International SMA Patient Registry

Muscular Atrophy, Spinal Clinical Trial

Official title:

International Spinal Muscular Atrophy Patient Registry

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00466349
• Other study ID #: 0202-03
• Status: Completed
• Start date: May 1986
• Est. completion date: January 2018

Study information

• Verified date: August 2018
• Source: Indiana University
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

This is a registry of individuals affected by Spinal Muscular Atrophy (SMA) and/or 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 and/or experimental therapies. The International SMA Patient Registry is supported by CureSMA.

Description:

This project is supported by CureSMA (http://curesma.org/).

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 2,500 families and over 3,000 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3000
• Est. completion date: January 2018
• Est. primary completion date: January 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Individuals or family members of individuals who have been diagnosed with SMA.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal

Locations

Country	Name	City	State
United States	Indiana University School of Medicine	Indianapolis	Indiana

Sponsors (2)

Lead Sponsor	Collaborator
Indiana University School of Medicine	Cure SMA

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Study is a patient registry		unlimited

External Links

•   website includes information on SMA, the International SMA Patient Registry (including forms to join), and related SMA links