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OpenTrials
Enrolling by Invitation

NCT Number: NCT04240821

Theophylline for Treatment of Pseudohypoparathyroidism

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.

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Key information

Conditions

Pseudohypoparathyroidism Albright Hereditary Osteodystrophy Bone Diseases Bone Diseases, Metabolic Calcium Metabolism Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Musculoskeletal Diseases Nutritional and Metabolic Diseases Pseudohypoparathyroidism Type 1a

Age range

2 year–99 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

About this study

Trial Objectives

  • The primary objective of this study is to evaluate the long-term safety of theophylline in patients with PHP.
  • 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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

  • History of a seizure disorder unrelated to hypocalcemia
  • History of a cardiac arrhythmia (not including bradycardia)
  • Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT >3x upper limit of normal)
  • Congestive heart failure
  • Current cigarette use or alcohol abuse
  • Pregnancy or intention to become pregnant during the next year
  • Active peptic ulcer disease
  • Current use of medications known to effect theophylline levels
  • History of hypersensitivity to theophylline or other medication components
  • Unable to comply with study procedures in the opinion of the investigator

Treatment and study plan

Theophylline ER

Drug

Oral theophylline

Other names: Theophylline elixir

Primary outcomes

  1. Adverse Events

    Time frame: 24 months

    Treatment-emergent adverse events will be compared before and during treatment

Secondary outcomes

  1. BMI

    Time frame: 24 months

    Change in BMI

Sponsors and collaborators

Lead sponsor

Jaclyn Tamaroff

Other

Registry information

Official study title

Open-Label Extension Study of Theophylline for Treatment of Pseudohypoparathyroidism

Important dates

Study start
2020
Primary completion
2026
Study completion
2026
First posted
Jan 27, 2020
Registry last updated
Apr 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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