where the LP injection is normally made. The needle will break the skin, but no needle insertion for lumbar puncture will occur.

Trial Locations (31)

7925

Red Cross War Memorial Childrens Hospital, Cape Town

10700

Siriraj Hospital, Bangkok

11211

Novartis Investigative Site, Riyadh

17579

Clinic for Special Children, Strasburg

23507

Children's Specialty Group/CHKD, Norfolk

38105

St Jude Children's Research Hospital, Memphis

44280

Hospital Civil De Guadalajara Fray Antonio Alcalde, Guadalajara

50300

Novartis Investigative Site, Kuala Lumpur

59100

Novartis Investigative Site, Kuala Lumpur

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

80756

Kaohsiung Medical University Hospital, Kaohsiung City

100000

National Children's Hospital, Hanoi

100034

Peking University First Hospital, Beijing

100069

Novartis Investigative Site, Beijing

100730

Novartis Investigative Site, Beijing

110029

AIIMS, Ansari Nagar, New Delhi

119074

Novartis Investigative Site, Singapore

310052

Novartis Investigative Site, Hangzhou

400010

Novartis Investigative Site, Chongqing

400016

P.D. Hinduja National Hospital & MRC, Mumbai

500034

Novartis Investigative Site, Hyderabad

510623

Novartis Investigative Site, Guangzhou

518034

Novartis Investigative Site, Shenzhen

610041

Novartis Investigative Site, Chengdu

700094

Novartis Investigative Site, Kolkata

06032

Connecticut Children's Medical Center, Farmington

81520-060

Novartis Investigative Site, Curitiba

05403 000

Novartis Investigative Site, São Paulo

2100 O

Paediatric Neurology, Copenhagen

110 060

Sir Ganga Ram Hospital, New Delhi

06720

Novartis Investigative Site, Mexico City

Sponsors
All Listed Sponsors
lead

Novartis Pharmaceuticals

INDUSTRY