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Effect of Theophylline in Pseudohypoparathyroidism

Effect of Theophylline in Pseudohypoparathyroidism

Recruiting
5-21 years
All
Phase 4

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Overview

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

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

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 details
    PHP Ia
    PHP IB
    Php1C

NCT03718403

Massachusetts General Hospital

27 January 2024

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