TRIAL READY (Clinical Trial Readiness)

• Sponsor: University of Miami
• 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

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

• 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

Groups and Cohorts

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

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

• 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

Contacts

Contact: Michael Benatar, MBChB, MS, DPhil; 305-243-4015; projectcreate@med.miami.edu

Contact: Anne Cooley; 844-837-1031; projectcreate@med.miami.edu

Locations

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

Sponsors and Collaborators

University of Miami

National Institutes of Health (NIH)

National Institute of Neurological Disorders and Stroke (NINDS)

More Information

• Responsible Party: Michael Benatar, Chief, Neuromuscular Division, University of Miami
• ClinicalTrials.gov Identifier: NCT03912987 History of Changes
• Other Study ID Numbers: 20180908; U01NS107027 ( U.S. NIH Grant/Contract )
• First Posted: April 12, 2019
• Last Update Posted: April 12, 2019
• Last Verified: April 2019

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

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; Atrophy; Pathological Conditions, Anatomical; Neurodegenerative Diseases; Neuromuscular Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Signs and Symptoms; Frontotemporal Lobar Degeneration; Aphasia; Speech Disorders; Language Disorders