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Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old
Sponsor: Vanderbilt University Medical Center
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.
Official title: Phase 2 Study of Theophylline Treatment for Pseudohypoparathyroidism
Key Details
Gender
All
Age Range
2 Years - 12 Years
Study Type
INTERVENTIONAL
Enrollment
34
Start Date
2020-07-13
Completion Date
2026-12-31
Last Updated
2026-02-23
Healthy Volunteers
No
Conditions
Interventions
Theophylline
oral theophylline
Placebo
Placebo capsule or elixir
Locations (1)
Vanderbilt University Medical Center
Nashville, Tennessee, United States