Rare Daily Staff
Spruce Biosciences said it has launched a natural history study of congenital adrenal hyperplasia, or CAH, a rare endocrine disorder caused by genetic mutations resulting in the inability to produce the critical stress hormone cortisol.
The company said the study will enable patients with congenital adrenal hyperplasia to contribute their experiences to advance knowledge of the disease, including its management and treatment. Patients interested in participating in the CAH Natural History Study can visit the site here.
Spruce expects to collect health-related patient information and have the study serve as a recruitment database that can be utilized to address the challenges associated with enrolling patients into clinical trials and advancing education, advocacy, and research initiatives.
Although CAH is part of the newborn screening program and highly identifiable – unlike many other rare diseases – there are currently no FDA-approved therapies for CAH.
“We believe this research will help transform the collective understanding and approach to treating CAH, helping patients and families get optimal care for generations to come,” said Alexis Howerton, CEO of Spruce Biosciences.
Spruce is currently conducting a mid-stage clinical trial to assess the safety and efficacy of SPR001, its experimental therapeutics for adults with classic CAH.
February 28, 2018
Photo: Alexis Howerton, CEO of Spruce Biosciences