The Midwest Regional Bone Dysplasia Clinic at UW Health was recently selected as a site for two new clinical trials aimed at helping the growth of children with achondroplasia, the most common form of short stature skeletal dysplasia. Janet Legare, MD, professor, Divisions of Genetics and Metabolism and Developmental Pediatrics and Rehabilitative Medicine, is site principal investigator for the trials.
PROPEL is a six-month observational study of the oral medication infigratinib, which blocks the activity of the overactive FGFR3 that tells the body to slow bone growth in people with achondroplasia. After the initial six-month observational study, children who participated in PROPEL will be enrolled in PROPEL3, a year-long phase-three randomized clinical trial of infigratinib. About 110 patients will be enrolled across all study sites in a 2:1 blinding (two patients will receive the drug for every one patient who does not receive the drug). After the study’s conclusion, the children will be able to remain on the medication (open label extension) until they stop growing. PROPEL3 is sponsored by BridgeBio Pharma’s QED Therapeutics. Recruitment for PROPEL is currently underway.
The Bone Dysplasia Clinic was also chosen as a sentinel site for a clinical trial looking at the effectiveness of a weekly injection of TransconCNP in infants. This therapy slowly releases C-type natriuretic peptide (CNP), which increases bone growth. TransconCNP works via the same mechanism as Voxzogo, the first FDA-approved drug that can help people with achondroplasia grow. TransconCNP is unique because it requires only a weekly injection instead of the daily injection required for Voxzogo use. The Infant TransconCNP trial is sponsored by Ascendis Pharma. It starts recruitment next month, will last 2 years, and will provide open label extension for participants.