Actively Recruiting
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Led by Novartis Pharmaceuticals · Updated on 2025-01-31
700
Participants Needed
99
Research Sites
1031 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases. The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options and also to characterize and assess long-term safety and effectiveness of OAV-101.
CONDITIONS
Official Title
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients treated with OAV-101 with a genetically confirmed diagnosis of SMA regardless of the date of diagnosis
- Appropriate consent or assent obtained for participation in the registry
- Patients in Compassionate Use Programs for OAV-101 such as Managed Access Program, Expanded Access Program, Single Patient IND, or Named Patient Program are eligible regardless of diagnosis date
You will not qualify if you...
- Currently enrolled in an interventional clinical trial involving an investigational drug to treat SMA
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 99 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
3
Loma Linda University Health
Loma Linda, California, United States, 92350
Completed
4
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
5
University of California Los Angeles Health
Los Angeles, California, United States, 90095
Actively Recruiting
6
Valley Children's Healthcare
Madera, California, United States, 93636
Actively Recruiting
7
Children's Hospital of Orange County
Madera, California, United States, 93638
Actively Recruiting
8
University of California Davis Health System
Sacramento, California, United States, 95817
Actively Recruiting
9
Rady Children's Hospital San Diego
San Diego, California, United States, 92123
Actively Recruiting
10
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
11
Connecticut Children's Medical Center
Farmington, Connecticut, United States, 06034
Actively Recruiting
12
Yale-New Haven Health System
New Haven, Connecticut, United States, 06510
Actively Recruiting
13
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida, United States, 33908
Actively Recruiting
14
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Completed
15
Adventist Health System - Florida
Orlando, Florida, United States, 32803
Completed
16
Jackson South Medical Center
Palmetto Bay, Florida, United States, 33157
Actively Recruiting
17
Indiana University Health University Hospital (IUHUH)
Indianapolis, Indiana, United States, 46202
Actively Recruiting
18
University of Iowa
Iowa City, Iowa, United States, 52242-1009
Actively Recruiting
19
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Actively Recruiting
20
University of Louisville
Louisville, Kentucky, United States, 40202
Actively Recruiting
21
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
22
University of Missouri Health System
Columbia, Missouri, United States, 65212
Actively Recruiting
23
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
24
Atlantic Health System
Morristown, New Jersey, United States, 07960
Actively Recruiting
25
University of Rochester Medical Center
Rochester, New York, United States, 14642
Actively Recruiting
26
The State University of New York
Stony Brook, New York, United States, 11794
Actively Recruiting
27
Duke Health
Durham, North Carolina, United States, 27705
Actively Recruiting
28
Akron Children's Hospital
Akron, Ohio, United States, 44308-1062
Completed
29
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3026
Actively Recruiting
30
University Hospitals
Cleveland, Ohio, United States, 44106
Completed
31
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
32
Oklahoma University Medical Center
Oklahoma City, Oklahoma, United States, 73104
Completed
33
Oregon Health and Science University
Portland, Oregon, United States, 97239-3098
Actively Recruiting
34
Penn State Hershey
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
35
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15224
Actively Recruiting
36
Prisma Health
Greenville, South Carolina, United States, 29615
Actively Recruiting
37
Methodist Le Bonheur Healthcare
Memphis, Tennessee, United States, 38103
Actively Recruiting
38
Child Neurology Consultants of Austin
Austin, Texas, United States, 78731
Completed
39
Children's Health
Dallas, Texas, United States, 75207
Completed
40
Cook Children's
Fort Worth, Texas, United States, 76104
Actively Recruiting
41
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
42
CHRISTUS Health
San Antonio, Texas, United States, 78207
Actively Recruiting
43
University of Utah
Salt Lake City, Utah, United States, 84113
Actively Recruiting
44
University of Virginia Health System
Charlottesville, Virginia, United States, 22908
Actively Recruiting
45
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507
Actively Recruiting
46
Virginia Commonwealth University Health System
Richmond, Virginia, United States, 23298
Actively Recruiting
47
Seattle Children's
Seattle, Washington, United States, 98105
Actively Recruiting
48
MultiCare Health System
Tacoma, Washington, United States, 98405
Actively Recruiting
49
University of Wisconsin
Madison, Wisconsin, United States, 53792
Actively Recruiting
50
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
51
University General Hospital Attikon
Chaïdári, Athens, Greece, 124 62
Actively Recruiting
52
Penteli Children's Hospital
Pentéli, Attikis, Greece, 152 36
Actively Recruiting
53
St. Sophia Children's Hospital
Thessaloniki, Greece, 115 27
Actively Recruiting
54
General Hospital of Thessaloniki Ippokrateio
Thessaloniki, Greece, 546 42
Actively Recruiting
55
Children's University Hospital-UCD School of Medicine Scoil an Leighis
Dublin, Ireland, 1 D01 XD99
Actively Recruiting
56
Soroka Medical Centre
Beersheba, Israel, 85025
Actively Recruiting
57
Wolfson Medical Center
Holon, Israel, 5822012
Actively Recruiting
58
Tel-Aviv Sourasky Medical Center
Holon, Israel, 582201
Actively Recruiting
59
Schneider- Children's Medical Center
Petah Tikva, Israel, 4920235
Actively Recruiting
60
Aichi Medical University Hospital
Nagakute-shi, Aichi-ken, Japan, 480-1195
Active, Not Recruiting
61
Fujita Health University Hospital
Toyoake-shi, Aichi-ken, Japan, 470-1192
Active, Not Recruiting
62
Chiba children's Hospital
Chiba, Chiba, Japan, 266-0007
Active, Not Recruiting
63
Kurume University Hospital
Kurume-shi, Fukuoka, Japan, 830-0011
Active, Not Recruiting
64
Gifu Prefectural General Medical Center
Gifu, Gifu, Japan, 500-8717
Active, Not Recruiting
65
Sapporo Medical University Hospital
Sapporo, Hokkaido, Japan, 060-8543
Active, Not Recruiting
66
Kagawa University Hospital
Kita-gun, Kagawa-ken, Japan, 761-0793
Active, Not Recruiting
67
Kanagawa Children's Medical Center
Yokohama, Kanagawa, Japan, 232-8555
Active, Not Recruiting
68
Kumamoto University Hospital
Kumamoto, Kumamoto, Japan, 860-8556
Active, Not Recruiting
69
Miyagi Children's Hospital
Sendai, Miyagi, Japan, 989-3126
Active, Not Recruiting
70
Obihiro Kosei Hospital
Hokkaido, Obihiro-shi, Japan, 080-0024
Active, Not Recruiting
71
Shiga Medical Center for Children
Moriyama-shi, Shiga, Japan, 524-0022
Active, Not Recruiting
72
Hamamatsu University School of Medicine, University Hospital
Hamamatsu, Shizuoka, Japan, 431-3192
Active, Not Recruiting
73
Jichi Medical University Hospital
Shimotsuke-shi, Tochigi, Japan, 329-0498
Active, Not Recruiting
74
Tokyo Medical University Hospital
Shinjuku-ku, Tokyo, Japan, 160-0023
Active, Not Recruiting
75
Center Hospital of the National Center for Global Health and Medicine
Shinjuku-ku, Tokyo, Japan, 162-8655
Active, Not Recruiting
76
Fukui Prefectural Hospital
Fukui, Japan, 910-8526
Active, Not Recruiting
77
Nikko Memorial Hospital
Hokkaido, Japan, 051-0005
Active, Not Recruiting
78
Dokkyo Medical University Saitama Medical Center
Saitama, Japan, 343-0845
Active, Not Recruiting
79
Uniwersytecki Szpital Dzieciec
Lublin, Poland, 20-093
Actively Recruiting
80
CHUC - Hospital Pediatrico
Coimbra, Portugal, 300-6020
Actively Recruiting
81
Centro Hospitalar Universitaria de Lisboa Central, EPE
Lisbon, Portugal, 1169-045
Actively Recruiting
82
Centro Hospitalar Universitario Lisboa Norte, EPE
Lisbon, Portugal, 1649-035
Actively Recruiting
83
Centro Hospitalar Universitario de Porto, EPE
Porto, Portugal, 4050-371
Actively Recruiting
84
Centro Hospitalar Universitario de Sao Joao, EPE
Porto, Portugal, 4200-319
Actively Recruiting
85
Centrul de Recuperare pentru Copii dr. Nicolae Robanescu
Bucharest, Romania, 041408
Actively Recruiting
86
Spitalul Clinic de Psihiatrie
Bucharest, Romania, 041914
Actively Recruiting
87
Almazov National Medical Research Centre
Saint Petersburg, Russia, 197341
Actively Recruiting
88
Kyungpook National University Hospital
Daegu, South Korea, 41944
Actively Recruiting
89
Seoul National University Bundang Hospital
Seongnam-si, South Korea, 13620
Actively Recruiting
90
Seoul National University Children's Hospital
Seoul, South Korea, 03080
Actively Recruiting
91
Severance Hospital
Seoul, South Korea, 03722
Actively Recruiting
92
Samsung Medical Centre
Seoul, South Korea, 06351
Actively Recruiting
93
Pusan National University Yangsan Hospital
Yangsan, South Korea, 50612
Actively Recruiting
94
Yongin Severance Hospital
Yongin-si, South Korea, 16995
Actively Recruiting
95
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, Taiwan, 80756
Actively Recruiting
96
China Medical University Hospital
Taichung, Taiwan, 404327
Actively Recruiting
97
National Taiwan University Hospital
Taipei, Taiwan, 100229
Actively Recruiting
98
Taipei Veterans General Hospital
Taipei, Taiwan, 11217
Actively Recruiting
99
Chang Gung Memorial Hospital Linkou Branch
Taoyuan, Taiwan, 333
Actively Recruiting
Research Team
N
Novartis Gene Therapies
CONTACT
N
Novartis Gene Therapies
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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