Spruce Biosciences which is a clinical-stage biotechnology company developing novel therapies for rare endocrine disorders asserted the launch of its Congenital Adrenal Hyperplasia Natural History Study.
The study is set to enable patients afflicted with Congenital adrenal hyperplasia, a rare disease to contribute their experiences to further the knowledge of the disease encompassing management and treatment. The study is being launched in line with the World Rare Disease Day. The day marks the observance and awareness of rare diseases and future implications.
Although CAH is part of the newborn screening program and highly identifiable , currently no FDA-approved therapies exist for CAH. The CAH Natural History Study is designed to collect health-related patient information and serve as a resource to advance education, advocacy, and research initiatives, as well as serve as a recruitment database that can be used to address the difficulties associated with enrolling patients into clinical trials necessary to advance the development of new therapeutics.
It is to be noted that CAH is a rare endocrine disorder caused by genetic mutations resulting in the inability to produce the critical ‘stress’ hormone, cortisol. The most common form of CAH, 21 hydroxylase deficiency, affects approximately 1 in 10,000 to 15,000 people in the United States.
Dr. Alexis Howerton, CEO, Spruce Biosciences said “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.”
Spruce is currently conducting a Phase 2 clinical trial to assess the safety and efficacy of SPR001 in adults with classic CAH