Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

January 30, 2025 updated by: Novartis Pharmaceuticals

A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Observational

Enrollment (Estimated)

700

Expanded Access

Approved for sale to the public. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

  • Name: Novartis Gene Therapies
  • Phone Number: +4161324111

Study Locations

  • Greece
      • Thessaloniki, Greece, 115 27
        • Recruiting
        • St. Sophia Children's Hospital
        • Contact:
        • Contact:
          • Maria Roser-Pons, MD, MD
      • Thessaloníki, Greece, 546 42
        • Recruiting
        • General Hospital of Thessaloniki Ippokrateio
        • Principal Investigator:
          • Dimitrios Zafeiriou, MD
        • Contact:
          • Dimitrios Zafeiriou, MD
          • Phone Number: +30 2310 99 29 63
          • Email: dizafeir@auth.gr
    • Athens
      • Chaïdári, Athens, Greece, 124 62
        • Recruiting
        • University General Hospital Attikon
        • Principal Investigator:
          • Argyrios Dinopoulos, MD
        • Contact:
    • Attikis
      • Penteli, Attikis, Greece, 152 36
        • Recruiting
        • Penteli Children's Hospital
        • Contact:
        • Contact:
          • Chrysanthi Tsimakidi
  • Ireland
      • Dublin, Ireland, 1 D01 XD99
        • Recruiting
        • Children's University Hospital-UCD School of Medicine Scoil an Leighis
        • Contact:
  • Israel
      • Be'er Sheva, Israel, 85025
        • Recruiting
        • Soroka Medical Centre
        • Contact:
        • Principal Investigator:
          • Iris Noyman, MD
      • Holon, Israel, 5822012
        • Recruiting
        • Wolfson Medical Center
        • Contact:
        • Principal Investigator:
          • Mira Ginsberg, MD
      • H̱olon, Israel, 582201
        • Recruiting
        • Tel-Aviv Sourasky Medical Center
        • Contact:
        • Principal Investigator:
          • Aviva Fattal, MD
      • Petah tikva, Israel, 4920235
        • Recruiting
        • Schneider- Children's Medical Center
        • Contact:
        • Principal Investigator:
          • Sharon Aharoni, MD
  • Japan
      • Fukui, Japan, 910-8526
        • Active, not recruiting
        • Fukui Prefectural Hospital
      • Hokkaido, Japan, 051-0005
        • Active, not recruiting
        • Nikko Memorial Hospital
      • Saitama, Japan, 343-0845
        • Active, not recruiting
        • Dokkyo Medical University Saitama Medical Center
    • Aichi
      • Nagakute-shi, Aichi, Japan, 480-1195
        • Active, not recruiting
        • Aichi Medical University Hospital
      • Toyoake-shi, Aichi, Japan, 470-1192
        • Active, not recruiting
        • Fujita Health University Hospital
    • Chiba
      • Chiba-shi, Chiba, Japan, 266-0007
        • Active, not recruiting
        • Chiba Children's Hospital
    • Fukuoka
      • Kurume-shi, Fukuoka, Japan, 830-0011
        • Active, not recruiting
        • Kurume University Hospital
    • Gifu
      • Gifu-shi, Gifu, Japan, 500-8717
        • Active, not recruiting
        • Gifu Prefectural General Medical Center
    • Hokkaido
      • Sapporo-shi, Hokkaido, Japan, 060-8543
        • Active, not recruiting
        • Sapporo Medical University Hospital
    • Kagawa
      • Kita-gun, Kagawa, Japan, 761-0793
        • Active, not recruiting
        • Kagawa University Hospital
    • Kanagawa
      • Yokohama-shi, Kanagawa, Japan, 232-8555
        • Active, not recruiting
        • Kanagawa Children's Medical Center
    • Kumamoto
      • Kumamoto-shi, Kumamoto, Japan, 860-8556
        • Active, not recruiting
        • Kumamoto University Hospital
    • Miyagi
      • Sendai-shi, Miyagi, Japan, 989-3126
        • Active, not recruiting
        • Miyagi Children's Hospital
    • Obihiro-shi
      • Hokkaido, Obihiro-shi, Japan, 080-0024
        • Active, not recruiting
        • Obihiro Kosei Hospital
    • Shiga
      • Moriyama-shi, Shiga, Japan, 524-0022
        • Active, not recruiting
        • Shiga Medical Center for Children
    • Shizuoka
      • Hamamatsu-shi, Shizuoka, Japan, 431-3192
        • Active, not recruiting
        • Hamamatsu University School of Medicine, University Hospital
    • Tochigi
      • Shimotsuke-shi, Tochigi, Japan, 329-0498
        • Active, not recruiting
        • Jichi Medical University Hospital
    • Tokyo
      • Shinjuku-ku, Tokyo, Japan, 160-0023
        • Active, not recruiting
        • Tokyo Medical University Hospital
      • Shinjuku-ku, Tokyo, Japan, 162-8655
        • Active, not recruiting
        • Center Hospital of the National Center for Global Health and Medicine
  • Korea, Republic of
      • Daegu, Korea, Republic of, 41944
        • Recruiting
        • Kyungpook National University Hospital
        • Principal Investigator:
          • Yunjeong Lee, MD
        • Contact:
      • Seongnam-si, Korea, Republic of, 13620
        • Recruiting
        • Seoul National University Bundang Hospital
        • Contact:
        • Principal Investigator:
          • Anna Cho, MD
      • Seoul, Korea, Republic of, 03722
        • Recruiting
        • Severance Hospital
        • Contact:
        • Principal Investigator:
          • Hoonchul Kang, MD
      • Seoul, Korea, Republic of, 03080
        • Recruiting
        • Seoul National University Children's Hospital
        • Contact:
          • MD
        • Contact:
        • Principal Investigator:
          • Jong Hee Chae
      • Seoul, Korea, Republic of, 06351
        • Recruiting
        • Samsung Medical Centre
        • Contact:
        • Principal Investigator:
          • Jiwon Lee
      • Yangsan, Korea, Republic of, 50612
        • Recruiting
        • Pusan National University Yangsan Hospital
        • Contact:
        • Principal Investigator:
          • Yunjin Lee, MD
      • Yongin-si, Korea, Republic of, 16995
        • Recruiting
        • Yongin Severance Hospital
        • Contact:
        • Principal Investigator:
          • Haneul Lee
  • Poland
      • Lublin, Poland, 20-093
        • Recruiting
        • Uniwersytecki Szpital Dzieciec
        • Contact:
          • Magdalena Chroscinska-Krawczyk, MD
          • Phone Number: (+48) 669 487 491
          • Email: magdalenachk@wp.pl
        • Principal Investigator:
          • Magdalena Chroscinska-Krawczyk, MD
  • Portugal
      • Coimbra, Portugal, 300-6020
        • Recruiting
        • CHUC - Hospital Pediatrico
        • Contact:
        • Principal Investigator:
          • Joana Ribeiro, MD
      • Lisboa, Portugal, 1649-035
        • Recruiting
        • Centro Hospitalar Universitario Lisboa Norte, EPE
        • Contact:
        • Contact:
        • Principal Investigator:
          • Teresa Moreno, MD
      • Lisboa, Portugal, 1169-045
        • Recruiting
        • Centro Hospitalar Universitaria de Lisboa Central, EPE
        • Contact:
        • Principal Investigator:
          • Jose Pedro Vieira, MD
      • Porto, Portugal, 4050-371
        • Recruiting
        • Centro Hospitalar Universitario de Porto, EPE
        • Contact:
        • Principal Investigator:
          • Cristina Garrido, MD
      • Porto, Portugal, 4200-319
        • Recruiting
        • Centro Hospitalar Universitário de São João, EPE
        • Contact:
        • Principal Investigator:
          • Raquel Carvalho Sousa
  • Romania
      • Bucharest, Romania, 041408
        • Recruiting
        • Centrul de Recuperare pentru Copii dr. Nicolae Robanescu
        • Contact:
          • Madalina Cristina Leanca, MD
          • Phone Number: (+40) 726 274 907
          • Email: mada332@yahoo.com
        • Principal Investigator:
          • Madalina Cristina Leanca
      • Bucharest, Romania, 041914
        • Recruiting
        • Spitalul Clinic de Psihiatrie
        • Contact:
        • Principal Investigator:
          • Loana Minciu
  • Russian Federation
      • Saint Petersburg, Russian Federation, 197341
        • Recruiting
        • Almazov National Medical Research Centre
        • Principal Investigator:
          • Natalia Petrova, md
        • Contact:
  • Taiwan
      • Kaohsiung city, Taiwan, 80756
        • Recruiting
        • Kaohsiung Medical University Chung-Ho Memorial Hospital
        • Principal Investigator:
          • Yuh-Jyh Jong, MD
        • Contact:
      • Taichung, Taiwan, 404327
      • Taipei, Taiwan, 11217
        • Recruiting
        • Taipei Veterans General Hospital
        • Contact:
        • Principal Investigator:
          • Dau-Ming NIU
      • Taipei, Taiwan, 100229
        • Recruiting
        • National Taiwan University Hospital
        • Principal Investigator:
          • Yin-Hsiu CHIEN, MD
        • Contact:
      • Taoyuan, Taiwan, 333
        • Recruiting
        • Chang Gung Memorial Hospital Linkou Branch
        • Contact:
        • Principal Investigator:
          • I-Jun CHOU, MD
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Recruiting
        • Phoenix Children's Hospital
        • Principal Investigator:
          • Saunder Bernes, MD
        • Contact:
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Recruiting
        • Arkansas Children's Hospital
        • Principal Investigator:
          • Kapil Arya, MD
        • Contact:
    • California
      • Loma Linda, California, United States, 92350
        • Completed
        • Loma Linda University Health
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital of Los Angeles
        • Contact:
        • Principal Investigator:
          • Leigh Maria Ramos-Platt, MD
        • Contact:
      • Los Angeles, California, United States, 90095
        • Recruiting
        • University of California Los Angeles Health
        • Contact:
        • Principal Investigator:
          • Perry Shieh, MD, PhD
      • Madera, California, United States, 93636
        • Recruiting
        • Valley Children's Healthcare
        • Contact:
        • Principal Investigator:
          • Raymund David, MD
      • Madera, California, United States, 93638
        • Recruiting
        • Children's Hospital of Orange County
        • Contact:
        • Contact:
        • Principal Investigator:
          • Amanda Fernandez, MD
      • Sacramento, California, United States, 95817
        • Recruiting
        • University of California Davis Health System
        • Contact:
        • Principal Investigator:
          • Nanette Joyce, DO
      • San Diego, California, United States, 92123
        • Recruiting
        • Rady Children's Hospital San Diego
        • Contact:
        • Principal Investigator:
          • Chamindra Konersman, MD
        • Contact:
    • Colorado
      • Aurora, Colorado, United States, 80045
    • Connecticut
      • Farmington, Connecticut, United States, 06034
      • New Haven, Connecticut, United States, 06510
        • Recruiting
        • Yale-New Haven Health System
        • Principal Investigator:
          • Cristian Ionita, MD
        • Contact:
    • Florida
      • Fort Myers, Florida, United States, 33908
        • Recruiting
        • Golisano Children's Hospital of Southwest Florida
        • Contact:
        • Contact:
        • Principal Investigator:
          • Britt Stroud, MD
      • Miami, Florida, United States, 33155
        • Completed
        • Nicklaus Children's Hospital
      • Orlando, Florida, United States, 32803
        • Completed
        • Adventist Health System - Florida
      • Palmetto Bay, Florida, United States, 33157
        • Recruiting
        • Jackson South Medical Center
        • Principal Investigator:
          • Paula Schleifer, MD
        • Contact:
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Indiana University Health University Hospital (IUHUH)
        • Contact:
        • Principal Investigator:
          • Marcia Felker, MD
        • Contact:
    • Iowa
      • Iowa City, Iowa, United States, 52242-1009
        • Recruiting
        • University of Iowa
        • Contact:
        • Contact:
        • Principal Investigator:
          • Katherine Mathews, MD
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Medical Center
        • Contact:
        • Principal Investigator:
          • Jeffrey Statland, MD
    • Kentucky
      • Louisville, Kentucky, United States, 40202
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota
        • Principal Investigator:
          • Peter Karachunski, MD
        • Contact:
    • Missouri
      • Columbia, Missouri, United States, 65212
        • Recruiting
        • University of Missouri Health System
        • Contact:
        • Principal Investigator:
          • Jane Emerson, MD
      • Saint Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University School of Medicine in St. Louis
        • Contact:
        • Principal Investigator:
          • Craig Zaidman, MD
    • New Jersey
      • Morristown, New Jersey, United States, 07960
        • Recruiting
        • Atlantic Health System
        • Contact:
        • Principal Investigator:
          • Darius Adams, MD
    • New York
      • Rochester, New York, United States, 14642
      • Stony Brook, New York, United States, 11794
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Recruiting
        • Duke Health
        • Contact:
        • Principal Investigator:
          • Mai Elmallah, MD
    • Ohio
      • Akron, Ohio, United States, 44308-1062
        • Completed
        • Akron Children's Hospital
      • Cincinnati, Ohio, United States, 45229-3026
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Contact:
        • Principal Investigator:
          • Cuixia Tian, MD
      • Cleveland, Ohio, United States, 44106
        • Completed
        • University Hospitals
      • Columbus, Ohio, United States, 43205
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Completed
        • Oklahoma University Medical center
    • Oregon
      • Portland, Oregon, United States, 97239-3098
        • Recruiting
        • Oregon Health and Science University
        • Contact:
        • Principal Investigator:
          • Erika Finanger, MD
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
      • Pittsburgh, Pennsylvania, United States, 15224
        • Recruiting
        • University of Pittsburgh Medical Center
        • Principal Investigator:
          • Hoda Abdel-Hamid, MD
        • Contact:
    • South Carolina
      • Greenville, South Carolina, United States, 29615
        • Recruiting
        • Prisma Health
        • Contact:
        • Principal Investigator:
          • Addie Hunnicutt, MD
        • Contact:
    • Tennessee
      • Memphis, Tennessee, United States, 38103
        • Recruiting
        • Methodist Le Bonheur Healthcare
        • Contact:
        • Principal Investigator:
          • Elena Caron, MD
    • Texas
      • Austin, Texas, United States, 78731
        • Completed
        • Child Neurology Consultants of Austin
      • Dallas, Texas, United States, 75207
        • Completed
        • Children's Health
      • Fort Worth, Texas, United States, 76104
        • Recruiting
        • Cook Children's
        • Principal Investigator:
          • Warren Marks, MD
        • Contact:
      • Houston, Texas, United States, 77030
        • Recruiting
        • Texas Children's Hospital
        • Principal Investigator:
          • Timothy Lotze, MD
        • Contact:
      • San Antonio, Texas, United States, 78207
        • Recruiting
        • CHRISTUS Health
        • Contact:
        • Principal Investigator:
          • Melissa Svoboda, MD
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Recruiting
        • University of Utah
        • Contact:
        • Principal Investigator:
          • Russell Butterfield, MD, PhD
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • Recruiting
        • University of Virginia Health System
        • Contact:
        • Principal Investigator:
          • Rebecca Scharf, MD
      • Norfolk, Virginia, United States, 23507
        • Recruiting
        • Children's Hospital of The King's Daughters
        • Principal Investigator:
          • Crystal Proud, MD
        • Contact:
      • Richmond, Virginia, United States, 23298
        • Recruiting
        • Virginia Commonwealth University Health System
        • Contact:
        • Principal Investigator:
          • Amy Harper, MD
    • Washington
      • Seattle, Washington, United States, 98105
      • Tacoma, Washington, United States, 98405
        • Recruiting
        • Multicare Health System
        • Principal Investigator:
          • Anisha Schwarz, MD
        • Contact:
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Recruiting
        • University of Wisconsin
        • Contact:
        • Principal Investigator:
          • Jennifer Kwon, MD
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Children's Hospital of Wisconsin
        • Contact:
        • Contact:
        • Principal Investigator:
          • Matthew Harmelink, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The study will enroll at least 500 patients with a genetically confirmed diagnosis of SMA. The registry will attempt to enroll all patients treated with OAV-101 in the registry until the end of 2026.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Ecologic or Community
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Prospective observational registry
This is a prospective, multi center, multinational, non-interventional observational registry.
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in probability of survival of all patients with SMA using Kaplan Meier method to estimate
Time Frame: Based on information collected at Baseline and every 6 months through 2 years of follow-up, then annually through 15 years of follow up.
Based on information collected at Baseline and every 6 months through 2 years of follow-up, then annually through 15 years of follow up.
Change from baseline Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) in infants with pre-symptomatic or type I SMA
Time Frame: Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
CHOP INTEND score ranges from 0 to 64 with higher scores indicating higher motor function
Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Change from baseline Hammersmith Infant Neurological Examination (HINE) in infants with pre-symptomatic, type I or type II SMA
Time Frame: Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
HINE score range from 0 to 26 with higher scores indicating more development.
Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) for patients with type II and III SMA
Time Frame: Baseline and every 6months through 2 years of follow up, then annually through 15 years of follow up
HFMSE score range from 0 to 66 with the higher scores indicating more development.
Baseline and every 6months through 2 years of follow up, then annually through 15 years of follow up
Incidence of treatment emergent adverse events
Time Frame: Through 15 years of follow up
Through 15 years of follow up
Incidence of treatment emergent serious adverse events
Time Frame: Through 15 years of follow up
Through 15 years of follow up
Incidence of treatment emergent adverse events related to therapy
Time Frame: Through 15 years of follow up
Through 15 years of follow up
Incidence of treatment emergent thrombocytopenia, hepatotoxicity and cardiac adverse events
Time Frame: Through 15 years of follow up
Through 15 years of follow up

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in rates of hospitalization
Time Frame: Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Change from baseline in Zarit Burden Interview
Time Frame: Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Zarit Burden Total Score ranges from 0 to 88. A higher score correlates with higher level of burden.
Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Change from baseline in PedsQL Patient interview
Time Frame: Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
PedsQL Total Scale Score is average of all items and ranges from 0 to 100. A higher score correlates with better Health-Related Quality of Life
Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Change from baseline in PedsQL Parent interview
Time Frame: Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
PedsQL Total Scale Score is average of all items and ranges from 0 to 100. A higher score correlates with better Health-Related Quality of Life
Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Change from baseline in percent of patients requiring ventilator support (BiPAP, Endotracheal tube)
Time Frame: : Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
: Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Change from baseline in in percent of patients requiring nutritional support (Gastrostomy Tube, Gastrojejunal tube (GT) with Nissen fundoplication, GT without Nissen fundoplication, Nasogastrictube, Nasojejunaltube or Percutaneous endoscopic gastrostomy)
Time Frame: Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
Change from baseline in in percent of patients requiring mobility device support (Ankle-Foot Orthoses, Supramalleolar Orthosis, Orthotic/shoe inserts, Knee immobilizers, Knee-Ankle-Foot Orthoses , Hand splints, Spinal bracing)
Time Frame: : Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up
: Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Collaborators

United BioSource, LLC

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 25, 2018

Primary Completion (Estimated)

June 30, 2038

Study Completion (Estimated)

June 30, 2038

Study Registration Dates

First Submitted

September 12, 2019

First Submitted That Met QC Criteria

November 20, 2019

First Posted (Actual)

November 22, 2019

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

January 30, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • COAV101A12001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

IPD Plan Description

Authorship of planned manuscripts for submission to medical journals shall be determined in accordance with the International Committee of Medical Journal Editors (ICMJE) Uniform Requirements for Manuscripts Submitted to Biomedical Journals (www.icmje.org).

The physician agrees that if the physician is part of a multi-center registry, the physician shall coordinate in advance any intended disclosure of the results of the registry with Novartis Gene Therapies to ensure that the results of individual physicians are not published or presented before those of the multi-center registry, unless otherwise agreed to in writing by Novartis Gene Therapies.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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