Amyotrophic Lateral Sclerosis (ALS) Families Project

ALS Clinical Trial

Official title:

Amyotrophic Lateral Sclerosis (ALS) Families Project

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03865420
• Other study ID #: AAAR7275
• Status: Recruiting
• Start date: September 11, 2018
• Est. completion date: September 2024

Study information

• Verified date: April 2024
• Source: Columbia University
• Contact: Elizabeth Harrington, MS, CGC
• Phone: 347-852-5315
• Email: ALSFamiliesProject[@]cumc.columbia.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 60
• Est. completion date: September 2024
• Est. primary completion date: September 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 105 Years

Eligibility

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

Related Conditions & MeSH terms

• ALS
• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease

Locations

Country	Name	City	State
United States	Columbia University	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Columbia University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time to emergence of symptoms attributable to gene mutations	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).	Up to 3 years