Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

Launched by VANDERBILT UNIVERSITY MEDICAL CENTER · Sep 9, 2020

Keywords

ClinConnect Summary

This clinical trial is looking at a medication called theophylline to see if it can help children aged 2 to 12 years who have a rare genetic disorder known as pseudohypoparathyroidism. This condition can cause problems like early weight gain, short stature, and issues with hormone function. Researchers believe that theophylline might help these children lose weight, slow down growth plate closure, and improve their body's response to hormones. The trial is currently recruiting participants, and it's important to note that eligibility includes having a clinical diagnosis of pseudohypoparathyroidism and being classified as obese.

Children who join the study will receive theophylline and will be monitored closely by healthcare professionals. They will likely have regular check-ups to assess how the medication is affecting their weight and hormone levels. This trial is crucial because it aims to find new treatment options for a condition that currently has very few effective therapies available. If you think your child might qualify or if you have questions about participation, it’s a good idea to talk with your doctor for more information.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Age 2 to 12 years old
• • Clinical diagnosis of PHP (per the EuroPHP network classification guidelines5): Presence of PTH resistance and/or ectopic ossification 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)
• • 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 (see protection of human subjects)
• • 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. Untreated hypothyroidism or uncontrolled PTH resistance (PTH \>2x upper limit of normal), or treatment of these disorders by medications other than calcitriol or levothyroxine (such as Cytomel or Armour thyroid)
• • 15. Unable to comply with study procedures in the opinion of the investigator