Jaclyn Tamaroff
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.
Trial Objectives 1. The primary objective of this study is to evaluate the long-term safety of theophylline in patients with PHP. 2. The secondary objectives of this study are to evaluate changes in BMI, hormone resistance and epiphyseal closure associated with theophylline treatment of patients with PHP. Study Design The proposed study is a single center, 24-month open label extension clinical trial for patients with PHP who complete the randomized clinical trial.
Inclusion Criteria: * 1\. Successful completion of the randomized clinical trial "Phase 2 Study of Theophylline Treatment of Pseudohypoparathyroidism IND 133103 (11/1/2016)". Exclusion Criteria: 1. History of a seizure disorder unrelated to hypocalcemia 2. History of a cardiac arrhythmia (not including bradycardia) 3. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal) 4. Congestive heart failure 5. Current cigarette use or alcohol abuse 6. Pregnancy or intention to become pregnant during the next year 7. Active peptic ulcer disease 8. Curre…
Oral theophylline
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