Theophylline Treatment for Pseudohypoparathyroidism

Launched by VANDERBILT UNIVERSITY MEDICAL CENTER · Jan 23, 2017

Keywords

ClinConnect Summary

This clinical trial is studying a medication called theophylline to see if it can help people with a genetic disorder called pseudohypoparathyroidism (PHP). This condition can lead to early obesity, short stature, and a higher risk of type 2 diabetes. Researchers believe that theophylline might help participants lose weight, improve how their body handles sugar, and slow down the growth plate closure in younger individuals. The trial is currently looking for participants aged 13 and older who have been diagnosed with PHP and are dealing with obesity.

If you or a family member are interested in participating, it’s important to know that there are specific criteria to qualify. Eligible participants must have a clinical diagnosis of PHP and a high body mass index (BMI) indicating obesity. However, certain health conditions or recent medication use might exclude someone from joining the trial. Participants can expect to contribute to important research while potentially benefiting from the treatment being studied. This trial is an opportunity to explore new treatment options for a condition that currently has limited choices.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Age 13 years and above
• • 2. Clinical diagnosis of PHP (per the EuroPHP network classification guidelines1): Presence of PTH resistance or ectopic classification OR brachydactyly type E plus 2 minor criteria (TSH resistance, other hormonal resistance, developmental delay, intrauterine or post-natal growth retardation, obesity/overweight, specific facial features)
• • 3. Obesity (BMI \>95th percentile for age/gender and/or ≥30 kg/m2)
• Exclusion Criteria:
• • 1. Use of a PDE inhibitor in the past 30 days
• • 2. History of a seizure disorder unrelated to hypocalcemia
• • 3. History of a cardiac arrhythmia (not including bradycardia)
• • 4. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal)
• • 5. Congestive heart failure
• • 6. Current cigarette use or alcohol abuse
• • 7. Pregnancy or intention to become pregnant during the next year
• • 8. Untreated hypothyroidism (defined as free thyroxine below the lower limit of normal)
• • 9. Active peptic ulcer disease
• • 10. Current use of medications known to effect theophylline levels
• • 11. History of hypersensitivity to theophylline or other medication components
• • 12. History of Major Depressive Disorder in the past 2 years, lifetime history of suicide attempt, history of any suicidal behavior in the past month, history of other sever psychiatric disorders (e.g. schizophrenia, bipolar disorder)
• • 13. PHQ-9 score is ≥15 or suicidal ideation of type 4 or 5 (C-SSR) in the past month
• • 14. Unable to comply with study procedures in the opinion of the investigator