Effect of Theophylline in Pseudohypoparathyroidism

Phase 4RecruitingNCT03718403

Massachusetts General Hospital100 enrolled

Overview

The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

OTHER

Masking

NONE

Enrollment

100

Conditions

PHP Ia PHP IB Php1C

Interventions

TheophyllineDRUG

Theophylline will be given to maintain a peak level between 10-15 mcg/mL

Eligibility

Sex: ALLMin age: 5 YearsMax age: 21 Years

Medical Language ↔ Plain English

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

Locations (1)

Massachusetts General Hospital

Boston, Massachusetts, United States

RECRUITING

Outcomes

Primary Outcomes

Effect of Theophylline on Bone Age in PHP

Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks

Time frame: 52 weeks

Central Contacts

AMITA SHARMA, MD

CONTACT

6177262908 asharma5@mgh.harvard.edu

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.