Effect of Theophylline in Pseudohypoparathyroidism

NCT ID: NCT03718403

Last Updated: 2022-07-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-11-11

Study Completion Date

2032-04-10

Brief Summary

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

Detailed Description

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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.

Conditions

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PHP Ia PHP IB Php1C

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Open label intervention study, off label use of theophylline
Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Theophylline

Intervention Type DRUG

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

Interventions

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Theophylline

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

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

5 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Massachusetts General Hospital

OTHER

Sponsor Role lead

Responsible Party

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Amita Sharma

Medical Director, Division of Pediatric Nephrology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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AMITA SHARMA, MD

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

Locations

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Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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AMITA SHARMA, MD

Role: CONTACT

6177262908

Facility Contacts

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Amita Sharma

Role: primary

617-726-2908

Other Identifiers

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Theophylline in PHP

Identifier Type: -

Identifier Source: org_study_id

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