NCT04240821

Theophylline for Treatment of Pseudohypoparathyroidism

Open-Label Extension Study of Theophylline for Treatment of Pseudohypoparathyroidism

• Status: Enrolling By Invitation
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 34 (estimated)
• Sponsor: Ashley Shoemaker · Academic / Other
• Sex: All
• Age: 2 Years – 99 Years
• Healthy volunteers: Accepted

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.

Detailed 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.

Conditions

• Pseudohypoparathyroidism
• Pseudohypoparathyroidism Type 1a
• Albright Hereditary Osteodystrophy

Interventions

Type	Name	Description
DRUG	Theophylline ER	Oral theophylline

Timeline

• Start date: 2020-05-22
• Primary completion: 2026-12-31
• Completion: 2026-12-31
• First posted: 2020-01-27
• Last updated: 2026-02-23

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study