Clinical trial NCT03912987: TRIAL READY (Clinical Trial Readiness)
Official title: TRIAL READY (Clinical Trial Readiness)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Amyotrophic Lateral Sclerosis
Frontotemporal Dementia
ALS-Frontotemporal Dementia
Primary Lateral Sclerosis
Progressive Muscular Atrophy
Lead sponsor: University of Miami
Sponsor class: OTHER
Collaborators: name: National Institutes of Health (NIH)
name: National Institute of Neurological Disorders and Stroke (NINDS)
First posted: 2019-04-12
Enrollment: 610
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

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.

Eligibility 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.

Primary outcomes:
measure: Longitudinal trajectories (and variability) of leading biological-fluid biomarker candidates.
timeFrame: 12 months
Secondary outcomes:
measure: Prognostic utility of leading biological-fluid biomarker candidates.
timeFrame: 12 months
Other outcomes:
Locations:
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Kansas City
country: United States
facility: University of Kansas
state: Kansas
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
Study officials:
affiliation: University of Miami
name: Michael Benatar
role: PRINCIPAL_INVESTIGATOR
References: