Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Launched by NOVARTIS PHARMACEUTICALS · Nov 20, 2019
Trial Information
Current as of June 27, 2025
Recruiting
Keywords
ClinConnect Summary
This clinical trial is a registry for patients diagnosed with Spinal Muscular Atrophy (SMA), a genetic condition that affects the nerves controlling muscle movement. SMA occurs when a specific gene is missing or altered, leading to weak muscles and, unfortunately, is one of the leading causes of infant death from genetic diseases. This registry aims to collect information on the long-term outcomes of patients with SMA as new treatments become available, including those that help replace or enhance the missing gene and support muscle health.
To participate, patients need to have a confirmed diagnosis of SMA since May 24, 2018, and must provide consent to join the study. The trial is open to patients of all ages and genders, except those currently involved in other clinical trials testing new SMA treatments. By joining this registry, participants will help researchers understand how new therapies are affecting SMA patients over time, which could lead to better care and outcomes for future patients.
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • Patients treated with OAV-101 with a genetically confirmed diagnosis of SMA regardless of the date of diagnosis.
- • Appropriate consent/assent has been obtained for participation in the registry
- Exclusion Criteria:
- • - Currently enrolled in an interventional clinical trial involving an investigational medicinal product to treat SMA.
- • Note: Patients who are participating in a Compassionate Use Program (CUP) for OAV-101 (Zolgensma) such as a Managed Access Program (MAP), an Expanded Access Program (EAP), Single Patient Investigational New Drug (IND) (SPI) or Named Patient Program (NPP) are eligible to enroll in the registry regardless of the date of a genetic or clinical diagnosis of SMA.
About Novartis Pharmaceuticals
Novartis Pharmaceuticals is a global healthcare company dedicated to reimagining medicine to improve and extend people's lives. With a strong focus on innovation, Novartis engages in the research, development, and commercialization of a broad range of therapies across various therapeutic areas, including oncology, cardiology, dermatology, and neuroscience. The company is committed to advancing scientific knowledge and patient care through clinical trials that prioritize safety and efficacy. Novartis leverages cutting-edge technology and collaborative partnerships to address unmet medical needs and deliver transformative treatments that enhance patient outcomes worldwide.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Kansas City, Kansas, United States
Minneapolis, Minnesota, United States
Madison, Wisconsin, United States
Rochester, New York, United States
Norfolk, Virginia, United States
Cincinnati, Ohio, United States
Oklahoma City, Oklahoma, United States
Akron, Ohio, United States
Phoenix, Arizona, United States
Pittsburgh, Pennsylvania, United States
Portland, Oregon, United States
Salt Lake City, Utah, United States
Houston, Texas, United States
Portland, Oregon, United States
Aurora, Colorado, United States
Miami, Florida, United States
Columbus, Ohio, United States
Fort Myers, Florida, United States
Milwaukee, Wisconsin, United States
San Diego, California, United States
Charlottesville, Virginia, United States
Louisville, Kentucky, United States
Taipei, , Taiwan
Little Rock, Arkansas, United States
Los Angeles, California, United States
Tacoma, Washington, United States
Taipei, , Taiwan
Taipei, , Taiwan
Richmond, Virginia, United States
Shinjuku Ku, Tokyo, Japan
Seattle, Washington, United States
Sacramento, California, United States
Seongnam Si, , Korea, Republic Of
Seoul, , Korea, Republic Of
Greenville, South Carolina, United States
Bucharest, , Romania
Fort Worth, Texas, United States
Pittsburgh, Pennsylvania, United States
Morristown, New Jersey, United States
Daegu, , Korea, Republic Of
Seoul, , Korea, Republic Of
Cleveland, Ohio, United States
Kurume Shi, Fukuoka, Japan
Shimotsuke Shi, Tochigi, Japan
Madera, California, United States
Sapporo Shi, Hokkaido, Japan
Kumamoto Shi, Kumamoto, Japan
Holon, , Israel
Greenville, South Carolina, United States
Saint Louis, Missouri, United States
Kita Gun, Kagawa, Japan
Columbia, Missouri, United States
Hershey, Pennsylvania, United States
Yangsan, , Korea, Republic Of
Seoul, , Korea, Republic Of
Loma Linda, California, United States
Fukui, , Japan
Kaohsiung City, , Taiwan
Saint Petersburg, , Russian Federation
Iowa City, Iowa, United States
Chiba Shi, Chiba, Japan
Austin, Texas, United States
Nagakute Shi, Aichi, Japan
Shinjuku Ku, Tokyo, Japan
Durham, North Carolina, United States
Taoyuan, , Taiwan
Taichung, North District, Taiwan
Salt Lake City, Utah, United States
Los Angeles, California, United States
Madera, California, United States
Farmington, Connecticut, United States
New Haven, Connecticut, United States
Orlando, Florida, United States
Palmetto Bay, Florida, United States
Indianapolis, Indiana, United States
Stony Brook, New York, United States
Memphis, Tennessee, United States
Dallas, Texas, United States
San Antonio, Texas, United States
Chaïdári, Athens, Greece
Thessaloníki, , Greece
Dublin, , Ireland
Be'er Sheva, , Israel
H̱olon, , Israel
Petah Tikva, , Israel
Toyoake Shi, Aichi, Japan
Gifu Shi, Gifu, Japan
Muroran Shi, Hokkaido, Japan
Yokohama Shi, Kanagawa, Japan
Sendai Shi, Miyagi, Japan
Hokkaido, Obihiro Shi, Japan
Moriyama Shi, Shiga, Japan
Hamamatsu Shi, Shizuoka, Japan
Lisboa, , Portugal
Yongin Si, , Korea, Republic Of
Seoul, , Korea, Republic Of
Penteli, Attikis, Greece
Thessaloniki, , Greece
Hokkaido, , Japan
Saitama, , Japan
Lublin, , Poland
Coimbra, , Portugal
Lisboa, , Portugal
Porto, , Portugal
Porto, , Portugal
Bucharest, , Romania
Patients applied
Trial Officials
Novartis Pharmaceuticals
Study Director
Novartis Pharmaceuticals
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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