Ignite Creation Date:
2024-05-06 @ 2:43 PM
Last Modification Date:
2024-10-26 @ 1:36 PM
Study NCT ID:
NCT04404764
Status:
COMPLETED
Last Update Posted:
2021-04-27
First Post:
2020-05-19
Brief Title:
Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III Observational Study
Sponsor:
Hospital Israelita Albert Einstein
Organization:
Hospital Israelita Albert Einstein
Study Overview
Official Title:
Characterization of the Clinical-epidemiological Profile of Patients With Spinal Muscular Atrophy SMA 5q Types II and III in Follow-up in the Brazilian Unified Public Health System A Cross-sectional Observational Study Registry
Status:
COMPLETED
Status Verified Date:
2021-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 Unified Public Health System SUS The study data will be based on patients medical records from several Brazilian public hospitals which will be defined by the Brazilian Ministry of Health MS
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 the Brazilian Unified Public Health System SUS This study aims to provide baseline data which in the future may be used by the Brazilian Ministry of Health MS to assess the effectiveness of nusinersen The clinical and epidemiological data will be collected from patients medical records such as the score for the Hammersmith Functional Motor Scale - Expanded HFMSE and the Revised Upper Limb Module RULM at baseline the WHO motor milestones at baseline disease duration age at the time of disease diagnosis age at the time of disease screening SMN2 gene copy number history of hospitalizations history and characterization of previous surgical procedures treatment dosage used patient caregivers profile ie a family member or companion who is responsible for taking care of the patient for most of the time Other variables of interest that will also be collected are patients age and gender geographic distribution attending physician expertise and care structure where the patients were treated The data acquisition will be performed using a paper and eletronic CRF Case report Form Written informed consent will be obtained from patients who meet the study elegibility criteria The study will be performed in Brazilian public hospitals centers that are able to provide the treatment with nusinersen under the SUS scope The total sample of patients and number of participating centers will be defined by the MS - SCTIE Secretariat of Science Technology and Strategic Supplies DECIT Department of Science and Technology However the initial estimation is a sample of 100 patients to be included in 10-15 centers
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None