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Enrolling by invitation NCT04240821

Theophylline for Treatment of Pseudohypoparathyroidism

Phase II Interventional Pseudohypoparathyroidism Pseudohypoparathyroidism Type 1a Albright Hereditary Osteodystrophy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Theophylline ER.
Who it may be relevant to
Registry conditions: Pseudohypoparathyroidism, Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy. Basic parameters: 2 years — 99 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Open-Label Extension Study of Theophylline for Treatment of Pseudohypoparathyroidism

Overview

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.

Interventions

  • Drug Theophylline ER
    Oral theophylline

Primary outcome measures

  • Adverse Events [Time frame: 24 months]
Secondary outcome measures (1)
  • BMI [Time frame: 24 months]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: Yes

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Vanderbilt University Medical Center — Nashville

Identifiers

NCT: NCT04240821 · IND 133013 Extension Study

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗