Theophylline for Treatment of Pseudohypoparathyroidism

Pseudohypoparathyroidism Clinical Trial

Official title: Open-Label Extension Study of Theophylline for Treatment of Pseudohypoparathyroidism

Status Enrolling by invitation

Clinical Trial 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.

Clinical Trial Description

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. ;

Related Conditions & MeSH terms

• Albright Hereditary Osteodystrophy
• Pseudohypoparathyroidism
• Pseudohypoparathyroidism Type 1a

• NCT number: NCT04240821
• Study type: Interventional
• Source: Vanderbilt University Medical Center
• Status: Enrolling by invitation
• Phase: Phase 2
• Start date: May 22, 2020
• Completion date: June 30, 2026