Last Update: Nov 04, 2024
A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
ClinicalTrials.gov Identifier:
Novartis Reference Number:COAV101A12001
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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. This is a prospective, multi center, multinational, non-interventional observational
study. All patients will be managed according to the clinical site's normal clinical
practice, i.e., the diagnostic and clinical treatment/practice process that a clinician
chooses according to their clinical judgement for an SMA patient. Clinical care will not
be driven by the protocol. No additional visits or investigations will be performed
beyond normal clinical practice. Patients will be followed for 15 years from enrolment or
until death, whichever is sooner.

Spinal Muscular Atrophy (SMA)
Recruiting
500
Sep 25, 2018
All
- (Child, Adult, Older Adult)

Interventions

Other

Prospective observational registry

This prospective observational registry will assess long-term outcomes of patients with a diagnosis of SMA.
Drug

Zolgensma

Zolgensma will be given to patients as per normal clinical practice and clinical care will not be mandated by the protocol. As such, the decision to prescribe Zolgensma is separate from the decision to include the patient in this study

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.

University General Hospital Attikon

Recruiting

Chaïdári,Athens,124 62,Greece

Argyrios Dinopoulos, MD
Argyrios Dinopoulos, MD

Penteli Children's Hospital

Recruiting

Penteli,Attikis,152 36,Greece

Chrysanthi Tsimakidi
Chrysanthi Tsimakidi

General Hospital of Thessaloniki Ippokrateio

Recruiting

Thessaloníki,546 42,Greece

Dimitrios Zafeiriou, MD
Dimitrios Zafeiriou, MD

St. Sophia Children's Hospital

Recruiting

Thessaloniki,115 27,Greece

Maria Roser-Pons, MD, MD
Maria Roser-Pons, MD

Children's University Hospital-UCD School of Medicine Scoil an Leighis

Recruiting

Dublin,1 d01 xd99,Ireland

Declan O Rourke, MD

Wolfson Medical Center

Recruiting

Holon,5822012,Israel

Mira Ginsberg, MD
Mira Ginsberg, MD

Tel-Aviv Sourasky Medical Center

Recruiting

H̱olon,582201,Israel

Aviva Fattal, MD
Aviva Fattal, MD

Schneider- Children's Medical Center

Recruiting

Petah tikva,4920235,Israel

Sharon Aharoni, MD
Sharon Aharoni, MD

Soroka Medical Centre

Recruiting

Be'er Sheva,85025,Israel

Iris Noyman, MD
Iris Noyman, MD

Seoul National University Children's Hospital

Recruiting

Seoul,03080,Korea, Republic of

Miae Kim, SC
Jong Hee Chae
MD

Kyungpook National University Hospital

Recruiting

Daegu,41944,Korea, Republic of

Yunjeong Lee, MD
Jeong Jin Yun, SC

Samsung Medical Centre

Recruiting

Seoul,06351,Korea, Republic of

Jiwon Lee
Min Jeong Kim, SC

Seoul National University Bundang Hospital

Recruiting

Seongnam-si,13620,Korea, Republic of

Anna Cho, MD
Anna Cho, MD

Yongin Severance Hospital

Recruiting

Yongin-si,16995,Korea, Republic of

Haneul Lee
Lae-song Noh, SC

Severance Hospital

Recruiting

Seoul,03722,Korea, Republic of

Hoonchul Kang, MD
Hoonchul Kang, MD

Pusan National University Yangsan Hospital

Recruiting

Yangsan,50612,Korea, Republic of

Yunjin Lee, MD
Yunjin Lee, MD

Uniwersytecki Szpital Dzieciec

Recruiting

Lublin,20-093,Poland

Magdalena Chroscinska-Krawczyk, MD
Magdalena Chroscinska-Krawczyk, MD

Centro Hospitalar Universitario de Sao Joao, EPE

Recruiting

Porto,4200-319,Portugal

Raquel Carvalho Sousa, MD
Raquel Carvalho Sousa

CHUC - Hospital Pediatrico

Recruiting

Coimbra,300-6020,Portugal

Joana Ribeiro, MD
Joana Ribeiro, MD

Centro Hospitalar Universitario Lisboa Norte, EPE

Recruiting

Lisboa,1649-035,Portugal

Teresa Moreno, MD
Teresa Moreno, MD
Joana Coelho, MD

Centro Hospitalar Universitaria de Lisboa Central, EPE

Recruiting

Lisboa,1169-045,Portugal

Jose Pedro Vieira, MD
Jose Pedro Vieira, MD

Centro Hospitalar Universitario de Porto, EPE

Recruiting

Porto,4050-371,Portugal

Cristina Garrido, MD
Cristina Garrido, MD

Centrul de Recuperare pentru Copii dr. Nicolae Robanescu

Recruiting

Bucharest,041408,Romania

Madalina Cristina Leanca, MD
Madalina Cristina Leanca

Spitalul Clinic de Psihiatrie

Recruiting

Bucharest,041914,Romania

Loana Minciu, MD
Loana Minciu

Almazov National Medical Research Centre

Recruiting

Saint Petersburg,197341,Russian Federation

Natalia Petrova, MD
Natalia Petrova, md

Chang Gung Memorial Hospital Linkou Branch

Recruiting

Taoyuan,333,Taiwan

I-Jun CHOU, MD
I-Jun CHOU, MD

China Medical University Hospital

Recruiting

Taichung,404327,Taiwan

I-Ching Chou, MD
I-Ching Chou

National Taiwan University Hospital

Recruiting

Taipei,100229,Taiwan

Yin-Hsiu CHIEN, MD
Yin-Hsiu CHIEN, MD

Taipei Veterans General Hospital

Recruiting

Taipei,11217,Taiwan

Dau-Ming NIU, MD
Dau-Ming NIU

Kaohsiung Medical University Chung-Ho Memorial Hospital

Recruiting

Kaohsiung city,80756,Taiwan

Yuh-Jyh Jong, MD
Yuh-Jyh Jong, MD

Jackson South Medical Center

Recruiting

Palmetto Bay,Florida,33157,United States

Paula Schleifer, MD
Paula Schleifer, MD

Penn State Hershey

Recruiting

Hershey,Pennsylvania,17033,United States

Ellen Stoute
Ashutosh Kumar, MD

Children's Hospital of Orange County

Recruiting

Madera,California,93638,United States

Amanda Fernandez, MD
Amanda Fernandez, MD
Linda Do

CHRISTUS Health

Recruiting

San Antonio,Texas,78207,United States

Melissa Svoboda, MD
Melissa Svoboda, MD

University of Missouri Health System

Recruiting

Columbia,Missouri,65212,United States

Anne Bonnett
Jane Emerson, MD

Yale-New Haven Health System

Recruiting

New Haven,Connecticut,06510,United States

Catherine Tsao
Cristian Ionita, MD

University of Wisconsin

Recruiting

Madison,Wisconsin,53792,United States

Jennifer Kwon, MD
Maggie Chilsen

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati,Ohio,45229-3026,United States

Cuixia Tian, MD
Emily Hunsaker

University of Kansas Medical Center

Recruiting

Kansas City,Kansas,66160,United States

Jeffrey Statland, MD
Rebecca Clay

Children's Hospital of Los Angeles

Recruiting

Los Angeles,California,90027,United States

Leigh Maria Ramos-Platt, MD
Martha Arellano-Garcia
Claudia Dozal

Methodist Le Bonheur Healthcare

Recruiting

Memphis,Tennessee,38103,United States

Farimah Salami
Elena Caron, MD

Rady Children's Hospital San Diego

Recruiting

San Diego,California,92123,United States

Napat Intarachumnum
Chamindra Konersman, MD
Chamindra Konersman, MD

Virginia Commonwealth University Health System

Recruiting

Richmond,Virginia,23298,United States

Amy Harper, MD
Ana Rosa Rezeq

University of Rochester Medical Center

Recruiting

Rochester,New York,14642,United States

Bohoon Lee
Kim Hart Kim_
Bohoon Lee, MD

Indiana University Health University Hospital (IUHUH)

Recruiting

Indianapolis,Indiana,46202,United States

Leanne Dunn
Marcia Felker, MD
Marcia Felker, MD

University of Pittsburgh Medical Center

Recruiting

Pittsburgh,Pennsylvania,15224,United States

Emma Sekscinski
Hoda Abdel-Hamid, MD

University of Utah

Recruiting

Salt Lake City,Utah,84113,United States

Russell Butterfield, MD, PhD
Sarah Moldt

Nationwide Children's Hospital

Recruiting

Columbus,Ohio,43205,United States

Megan Waldrop, MD
Megan Waldrop

Cook Children's

Recruiting

Fort Worth,Texas,76104,United States

Kayla Blough
Warren Marks, MD

Children's Hospital Colorado

Recruiting

Aurora,Colorado,80045,United States

Julie Parsons, MD
Margaret Finlay

Phoenix Children's Hospital

Recruiting

Phoenix,Arizona,85016,United States

Alison Lane
Saunder Bernes, MD

Washington University School of Medicine in St. Louis

Recruiting

Saint Louis,Missouri,63110,United States

Craig Zaidman, MD
Natalie Goedeker

Golisano Children's Hospital of Southwest Florida

Recruiting

Fort Myers,Florida,33908,United States

Britt Stroud, MD
Britt Stroud, MD
Molly Arnstrom

Children's Hospital of Wisconsin

Recruiting

Milwaukee,Wisconsin,53226,United States

Matthew Harmelink, MD
Rebecca Rehborg
Rupa Nallamothu

University of Louisville

Recruiting

Louisville,Kentucky,40202,United States

Arpita Lakhotia, MD
Lauren Evanczyk

University of California Los Angeles Health

Recruiting

Los Angeles,California,90095,United States

Huynh Jennifer
Perry Shieh, MD, PhD

The State University of New York

Recruiting

Stony Brook,New York,11794,United States

Christine Pol
Simona Treidler, MD

Seattle Children's

Recruiting

Seattle,Washington,98105,United States

Marissa Robertson
Seth Perlman, MD

Prisma Health

Recruiting

Greenville,South Carolina,29615,United States

Addie Hunnicutt Hunnicutt
Addie Hunnicutt, MD
Addie Hunnicutt

University of Virginia Health System

Recruiting

Charlottesville,Virginia,22908,United States

Chelsea Masterson
Rebecca Scharf, MD

University of California Davis Health System

Recruiting

Sacramento,California,95817,United States

Nanette Joyce, DO
Rachel (Ja Yoon) Baek

Oregon Health and Science University

Recruiting

Portland,Oregon,97239-3098,United States

Bryn McCarthy
Erika Finanger, MD

Texas Children's Hospital

Recruiting

Houston,Texas,77030,United States

Felize Singleton
Timothy Lotze, MD

Connecticut Children's Medical Center

Recruiting

Farmington,Connecticut,06034,United States

Gyula Acsadi, MD, PhD
Hendriana Nielsen
James Santanelli

Arkansas Children's Hospital

Recruiting

Little Rock,Arkansas,72202,United States

Kapil Arya, MD
Kapil Arya

University of Iowa

Recruiting

Iowa City,Iowa,52242-1009,United States

Carrie Stephan
Chandra Miller
Katherine Mathews, MD

Atlantic Health System

Recruiting

Morristown,New Jersey,07960,United States

Darius Adams, MD
Darius Adams, MD

Valley Children's Healthcare

Recruiting

Madera,California,93636,United States

Raymund David, MD
Raymund David, MD

University of Minnesota

Recruiting

Minneapolis,Minnesota,55455,United States

Peter Karachunski, MD
Seth Stafki, MD

MultiCare Health System

Recruiting

Tacoma,Washington,98405,United States

Anisha Schwarz, MD
Beky Stone

Duke Health

Recruiting

Durham,North Carolina,27705,United States

Karen Cornett
Mai Elmallah, MD

Children's Hospital of The King's Daughters

Recruiting

Norfolk,Virginia,23507,United States

Cassandra Todd
Crystal Proud, MD

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