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Infants With Spinal Muscular Atrophy Type I

NCT01547871 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 4

Last updated 2019-12-12

No results posted yet for this study

Summary

Background:

\- Spinal muscular atrophy type 1 (SMA 1) causes severe muscle weakness and problems with eating and breathing. The symptoms begin in infancy, and children affected with SMA 1 often die in early childhood. Researchers want to collect information on how SMA symptoms progress in first two years.

Objectives:

\- To study how the symptoms of SMA 1 progress in infants and children.

Eligibility:

\- Infants and children with SMA 1 born on or after January 1, 2007.

Design:

* Researchers will review the child s medical records and talk with parents by telephone.
* For children who are under 2 years of age, the researchers will review the child s medical records and speak with you on telephone every 2-4 months. Phone calls with parents will take about 10 minutes and will involve questions about symptoms of SMA 1. Children will be followed until age 2.- Researchers are also interested in looking at medical records of children who are no longer alive or who are more than 2 years of age.

Parents or children do not have to come to the NIH. They will provide consent to view these records, and information over the telephone.

\- No treatment or care will be provided as part of this study.

Conditions

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    lead NIH

Principal Investigators

  • Minal J Bhanushali, M.D. · National Institute of Neurological Disorders and Stroke (NINDS)

Eligibility

Min Age
1 Year
Max Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-02-07
Completion
2016-04-14

Countries

  • United States

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01547871 on ClinicalTrials.gov