Ignite Creation Date:
2024-05-06 @ 5:55 PM
Last Modification Date:
2024-10-26 @ 2:38 PM
Study NCT ID:
NCT05475691
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-03-08
First Post:
2022-07-19
Brief Title:
Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America
Sponsor:
Hospital Israelita Albert Einstein
Organization:
Hospital Israelita Albert Einstein
Study Overview
Official Title:
Protocol-LATAM RegistrAME Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America - a Regional Registry
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-03
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:
RegistrAME
Brief 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 contextThe 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
Detailed Description:
This is a retrospective and prospective multicenter non-randomized registry in Latin America The variables included in the RegistrAME registry are based on the core items defined by the TREAT-NMD for SMA registries and the RegistrAME steering committee consensus Items such as demographic characteristics date of genetic test result clinical diagnosis functional status and pulmonary function among others are included in RegistrAME The RegistrAME registry will allow the inclusion of retrospective clinical data in those centers where natural history studies of for spinal muscular atrophy are currently being conducted RegistrAME will also offer a standardized structure for prospective data collection in all centers The current aim of this registry is to include centres in LATAM meeting the structural and personnel requirements for performing the planned regular registry-related investigations These reference centers in LATAM Latin America will be selected from COEs which 1 have the potential to enroll and make the proper patient follow up 2 have experience in treating SMA and 3 have experience in conducting clinical trials An electronic Case Report Form e-CRF will be created by the ARO Academic Research Organization from Hospital Albert Einstein using REDCap Research Electronic Data Capture The electronic Case Report Form e-CRF created to meet international standards for data protection and quality management and to harmonize the platform with those currently used by other countries No interventions will be performed in this study the RegistrAME is observational study non-randomized international multicenter study Registration of patients in Latin America Data collection aims to gather as much information as possible regarding the clinical profile of patients interventions performed in the routine of care and clinical evolution over time Real World Evidence-RWE After confirmation of eligibility and informed consent patients will undergo medical evaluation and then retrospective data collection when possible and limited to 6 months before the patient inclusion in the study baseline data and continuation of longitudinal data collection will be started Data entry is planned to be performed every carried out at intervals of 4 to 6 months according to the type of SMA depending on the regular healthcare planning of each clinical site The study will assess disease progression both the natural history of the disease and the effectiveness of different SMA specific drug treatments on patient outcomes Duration of disease survival with or without ventilatory support motor function pulmonary function developmental milestones achieved growth parameters orthopedic symptoms functional assessments CHOP-INTEND Childrens Hospital of Philadelphia Infant Test of Neuromuscular Disorders HINE-2 Hammersmith Infant Neuromuscular Examination - session 2 HFMSE Hammersmith Motor Functional Scale Expanded RULM Revised Upper Limb Module and 6MWT The six minute walking test will be analyzed depending on the functional capacity of the patients and 5q SMA type over time
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