Gene Therapy's long shadow: 5-Year watch on SMA kids

NCT ID NCT05335876

Recruiting now Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

Summary

This study follows 175 patients with spinal muscular atrophy (SMA) for five years after they received the gene therapy OAV101 in earlier trials. The main goal is to monitor the long-term safety of the treatment and see if its benefits in improving motor skills are sustained over time. Researchers will track serious side effects and measure abilities like sitting, crawling, and walking.

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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
RECRUITING

Sydney, New South Wales, 2031, Australia
Novartis Investigative Site
RECRUITING

Leuven, 3000, Belgium
Novartis Investigative Site
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Curitiba, Paraná, 81520-060, Brazil
Novartis Investigative Site
RECRUITING

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

Montreal, Quebec, H4A 3J1, Canada
Novartis Investigative Site
RECRUITING

Chongqing, Chongqing Municipality, 400010, China
Novartis Investigative Site
RECRUITING

Guangzhou, Guangdong, 510623, China
Novartis Investigative Site
RECRUITING

Chengdu, Sichuan, 610041, China
Novartis Investigative Site
RECRUITING

Beijing, 100034, China
Novartis Investigative Site
RECRUITING

Beijing, 100069, China
Novartis Investigative Site
RECRUITING

Beijing, 100730, China
Novartis Investigative Site
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Copenhagen, 2100 O, Denmark
Novartis Investigative Site
RECRUITING

Bron, 69677, France
Novartis Investigative Site
RECRUITING

Garches, 92380, France
Novartis Investigative Site
RECRUITING

Strasbourg, 67000, France
Novartis Investigative Site
RECRUITING

Toulouse, 31059, France
Novartis Investigative Site
RECRUITING

Roma, RM, 00168, Italy
Novartis Investigative Site
RECRUITING

Kurume, Fukuoka, 830-0011, Japan
Novartis Investigative Site
RECRUITING

Shinjuku Ku, Tokyo, 1628666, Japan
Novartis Investigative Site
RECRUITING

Kuala Lumpur, 50300, Malaysia
Novartis Investigative Site
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Kuala Lumpur, 59100, Malaysia
Novartis Investigative Site
RECRUITING

Utrecht, 3584 CX, Netherlands
Novartis Investigative Site
RECRUITING

Riyadh, 11211, Saudi Arabia
Novartis Investigative Site
RECRUITING

Singapore, 119074, Singapore
Novartis Investigative Site
RECRUITING

Barcelona, 08035, Spain
Novartis Investigative Site
RECRUITING

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
RECRUITING

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)