One-Shot gene therapy tested to help kids with rare muscle disease

NCT ID NCT05089656

Completed Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

Summary

This study tested a single-dose gene therapy called OAV101 in children and teens (ages 2 to 18) with spinal muscular atrophy (SMA) type 2. The goal was to see if the treatment could safely improve muscle movement and function. It was a large, late-stage trial where some participants received the real treatment and others received a sham procedure for comparison.

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Contacts and locations

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Locations

AIIMS, Ansari Nagar

New Delhi, New Delhi, 110029, India
Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Child Hosp Of The Kings Daughters

Norfolk, Virginia, 23507, United States
Children's Specialty Group/CHKD

Norfolk, Virginia, 23507, United States
Clinic for Special Children

Strasburg, Pennsylvania, 17579, United States
Connecticut Children's Medical Center

Farmington, Connecticut, 06032, United States
Hospital Civil De Guadalajara Fray Antonio Alcalde

Guadalajara, Jalisco, 44280, Mexico
Kaohsiung Medical University Hospital

Kaohsiung City, 80756, Taiwan
National Children's Hospital

Hanoi, 100000, Vietnam
Novartis Investigative Site

Curitiba, Paraná, 81520-060, Brazil
Novartis Investigative Site

São Paulo, São Paulo, 05403 000, Brazil
Novartis Investigative Site

Beijing, Beijing Municipality, 100034, China
Novartis Investigative Site

Chongqing, Chongqing Municipality, 400010, China
Novartis Investigative Site

Guangzhou, Guangdong, 510623, China
Novartis Investigative Site

Shenzhen, Guangdong, 518034, China
Novartis Investigative Site

Chengdu, Sichuan, 610041, China
Novartis Investigative Site

Hangzhou, Zhejiang, 310052, China
Novartis Investigative Site

Beijing, 100069, China
Novartis Investigative Site

Beijing, 100730, China
Novartis Investigative Site

Copenhagen, 2100 O, Denmark
Novartis Investigative Site

New Delhi, National Capital Territory of Delhi, 110029, India
Novartis Investigative Site

New Delhi, National Capital Territory of Delhi, 110060, India
Novartis Investigative Site

Kolkata, West Bengal, 700094, India
Novartis Investigative Site

Hyderabad, 500034, India
Novartis Investigative Site

Mumbai, 400016, India
Novartis Investigative Site

Kuala Lumpur, 50300, Malaysia
Novartis Investigative Site

Kuala Lumpur, 59100, Malaysia
Novartis Investigative Site

Guadalajara, Jalisco, 44280, Mexico
Novartis Investigative Site

Mexico City, Mexico City, 06720, Mexico
Novartis Investigative Site

Riyadh, 11211, Saudi Arabia
Novartis Investigative Site

Singapore, 119074, Singapore
Novartis Investigative Site

Cape Town, 7925, South Africa
Novartis Investigative Site

Kaohsiung City, 80756, Taiwan
Novartis Investigative Site

Bangkok, 10700, Thailand
Novartis Investigative Site

Hanoi, 100000, Vietnam
P.D. Hinduja National Hospital & MRC

Mumbai, 400016, India
Paediatric Neurology

Copenhagen, 2100 O, Denmark
Peking University First Hospital

Beijing, 100034, China
Red Cross War Memorial Childrens Hospital

Cape Town, 7925, South Africa
Sir Ganga Ram Hospital

New Delhi, National Capital Territory of Delhi, 110 060, India
Siriraj Hospital

Bangkok, 10700, Thailand
St Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Conditions

Explore the condition pages connected to this study.

TYPE 2 SPINAL MUSCULAR ATROPHY