Ignite Creation Date:
2024-05-05 @ 5:29 PM
Last Modification Date:
2024-10-26 @ 9:32 AM
Study NCT ID:
NCT00466349
Status:
COMPLETED
Last Update Posted:
2018-08-07
First Post:
2007-04-24
Brief Title:
International SMA Patient Registry
Sponsor:
Indiana University School of Medicine
Organization:
Indiana University
Study Overview
Official Title:
International Spinal Muscular Atrophy Patient Registry
Status:
COMPLETED
Status Verified Date:
2018-08
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 is a registry of individuals affected by Spinal Muscular Atrophy SMA andor family members of individuals affected by SMA The purpose of the registry is to allow researchers studying the biological basis of SMA and potential therapies in SMA access to individuals interested in participating in research andor experimental therapies The International SMA Patient Registry is supported by CureSMA
Detailed Description:
This project is supported by CureSMA httpcuresmaorg
The Registry functions as a liaison between patients and families interested in participating in research and researchers interested in studying SMA The Registry contains information from 2500 families and over 3000 individuals with SMA from all over the world and continues to grow
The Registry has helped recruit participants for clinical trials and has provided data for important SMA research studies The Registry helps centralize information on this rare genetic disease provides families a way to learn about research studies and provides researchers a way to find research participants
Individuals and families affected by SMA are invited to join the Registry Participants are asked to complete questionnaires about the symptoms treatment medications and other experiences with SMA Visit the Participant Portal for more information on how to sign up for the Registry
Participant information is stored in a secure database Researchers who are interested in studying SMA can request two types of data from the Registry de-identified information and identifiable information De-identified information does not contain any names or personal identifiers and can be given to researchers without having to contact Registry families Identifiable information includes information that can identify you and will never be released without getting your written permission to do so Identifiable information includes data such as names and dates of birth Some researchers may also request contact with families to obtain specific information or to request participation in a research study In these instances the Registry will contact each potential participant to ask if they are willing to share their identifiable information for a research project
The Registry recognizes the importance of global collaboration Working together with researchers from all over the world is very important in the investigation of SMA In 2008 the Registry joined the group Translational Research in Europe for the Assessment and Treatment of Neuromuscular Diseases TREAT-NMD in a global collaboration to further the research goals of the neuromuscular disease community TREAT-NMD has developed a global database that compiles deidentified data transferred from participating registries around the world The main objective of the TREAT-NMD database is to assess the feasibility of clinical trials to facilitate the planning of clinical trials and to support the enrollment of patients in clinical trials This international database will also be used to answer questions regarding the prevalence of particular neuromuscular disorders and assessing the support of other activities such as assessing standards of care and diagnosis
The Registry functions as a liaison between patients and families interested in participating in research and researchers interested in studying SMA The Registry contains information from 2500 families and over 3000 individuals with SMA from all over the world and continues to grow
The Registry has helped recruit participants for clinical trials and has provided data for important SMA research studies The Registry helps centralize information on this rare genetic disease provides families a way to learn about research studies and provides researchers a way to find research participants
Individuals and families affected by SMA are invited to join the Registry Participants are asked to complete questionnaires about the symptoms treatment medications and other experiences with SMA Visit the Participant Portal for more information on how to sign up for the Registry
Participant information is stored in a secure database Researchers who are interested in studying SMA can request two types of data from the Registry de-identified information and identifiable information De-identified information does not contain any names or personal identifiers and can be given to researchers without having to contact Registry families Identifiable information includes information that can identify you and will never be released without getting your written permission to do so Identifiable information includes data such as names and dates of birth Some researchers may also request contact with families to obtain specific information or to request participation in a research study In these instances the Registry will contact each potential participant to ask if they are willing to share their identifiable information for a research project
The Registry recognizes the importance of global collaboration Working together with researchers from all over the world is very important in the investigation of SMA In 2008 the Registry joined the group Translational Research in Europe for the Assessment and Treatment of Neuromuscular Diseases TREAT-NMD in a global collaboration to further the research goals of the neuromuscular disease community TREAT-NMD has developed a global database that compiles deidentified data transferred from participating registries around the world The main objective of the TREAT-NMD database is to assess the feasibility of clinical trials to facilitate the planning of clinical trials and to support the enrollment of patients in clinical trials This international database will also be used to answer questions regarding the prevalence of particular neuromuscular disorders and assessing the support of other activities such as assessing standards of care and diagnosis
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None