TRIAL READY (Clinical Trial Readiness)
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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
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Condition or disease |
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Amyotrophic Lateral Sclerosis Frontotemporal Dementia ALS-Frontotemporal Dementia Primary Lateral Sclerosis Progressive Muscular Atrophy |
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 |

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

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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 |
Inclusion Criteria:
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Member of at least one of the following categories:
- Individuals with a clinical diagnosis of ALS or a related disorder, including FTD, ALS-FTD, PLS, and PMA.
- 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.

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
Contact: Michael Benatar, MBChB, MS, DPhil | 305-243-4015 | projectcreate@med.miami.edu | |
Contact: Anne Cooley | 844-837-1031 | projectcreate@med.miami.edu |
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 |
Principal Investigator: | Michael Benatar | University of Miami |
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 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
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 |