Php1C, PHP Ia, PHP IB
Conditions
Keywords
PHP
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.
Interventions
DRUGTheophylline
Theophylline will be given to maintain a peak level between 10-15 mcg/mL
Sponsors
Massachusetts General Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Intervention model description
Open label intervention study, off label use of theophylline
Eligibility
Sex/Gender
ALL
Age
5 Years to 21 Years
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
United States
Contacts
Primary ContactAMITA SHARMA, MD
Outcome results
None listed