Effect of Theophylline in Pseudohypoparathyroidism

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

Study Overview

• Status: Recruiting
• Conditions: PHP Ia; PHP IB; Php1C
• Intervention / Treatment: Drug: Theophylline

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.

• Study Type: Interventional
• Enrollment (Anticipated): 100
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: AMITA SHARMA, MD; Phone Number: 6177262908; Email: asharma5@mgh.harvard.edu

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Recruiting
      • Massachusetts General Hospital
      • Contact: Amita Sharma; Phone Number: 617-726-2908; Email: asharma5@mgh.harvard.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 21 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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.	Drug: Theophylline; Theophylline will be given to maintain a peak level between 10-15 mcg/mL

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Effect of Theophylline on Bone Age in PHP	Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks	52 weeks

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Investigators: Principal Investigator: AMITA SHARMA, MD, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): November 11, 2019
• Primary Completion (Anticipated): April 10, 2030
• Study Completion (Anticipated): April 10, 2032

Study Registration Dates

• First Submitted: September 8, 2017
• First Submitted That Met QC Criteria: October 22, 2018
• First Posted (Actual): October 24, 2018

Study Record Updates

• Last Update Posted (Actual): July 22, 2022
• Last Update Submitted That Met QC Criteria: July 20, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Keywords: PHP
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Metabolism, Inborn Errors; Bone Diseases, Metabolic; Calcium Metabolism Disorders; Metal Metabolism, Inborn Errors; Pseudohypoparathyroidism; Pseudopseudohypoparathyroidism; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Vasodilator Agents; Autonomic Agents; Peripheral Nervous System Agents; Enzyme Inhibitors; Purinergic Antagonists; Purinergic Agents; Bronchodilator Agents; Anti-Asthmatic Agents; Respiratory System Agents; Phosphodiesterase Inhibitors; Purinergic P1 Receptor Antagonists; Theophylline
• Other Study ID Numbers: Theophylline in PHP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes