Trials / Enrolling By Invitation
Enrolling By InvitationNCT02327845
Phenotype, Genotype & Biomarkers in ALS and Related Disorders
- Status
- Enrolling By Invitation
- Phase
- —
- Study type
- Observational
- Enrollment
- 700 (estimated)
- Sponsor
- University of Miami · Academic / Other
- Sex
- All
- Age
- —
- Healthy volunteers
- Accepted
Summary
The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders.
Detailed description
This study will recruit patients with ALS, ALS-FTD, PLS, HSP, and PMA, with a focus on incident cases. Patients with both familial and sporadic forms of these diseases will be enrolled and followed longitudinally using a standardized set of evaluations. Biological samples (blood, urine, CSF) will be collected from all study participants, and will be used for biomarker discovery and validation. Family members of affected individuals may also be enrolled and asked to contribute DNA and biological samples to aid genetic and biomarker discovery.
Conditions
- Amyotrophic Lateral Sclerosis
- Frontotemporal Dementia
- Primary Lateral Sclerosis
- Hereditary Spastic Paraplegia
- Progressive Muscular Atrophy
- Multisystem Proteinopathy
Timeline
- Start date
- 2015-04-01
- Primary completion
- 2026-07-01
- Completion
- 2026-08-01
- First posted
- 2014-12-30
- Last updated
- 2025-07-03
Locations
15 sites across 3 countries: United States, Germany, South Africa
Source: ClinicalTrials.gov record NCT02327845. Inclusion in this directory is not an endorsement.