Effect of Theophylline in Pseudohypoparathyroidism

Mass General Brigham

Status and phase: Enrolling; Phase 4

Conditions: Php1C; PHP IB; PHP Ia

Treatments: Drug: Theophylline

Study type: Interventional

Funder types: Other

Identifiers

NCT03718403

Theophylline in PHP

Details and patient eligibility

About

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

Full 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.

Enrollment: 100 estimated patients

Sex: All

Ages: 5 to 21 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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100 participants in 1 patient group

Single arm open labeled intervention study

Experimental group

Description:

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.

Treatment:

Drug: Theophylline