Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

PHASE3RecruitingINTERVENTIONAL
Enrollment

175

Participants

Timeline

Start Date

December 19, 2022

Primary Completion Date

June 26, 2030

Study Completion Date

June 26, 2030

Conditions
Spinal Muscular Atrophy (SMA)
Interventions
BIOLOGICAL

onasemnogene abeparvovec

Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.

Trial Locations (29)

2031

RECRUITING

Novartis Investigative Site, Randwick

3000

RECRUITING

Novartis Investigative Site, Leuven

10002

RECRUITING

Novartis Investigative Site, Taipei

10700

RECRUITING

Novartis Investigative Site, Bangkok

11211

RECRUITING

Novartis Investigative Site, Riyadh

23507

RECRUITING

Child Hosp Of The Kings Daughters, Norfolk

31059

RECRUITING

Novartis Investigative Site, Toulouse

50300

RECRUITING

Novartis Investigative Site, Kuala Lumpur

59100

RECRUITING

Novartis Investigative Site, Kuala Lumpur

67000

RECRUITING

Novartis Investigative Site, Strasbourg

69677

RECRUITING

Novartis Investigative Site, Bron

80756

RECRUITING

Novartis Investigative Site, Kaohsiung City

92380

RECRUITING

Novartis Investigative Site, Garches

100000

RECRUITING

Novartis Investigative Site, Hanoi

100034

RECRUITING

Novartis Investigative Site, Beijing

100069

RECRUITING

Novartis Investigative Site, Beijing

119074

RECRUITING

Novartis Investigative Site, Singapore

400010

RECRUITING

Novartis Investigative Site, Chongqing

510623

RECRUITING

Novartis Investigative Site, Guangzhou

610041

RECRUITING

Novartis Investigative Site, Chengdu

H4A 3J1

RECRUITING

Novartis Investigative Site, Montreal

2100 O

RECRUITING

Novartis Investigative Site, Copenhagen

00168

RECRUITING

Novartis Investigative Site, Roma

830-0011

RECRUITING

Novartis Investigative Site, Kurume

162 8666

RECRUITING

Novartis Investigative Site, Shinjuku Ku

3584CX

RECRUITING

Novartis Investigative Site, Utrecht

08035

RECRUITING

Novartis Investigative Site, Barcelona

WC1N 3JH

RECRUITING

Novartis Investigative Site, London

NE1 4LP

RECRUITING

Novartis Investigative Site, Newcastle upon Tyne

Sponsors
All Listed Sponsors
lead

Novartis Pharmaceuticals

INDUSTRY