SMA patients who got gene therapy to be tracked for 5 more years

NCT ID NCT05335876

Recruiting now Knowledge-focused Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jan 21, 2026 · Last updated May 16, 2026 · Updated 19 times

Summary

This study follows about 175 people with spinal muscular atrophy (SMA) who already received the gene therapy OAV101 in a previous clinical trial. Researchers will monitor them for 5 years to check for any serious side effects and to see how their motor skills develop. The goal is to understand the long-term safety and benefits of this treatment.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

Phone: •••-•••-••••

Locations

Child Hosp Of The Kings Daughters
RECRUITING

Norfolk, Virginia, 23507, United States

Contact

Contact Phone: •••-•••-•••• Email: •••••@•••••
Novartis Investigative Site
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Sydney, New South Wales, 2031, Australia
Novartis Investigative Site
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Leuven, 3000, Belgium
Novartis Investigative Site
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Curitiba, Paraná, 81520-060, Brazil
Novartis Investigative Site
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São Paulo, São Paulo, 05403 000, Brazil
Novartis Investigative Site
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Montreal, Quebec, H4A 3J1, Canada
Novartis Investigative Site
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Chongqing, Chongqing Municipality, 400010, China
Novartis Investigative Site
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Guangzhou, Guangdong, 510623, China
Novartis Investigative Site
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Chengdu, Sichuan, 610041, China
Novartis Investigative Site
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Beijing, 100034, China
Novartis Investigative Site
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Beijing, 100069, China
Novartis Investigative Site
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Beijing, 100730, China
Novartis Investigative Site
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Copenhagen, 2100 O, Denmark
Novartis Investigative Site
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Bron, 69677, France
Novartis Investigative Site
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Garches, 92380, France
Novartis Investigative Site
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Strasbourg, 67000, France
Novartis Investigative Site
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Toulouse, 31059, France
Novartis Investigative Site
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Roma, RM, 00168, Italy
Novartis Investigative Site
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Kurume, Fukuoka, 830-0011, Japan
Novartis Investigative Site
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Shinjuku Ku, Tokyo, 1628666, Japan
Novartis Investigative Site
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Kuala Lumpur, 50300, Malaysia
Novartis Investigative Site
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Kuala Lumpur, 59100, Malaysia
Novartis Investigative Site
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Utrecht, 3584 CX, Netherlands
Novartis Investigative Site
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Riyadh, 11211, Saudi Arabia
Novartis Investigative Site
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Singapore, 119074, Singapore
Novartis Investigative Site
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Barcelona, 08035, Spain
Novartis Investigative Site
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Kaohsiung City, 80756, Taiwan
Novartis Investigative Site
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Taipei, 10002, Taiwan
Novartis Investigative Site
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Bangkok, 10700, Thailand
Novartis Investigative Site
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London, WC1N 3JH, United Kingdom
Novartis Investigative Site
RECRUITING

Newcastle upon Tyne, NE1 4LP, United Kingdom
Novartis Investigative Site
RECRUITING

Hanoi, 100000, Vietnam

Conditions

Explore the condition pages connected to this study.

SPINAL MUSCULAR ATROPHY (SMA)