Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study

CompletedOBSERVATIONAL
Enrollment

155

Participants

Timeline

Start Date

May 27, 2020

Primary Completion Date

April 23, 2021

Study Completion Date

April 23, 2021

Conditions
Spinal Muscular Atrophy
Interventions
DRUG

Nusinersen Injectable Product

The patient´s treatment is provided by SUS. The following dosage scheme of nurinersen is the one approved by ANVISA (Brazilian National Health Surveillance Agency): Nusinersen, 12 mg (5 mL) on days 0 (zero), 14 and 28. A fourth dose will be on day 63 with a maintenance dose once every 4 (four) months. The study will NOT have direct influence on the care received by patients. Data on adherence, interventions, hospitalizations, mechanical ventilation, procedures and adverse events will be obtained from the patients´ medical records.

Trial Locations (9)

Unknown

Universidade Federal de Minas Gerais - Hospital das Clínicas, Belo Horizonte

Hospital de Clínicas da Universidade Estadual de Campinas - Unicamp, Campinas

Associação Hospitalar de Prot Infancia Dr Raul Carneiro - Hospital Infantil Pequeno Príncipe, Curitiba

Hospital Infantil Dr. Albert Sabin, Fortaleza

Hospital de Clínicas de Porto Alegre, Porto Alegre

Hospital Universitario Pedro Ernesto, Rio de Janeiro

Instituto de Puericultura e Pediatria Martagão Gesteira da Universidade Federal do rio de Janeiro - UFRJ, Rio de Janeiro

Hospital das Clínicas da Faculdade de Medicina de São Paulo - HCFMUSP, São Paulo

Universidade Federal de São Paulo, São Paulo

All Listed Sponsors
collaborator

Ministry of Health, Brazil

OTHER_GOV

lead

Hospital Israelita Albert Einstein

OTHER

NCT04404764 - Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study | Biotech Hunter | Biotech Hunter