Status:
COMPLETED
Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study
Lead Sponsor:
Hospital Israelita Albert Einstein
Collaborating Sponsors:
Ministry of Health, Brazil
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
6+ years
Brief Summary
This study aims to characterize the clinical-epidemiological profile and baseline characteristics of patients with spinal muscular atrophy (SMA) 5q types II and III in follow-up at the Brazilian Unifi...
Detailed Description
This is a retrospective cross-sectional observational study to characterize the clinical and epidemiological profile of patients with spinal muscular atrophy (SMA) 5q types II and III, in follow-up at...
Eligibility Criteria
Inclusion
- Patient Eligibility Criteria:
- Participants of both sexes, in any age group, who have a clinical diagnosis of SMA 5q type II or type III, in follow-up at SUS, undergoing treatment with nusinersen or not
- Clinical and molecular diagnosis of SMA 5q type II (disease started after 6 months of age), or Clinical and molecular diagnosis of SMA 5q type III (disease started after 18 months of age)
Exclusion
- Refusal to provide written informed consent (either the patient or a legal representative)
- Symptom onset after 19 years of age
- Need for invasive ventilatory support for 16 hours or more per day for more than 21 consecutive days
- Be participating or have participated in another clinical study aimed at specific treatment of SMA 5q other than with the drug nusinersen
- Having undergone treatment with gene therapy
- Caretaker Eligibility Criteria:
- Inclusion Criteria:
- • First-degree family member or companion responsible for taking care of the patient with clinical diagnosis of SMA 5q type II or type III
Key Trial Info
Start Date :
May 27 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
April 23 2021
Estimated Enrollment :
155 Patients enrolled
Trial Details
Trial ID
NCT04404764
Start Date
May 27 2020
End Date
April 23 2021
Last Update
April 27 2021
Active Locations (9)
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1
Universidade Federal de Minas Gerais - Hospital das Clínicas
Belo Horizonte, Brazil
2
Hospital de Clínicas da Universidade Estadual de Campinas - Unicamp
Campinas, Brazil
3
Associação Hospitalar de Prot Infancia Dr Raul Carneiro - Hospital Infantil Pequeno Príncipe
Curitiba, Brazil
4
Hospital Infantil Dr. Albert Sabin
Fortaleza, Brazil