The Clinical Research Center at Marshfield Clinic Research Institute anticipates reaching enrollment target by June in a phase 3 clinical trial for a drug expected to reduce obesity in patients with Bardet-Biedl syndrome (BBS). The Research Institute is the first site to begin enrollment.
BBS is a rare genetic disorder that affects almost every organ system. Common signs of BBS include obesity, impaired vision and chronic kidney disease. It affects less than 3,000 individuals in the U.S. It is inherited in an autosomal recessive manner (both parents passing on one BBS gene to their child) and evolves slowly throughout the first decade of life, although there is a lot of variability.
The research team was able to identify and enroll study participants due to the Clinical Registry Investigating Bardet-Biedl Syndrome, which is the largest worldwide registry examining the long-term health of individuals with BBS. This registry along with a multispecialty clinic are important parts of the Center of Excellence model Marshfield Clinic Health System provides to support patients with BBS.
“If it wasn’t for the BBS Center of Excellence here, we wouldn’t be able to do this,” said Dr. Robert Haws, lead researcher on this trial and director of Clinical Research.
How it works
Our body produces the hormone leptin that is important in telling our brain that we are full and do not need to eat.
In those with BBS, leptin is unable to communicate with the part of the brain that controls appetite. What this drug does is bypass that signal disruption. It then goes to the second messenger and transmits the signal to the brain to stop eating.
In some of the rare genetic obesity disorders such as BBS, previous medications that have been used have been ineffective, but the phase 2 clinical trial showed promising results. Ten people were enrolled and some patients lost more than 100 pounds.
The clinical trial is sponsored by Rhythm, a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity.
Dr. Haws and his team are engaging Dermatology, Phlebotomy, Cardiology, Nutrition Services, Integrated Research and Development Laboratory and Pharmacy at the Health System to support this clinical trial.
“We have a great collaboration of people throughout the Health System that have helped make this possible,” said Dr. Haws. “What this study shows is the phenomenal ability that the Research Institute has to be the world’s leader in rare disease research.”