The Pre-Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study

The recruitment status of this study is unknown because the information has not been verified recently.
Verified April 2012 by University of Miami.
Recruitment status was  Recruiting
Muscular Dystrophy Association
Information provided by (Responsible Party):
Michael Benatar, University of Miami Identifier:
First received: April 24, 2006
Last updated: April 3, 2012
Last verified: April 2012

The investigators aim to recruit unaffected (healthy) people from families with a known genetic mutation in which at least two relatives have been affected with Amyotrophic Lateral Sclerosis (ALS). Our goal is to identify factors, both genetic and environmental, which put people at risk for developing ALS in the future.

Amyotrophic Lateral Sclerosis

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: The Pre-Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study

Resource links provided by NLM:

Further study details as provided by University of Miami:

Primary Outcome Measures:
  • Risk Factors for Progression to familial ALS [ Time Frame: Years ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA

serum, plasma, CSF, DNA, urine, cell lines

Estimated Enrollment: 300
Study Start Date: April 2006
Detailed Description:

Healthy people from familial ALS families with a known genetic mutation will be included in this study. The investigators encourage people who know that they carry the mutation that affects their family as well as those who do not know their genetic status to contact us. Those who wish to participate and to learn the results of genetic testing, may do so after undergoing genetic counseling. It is also possible to participate without learning the results of genetic testing. Participants in the study will travel to Miami (at our expense) on an annual basis.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Healthy individuals who harbor a mutation in a gene associated with ALS.


Inclusion Criteria

  • A member of a family in which at least two relatives have been or currently are affected with ALS.
  • The presence of a genetic change (mutation) in a gene associated with ALS, in a family member.
  • No symptoms to suggest the presence of ALS (i.e. study participants must currently be healthy).
  • Willingness to undergo genetic testing, with the option of whether or not to learn the results.
  • Willingness to travel to Miami annually for in-person study evaluations.

Exclusion Criteria

  • Diagnosis of ALS
  Contacts and Locations
Please refer to this study by its identifier: NCT00317616

Contact: Eliana Reyes 888-413-9315

United States, Florida
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact: Eliana Reyes    888-413-9315   
Sponsors and Collaborators
Michael Benatar
Muscular Dystrophy Association
Principal Investigator: Michael G Benatar, MD, PhD. University of Miami
  More Information

No publications provided

Responsible Party: Michael Benatar, MBChB, MS, DPhil, University of Miami Identifier: NCT00317616     History of Changes
Other Study ID Numbers: 1369-2005
Study First Received: April 24, 2006
Last Updated: April 3, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by University of Miami:
genetic testing
genetic counseling

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes processed this record on April 15, 2014