Theophylline for Treatment of Pseudohypoparathyroidism

Last updated: October 29, 2024
Sponsor: Ashley Shoemaker
Overall Status: Active - Enrolling

Phase

2

Condition

Parathyroid Disorders

Mccune-albright Syndrome

Hypoparathyroidism

Treatment

Theophylline ER

Clinical Study ID

NCT04240821
IND 133013 Extension Study
  • Ages 2-99
  • All Genders
  • Accepts Healthy Volunteers

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Successful completion of the randomized clinical trial "Phase 2 Study ofTheophylline Treatment of Pseudohypoparathyroidism IND 133103 (11/1/2016)".

Exclusion

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 upperlimit 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. Current use of medications known to effect theophylline levels

  9. History of hypersensitivity to theophylline or other medication components

  10. Unable to comply with study procedures in the opinion of the investigator

Study Design

Total Participants: 34
Treatment Group(s): 1
Primary Treatment: Theophylline ER
Phase: 2
Study Start date:
May 22, 2020
Estimated Completion Date:
December 31, 2026

Study 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.

Connect with a study center

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37212
    United States

    Site Not Available

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