Effect of Theophylline in Pseudohypoparathyroidism
Massachusetts General Hospital
Summary
The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.
Description
It is an open label intervention study for the off-label use of theophylline in psuedohypoparathyroidism (PHP).Theophylline is a is non-specific phosphodiesterase inhibitor which increases the levels of secondary messenger,cAMP. PHP patients have end organ resistance to hormones involving cAMP signaling. Thus, theophylline has a potential to abrogate hormone resistance. Also, it may have a role in bone maturation, neurocognition and metabolism.
Eligibility
- Age range
- 5–21 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Subjects with known diagnosis of PHP1A and PHP1B * Age \>5 years Exclusion Criteria: * • Significant kidney disease, i.e. any CKD stage * Hypercalciuria * Kidney stones * Severe Heart disease * Uncontrolled seizure disorder * Peptic ulcer * Liver dysfunction * Bleeding disorder * Allergy to theophylline preparation * Muscle disorder * Infection * Any neurologic disease * Chronic drugs interacting with theophylline
Interventions
- DrugTheophylline
Theophylline will be given to maintain a peak level between 10-15 mcg/mL
Location
- Massachusetts General HospitalBoston, Massachusetts