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Amyotrophic Lateral Sclerosis (ALS) Families Project

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03865420
Recruitment Status : Recruiting
First Posted : March 6, 2019
Last Update Posted : October 12, 2020
Sponsor:
Information provided by (Responsible Party):
Columbia University

Tracking Information
First Submitted Date February 25, 2019
First Posted Date March 6, 2019
Last Update Posted Date October 12, 2020
Actual Study Start Date September 11, 2018
Estimated Primary Completion Date September 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: March 5, 2019)
Time to emergence of symptoms attributable to gene mutations [ Time Frame: Up to 3 years ]
Emergence of symptoms will defined by the development of any of the following: a) any weakness on neurological examination, b) evidence of nerve loss on electromyography (EMG)-nerve conduction studies, or or c) evidence of cognitive impairment on the ALS-Cognitive Behavioral Scale (ALS-CBS).
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Amyotrophic Lateral Sclerosis (ALS) Families Project
Official Title Amyotrophic Lateral Sclerosis (ALS) Families Project
Brief Summary This program provides family members of individuals with familial ALS the opportunity to contribute to research focused on learning more about why motor neuron degeneration begins and how or why it progresses. This study provides genetic counseling and testing to help participants understand and manage their risk and determine if they want to learn their genetic status. This study will follow unaffected ALS gene mutation carriers on an annual basis to gather essential information that will ultimately help researchers develop novel therapies for the prevention and treatment of ALS.
Detailed Description

Approximately 10% of people with amyotrophic lateral sclerosis (ALS), or Lou Gehrig's Disease, have a family history of ALS or a related condition called frontotemporal dementia (FTD). In most of these familial cases, and a significant number of "sporadic" patients with no family history, a mutation is present in one of a growing number of genes that have been associated with ALS and/or FTD.

The ALS Families Project will study unaffected carriers of ALS/FTD-associated gene mutations to investigate the first steps in the disease process that leads to motor neuron degeneration, with the goal of identifying early disease targets and points of intervention to slow or stop disease onset and progression.

Unaffected individuals who have either a family member with a known ALS/FTD-associated gene mutation or have a strong family history of ALS and FTD are invited to participate in the ALS Families Project. For those who enroll, research visits will occur every 6-12 months.

Study Type Observational
Study Design Observational Model: Family-Based
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
DNA, RNA, plasma, serum and Peripheral blood mononuclear cells (PBMCs)
Sampling Method Non-Probability Sample
Study Population Unaffected individuals who have either a family member with a known ALS/FTD-associated gene mutation or have a strong family history of ALS and FTD.
Condition ALS
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: March 5, 2019)
60
Original Estimated Enrollment Same as current
Estimated Study Completion Date September 2022
Estimated Primary Completion Date September 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Men or women of any race or ethnicity aged 18 or older
  • No symptoms of ALS or fronto-temporal dementia at enrollment
  • Have a first degree relative who had an ALS-spectrum diagnosis with a confirmed ALS-spectrum gene mutation; or already have had genetic testing and have tested positive for an ALS-spectrum gene mutation.
  • Willing to undergo genetic testing with option of whether or not to learn results
  • Willing to travel to Columbia University Irving Medical Center (CUIMC) every 6-24 months for study procedures
  • Capable of providing informed consent and following study procedures, or has a legally authorized representative who is able to consent for the subject.

Exclusion Criteria:

  • Known HIV
  • Known hepatitis B
  • Known hepatitis C
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 105 Years   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Elizabeth Harrington, MS, CGC 347-852-5315 ALSFamiliesProject@cumc.columbia.edu
Contact: Matthew Harms, MD
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03865420
Other Study ID Numbers AAAR7275
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Columbia University
Study Sponsor Columbia University
Collaborators Not Provided
Investigators
Principal Investigator: Matthew Harms, MD Columbia University
PRS Account Columbia University
Verification Date October 2020