Effect of Theophylline in Pseudohypoparathyroidism

NCT03718403 | Recruiting Phase 4 DMC FDA Drug

• 1 other identifier: Theophylline in PHP
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

PHP Ia; PHP IB; Php1C

Keywords

PHP

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

Single arm open labeled intervention study

EXPERIMENTAL

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

Interventions

Theophylline DRUG

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

Single arm open labeled intervention study

Eligibility Criteria

• Age: 5 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subjects with known diagnosis of PHP1A and PHP1B
• Age \>5 years

You may not qualify if:

• 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

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

MeSH Terms

Conditions

Pseudohypoparathyroidism; Pseudohypoparathyroidism Type 1C

Interventions

Theophylline

Condition Hierarchy (Ancestors)

Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Calcium Metabolism Disorders

Intervention Hierarchy (Ancestors)

Xanthines; Alkaloids; Heterocyclic Compounds; Purinones; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• AMITA SHARMA, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

AMITA SHARMA, MD

CONTACT

6177262908; asharma5@mgh.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Director, Division of Pediatric Nephrology

Model Details: Open label intervention study, off label use of theophylline

Study Record Dates

• First Submitted: September 8, 2017
• First Posted: October 24, 2018
• Study Start: November 11, 2019
• Primary Completion (Estimated): April 10, 2030
• Study Completion (Estimated): April 10, 2032
• Last Updated: July 22, 2022

Record last verified: 2022-07

Locations

US (1)