Effect of Theophylline in Pseudohypoparathyroidism
- Registration Number
- NCT03718403
- Lead Sponsor
- Massachusetts General Hospital
- Brief Summary
The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.
- Detailed 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 100
- Subjects with known diagnosis of PHP1A and PHP1B
- Age >5 years
-
• 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Single arm open labeled intervention study Theophylline Subjects with PHP will be given theophylline to decrease the end organ resistance by increasing levels of cAMP, a second messenger. Theophylline will be dosed twice a day for a period of 52 weeks.
- Primary Outcome Measures
Name Time Method Effect of Theophylline on Bone Age in PHP 52 weeks Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States