Effect of Theophylline in Pseudohypoparathyroidism
Purpose
The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.
Conditions
- PHP Ia
- PHP IB
- Php1C
Eligibility
- Eligible Ages
- Between 5 Years and 21 Years
- Eligible Genders
- All
- Accepts 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
Study Design
- Phase
- Phase 4
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Intervention Model Description
- Open label intervention study, off label use of theophylline
- Primary Purpose
- Other
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Single arm open labeled intervention study |
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. |
|
Recruiting Locations
Massachusetts General Hospital
Boston, Massachusetts 02114
Boston, Massachusetts 02114
More Details
- Status
- Recruiting
- Sponsor
- Massachusetts General Hospital
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.