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Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America

NCT05475691 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 361

Last updated 2024-03-08

No results posted yet for this study

Summary

The natural history of SMA patients has changed, due to the improvements in treatment and technological advances. The systematic collection of data from routine clinical practice in multiple Latin American countries, harmonized to an internationally aligned core data set, is important to advancing the understanding the natural history of disease in the region and the influence of different drug treatments on patient outcomes. These data are critical to improving the care of these patients. So far, clinical trials regarding therapeutic approaches for SMA patients only cover a subgroup of the broad spectrum of severity of SMA. Thus, there is a strong need to monitor the full range of treated and untreated SMA patients in a real-world context.The aim of this study is to set up a regional healthcare provider (HCP) entered registry. The planned SMA registry will provide an online platform to collect longitudinal data on SMA patients across Latin America to achieve a better understanding of the clinical characteristics of SMA patients, the natural history of the disease, the use of DMTs and patients' outcomes, as well as to support further research projects and regional data generation.

Conditions

Sponsors & Collaborators

  • Biogen

    collaborator INDUSTRY

  • Hospital Israelita Albert Einstein

    lead OTHER

Principal Investigators

  • Otávio Berwanger, PhD · Hospital Albert Einstein

  • Henrique Fonseca, PhD · Hospital Albert Einstein

Eligibility

Min Age
15 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-17
Primary Completion
2024-08-30
Completion
2025-01-30

Countries

  • Argentina
  • Brazil
  • Chile
  • Colombia
  • Mexico
  • Uruguay

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05475691 on ClinicalTrials.gov