Study of Theophylline Treatment in Children and Young Adults with Pseudohypoparathyroidism

What this Trial Is About

Researchers are evaluating the effects of the drug theophylline in 100 individuals aged 5 to 21 who have Pseudohypoparathyroidism PHP, a condition characterized by resistance to hormones that use cAMP signaling. The study explores whether theophylline, which increases cAMP levels by inhibiting phosphodiesterase, can reduce hormone resistance and influence bone maturation, brain function, and metabolism in these patients. This is an open-label intervention study focusing on the off-label use of theophylline in PHP. Participants will receive theophylline twice daily for 52 weeks. The dosage will be adjusted to maintain a peak blood level between 10 and 15 mcgmL. This single-arm study does not include a placebo group, and all subjects will be given theophylline to assess its impact on their condition. During the study, participants will be monitored for changes in bone age over the 52-week treatment period. Assessments will likely include clinical evaluations and laboratory tests to track the drugs effects and safety. The study is sponsored by Massachusetts General Hospital and is expected to continue through April 2032, ensuring long-term observation of outcomes and safety.

Effect of Theophylline in Pseudohypoparathyroidism