NCT00466349

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
3,000

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started May 1986

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 1986

Completed
21 years until next milestone

First Submitted

Initial submission to the registry

April 24, 2007

Completed
3 days until next milestone

First Posted

Study publicly available on registry

April 27, 2007

Completed
10.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2018

Completed
Last Updated

August 7, 2018

Status Verified

August 1, 2018

Enrollment Period

31.7 years

First QC Date

April 24, 2007

Last Update Submit

August 6, 2018

Conditions

Muscular Atrophy, Spinal

Keywords

SMA Type ISMA Type IISMA Type IIISMNWerdnig-Hoffman DiseaseSpinal Muscular AtrophyRegistrySMA Type IV

Outcome Measures

Primary Outcomes (1)

  • Study is a patient registry

    unlimited

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Indiana University School of Medicine

Indianapolis, Indiana, 46202, United States

Location

MeSH Terms

Conditions

Muscular Atrophy, SpinalSpinal Muscular Atrophies of Childhood

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular DiseasesHeredodegenerative Disorders, Nervous SystemGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Tatiana Foroud, PhD

    Indiana University School of Medicine

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
FAMILY BASED
Time Perspective
OTHER
Target Duration
50 Years
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Department Chair

Study Record Dates

First Submitted

April 24, 2007

First Posted

April 27, 2007

Study Start

May 1, 1986

Primary Completion

January 1, 2018

Study Completion

January 1, 2018

Last Updated

August 7, 2018

Record last verified: 2018-08

Locations

US(1)