Researchers are looking for people to participate in a clinical study designed to help scientists better understand the relationship between the phenotype (disease characteristics) and genotype (genetic makeup) of ALS (amyotrophic lateral sclerosis) and related diseases including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD), among others. In addition, the researchers plan to use data from the study to develop and validate biomarkers that may help inform therapy development for these disorders.
The CReATe Consortium’s PGB study is a longitudinal observational study — a type of study in which investigators collect information (including biological samples) at multiple time points from a group of participants according to a protocol or research plan. Unlike a clinical trial, the PGB study does not offer an experimental treatment or intervention. But genetic testing is performed and participants may choose to learn the results.
Each participant’s involvement in the study is expected to last 18-24 months, and requires participants to make a visit to one of the study sites every three to six months. At each visit, blood, urine and, if the participant is willing, cerebrospinal fluid samples will be collected, and muscle strength and respiratory function will be tested. Several questionnaires will also be completed, including the ALSFRS-R (ALS Functional Rating Scale-Revised), which measures the participant’s independence and abilities associated with various activities of daily living.
In order to be eligible to participate, individuals must have a diagnosis of ALS or a related neurodegenerative disorder, be able and willing to comply with study procedures, and meet other criteria.
Study sites are located across the U.S. (in California, Florida, Iowa, Kansas, Minnesota, North Carolina, Ohio, Pennsylvania, Texas, and Virginia so far, with others potentially added in the future).Locations of international sites currently include Alberta, Canada and Cape Town, South Africa.
To learn more about the PGB study, including complete inclusion and exclusion criteria, visit ClinicalTrials.gov and enter NCT02327845 into the search box, or contact Sumaira Hussain, CReATe Project Manager, at 844-837-1031 or projectcreate@med.miami.edu. You can also learn more about the CReATe Consortium at www.rdcrn.org/create, and sign up for CReATe Connect at www.rdcrn.org/createconnect to receive updates and to participate in various educational and research activities.
