Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old
Status:
Recruiting
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 and resistance to multiple hormones. This phase 2
clinical trial and open-label extension study will test the efficacy of theophylline, a
phosphodiesterase inhibitor, in pseudohypoparathyroidism. We hypothesize that theophylline
will cause weight loss, slow the rate of growth plate closure and decrease hormone resistance
in children.