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TRIAL READY (Clinical Trial Readiness)

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: NCT03912987
Recruitment Status : Recruiting
First Posted : April 12, 2019
Last Update Posted : September 16, 2020
Sponsor:
Collaborators:
National Institutes of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
Michael Benatar, University of Miami

Study Description
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Brief Summary:
This study, being conducted under the auspice of the CReATe Consortium, will enroll patients with ALS and related disorders as well as healthy controls, with the goal of facilitating clinical validation of leading biological-fluid based biomarker candidates that may aid therapy development for patients with ALS and related disorders.

Condition or disease
Amyotrophic Lateral Sclerosis Frontotemporal Dementia ALS-Frontotemporal Dementia Primary Lateral Sclerosis Progressive Muscular Atrophy

Detailed Description:
This multi-center study aims to clinically validate leading biological-fluid-based biomarker candidates as potential prognostic and pharmacodynamic biomarkers that have the potential to facilitate therapy development for patients with ALS and related disorders. Biomarker candidates that will be considered include: urinary p75 neurotrophin receptor extracellular domain (p75ECD), blood and cerebrospinal fluid (CSF) phosphorylated neurofilament heavy (pNfH), blood and CSF neurofilament light (NfL) and, in the population with a C9orf72 hexanucleotide repeat expansion, peripheral blood mononuclear cell (PBMC) and CSF levels of the dipeptide repeat protein poly(GP). In pursuit of these goals, the CReATe Consortium is already collecting longitudinal biological samples from patients with ALS and related disorders through the ongoing Phenotype-Genotype-Biomarker (PGB) study. TRIAL READY aims to identify additional patients with the C9orf72 hexanucleotide repeat expansion mutation (HREM), the most common genetic cause of ALS, who may be further followed through the PGB study. This study will also enroll and longitudinally evaluate a cohort of age- and gender-match healthy controls.
Study Design
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Study Type : Observational
Estimated Enrollment : 610 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: TRIAL READY (Clinical Trial Readiness)
Actual Study Start Date : January 22, 2019
Estimated Primary Completion Date : January 2022
Estimated Study Completion Date : January 2022

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort
Affected
Affected with ALS or a related disorder, including ALS-FTD, FTD, PLS, and PMA.
Healthy Controls
Those never diagnosed with and not at particular risk for developing ALS or a related disorder.


Outcome Measures
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Primary Outcome Measures :
  1. Longitudinal trajectories (and variability) of leading biological-fluid biomarker candidates. [ Time Frame: 12 months ]
    In a population of patients with ALS and related disorders who would meet typical clinical trial eligibility, this study aims to define the natural history (and variability) of urinary neurotrophin receptor extracellular domain (p75ECD); blood and cerebrospinal fluid (CSF) neurofilament light (NfL) and phosphorylated neurofilament heavy (pNfH); and among patients with the C9orf72 hexanucleotide repeat expansion mutation (HREM), CSF and peripheral blood mononuclear cell (PBMC) poly(GP).


Secondary Outcome Measures :
  1. Prognostic utility of leading biological-fluid biomarker candidates. [ Time Frame: 12 months ]
    In a population of patients with ALS and related disorders who would meet typical clinical trial eligibility, quantify the prognostic value that baseline biomarker levels add to readily available clinical factors that are known to predict prognosis.


Biospecimen Retention:   Samples With DNA
DNA, serum, plasma, buffy coat, urine and CSF

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Patients with ALS or a related neurodegenerative disorder, including FTD, ALS-FTD, PLS and PMA. Individuals never diagnosed with and not at particular risk of developing ALS or a related disorder.
Criteria

Inclusion Criteria:

  • Member of at least one of the following categories:

    1. Individuals with a clinical diagnosis of ALS or a related disorder, including FTD, ALS-FTD, PLS, and PMA.
    2. Individuals never diagnosed with and not at particular risk of developing ALS or a related disorder.
  • Able and willing to comply with relevant procedures.

Exclusion Criteria:

  • A condition or situation which, in the PI's opinion, could confound the study finding or may interfere significantly with the individual's participation and compliance with the study protocol. This includes (but is not limited to) neurological, psychological and/or medical conditions.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03912987


Contacts
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Contact: Michael Benatar, MBChB, MS, DPhil 305-243-4015 projectcreate@med.miami.edu
Contact: Anne Cooley 844-837-1031 projectcreate@med.miami.edu

Locations
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United States, Florida
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact: Kristina Reyes    305-243-4997    projectcreate@miami.edu   
Principal Investigator: Michael Benatar, MBChB, MS, DPhil         
United States, Kansas
University of Kansas Recruiting
Kansas City, Kansas, United States, 66160
Contact: Collin Gerringer    913-574-0008    cgerringer@kumc.edu   
Principal Investigator: Jeffrey Statland, MD         
United States, Pennsylvania
University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Luis Rosario    215-898-3081    luis.rosario@pennmedicine.upenn.edu   
Principal Investigator: Corey McMillan, PhD         
Sponsors and Collaborators
University of Miami
National Institutes of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
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Principal Investigator: Michael Benatar University of Miami
More Information
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Responsible Party: Michael Benatar, Chief, Neuromuscular Division, University of Miami
ClinicalTrials.gov Identifier: NCT03912987    
Other Study ID Numbers: 20180908
U01NS107027 ( U.S. NIH Grant/Contract )
First Posted: April 12, 2019    Key Record Dates
Last Update Posted: September 16, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Dementia
Muscular Atrophy
Frontotemporal Dementia
Aphasia, Primary Progressive
Pick Disease of the Brain
Muscular Atrophy, Spinal
Pathologic Processes
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
 Neurodegenerative Diseases
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Diseases
Spinal Cord Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Frontotemporal Lobar Degeneration
Aphasia
Speech Disorders
Language Disorders
Communication Disorders