NCT03718403PHASE4Recruiting

Effect of Theophylline in Pseudohypoparathyroidism

Massachusetts General Hospital

Start Date

11/11/2019

Completion Date

4/10/2032

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.

Eligibility Criteria

Age Range: 5 years to 21 years

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

DRUG

Theophylline

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Conditions

PHP IaPHP IBPhp1C

Locations

Massachusetts General Hospital

Boston, Massachusetts 02114

United States