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ACTIVE NOT RECRUITING
NCT04551170
PHASE2

Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

Sponsor: Vanderbilt University Medical Center

View on ClinicalTrials.gov

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

Interventions

DRUG

Theophylline

oral theophylline

DRUG

Placebo

Placebo capsule or elixir

Locations (1)

Vanderbilt University Medical Center

Nashville, Tennessee, United States