Answer ALS: Individualized Initiative for ALS Discovery

Answer ALS -Creation of a Large Bio-repository of iPS Cells, Cell Lines, and Bio-fluid Samples, Combined With Clinical Information to Rapidly Advance Therapeutics That Could Treat ALS

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT02574390

Acronym

AnswerALS

Enrollment

1049

Registered

2015-10-12

Start date

2015-12-31

Completion date

2020-01-01

Last updated

2020-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Amyotrophic Lateral Sclerosis, Asymptomatic ALS Gene Carriers, Flail Arm ALS, Healthy Controls, Monomelic Amyotrophy, Motor Neuron Disease, Primary Lateral Sclerosis, Progressive Muscular Atrophy

Keywords

ALS, stem cell, biomarker, disease progression

Brief summary

Creation of a large repository of induced pluripotent stem cells (iPSC), bio-fluid samples (blood and spinal fluid (optional)), and cell lines for ALS gene identification. This will be combined carefully with collected measures of the pattern of the symptoms people with ALS have and how these change over time. People with other motor neuron diseases and healthy controls will be included as comparisons

Detailed description

Patients will have 5 study visits; screening, 3, 6, 9 and 12 months. There will be a one year post-participation follow-up period, during which they will receive an email or phone call interview once every 3 months. During the first year, samples will be collected, breathing, muscle strength, spasticity, general function and cognitive behavior will be assessed. Healthy controls will have 2 study visits during which blood samples will be collected and questionnaires given.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to 100 Years

Healthy volunteers

Yes

Inclusion criteria

* Participants with familial or sporadic ALS diagnosed as possible, laboratory-supported probable, probable or definite according to the World Federation of Neurology (WFN) El Escorial criteria, Primary Lateral Sclerosis Flail Arm ALS, Progressive Muscular Atrophy, Monomelic Amyotrophy, Motor Neuron Disease, Asymptomatic ALS Gene Carriers * Participants who are ages 18-100, inclusive.

Exclusion criteria

* Participants with Spinal-Bulbar Muscular Atrophy * Known diagnosis of HIV/AIDS, Hepatitis B, or Hepatitis C.

Design outcomes

Primary

Measure	Time frame	Description
ALS Functional Rating Scale-Revised (ALSFRS-R)	once every 3 months for one year	12 questions about patient's ability to function in certain activities of daily living. Each question is out of 4 with 4 being normal and 0 being completely impaired.
ALS Cognitive Behavioral Scale (ALS-CBS)	once every 3 months for one year	short measure of cognition and behavior in patients with ALS. The cognitive portion consists of 8 tasks with a perfect score being 20. The behavioral portion measures changes in personality and behavior since the onset of ALS symptoms as well as mood, pseudobulbar affect and fatigue and is completed by a family member or caregiver. A normal score is 45.
Slow Vital Capacity (SVC)	once every 3 months for one year	measurement of the maximum amount of air that can be exhaled following a deep breath.
Strength Testing with Hand Held Dynamometer (HHD)	once every 3 months for one year	muscle strength testing performed on upper and lower limbs, ankles, wrists and fingers using a small hand held device. These measurements are followed over time and compared to measure decline.

Countries

United States

Outcome results