Theophylline for Treatment of Pseudohypoparathyroidism
Status:
Enrolling by invitation
Trial end date:
2025-06-30
Target enrollment:
Participant gender:
Summary
Pseudohypoparathyroidism is a genetic disorder with limited treatment options, characterized
by early-onset obesity, short stature, hormone resistance and cognitive impairment. This
phase 2 clinical trial will test the efficacy of theophylline, a phosphodiesterase inhibitor,
in pseudohypoparathyroidism. We hypothesize that theophylline will cause weight loss, improve
glucose tolerance and decrease hormone resistance in children and young adults.