Theophylline for Treatment of Pseudohypoparathyroidism

NCT ID: NCT04240821

Last Updated: 2025-11-12

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

ENROLLING_BY_INVITATION

Clinical Phase

PHASE2

Total Enrollment

34 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-05-22

Study Completion Date

2026-12-31

Brief Summary

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Pseudohypoparathyroidism is a genetic disorder with limited treatment options, characterized by early-onset obesity, short stature, hormone resistance and cognitive impairment. This phase 2 clinical trial will test the efficacy of theophylline, a phosphodiesterase inhibitor, in pseudohypoparathyroidism. We hypothesize that theophylline will cause weight loss, improve glucose tolerance and decrease hormone resistance in children and young adults.

Detailed Description

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Trial Objectives

1. The primary objective of this study is to evaluate the long-term safety of theophylline in patients with PHP.
2. The secondary objectives of this study are to evaluate changes in BMI, hormone resistance and epiphyseal closure associated with theophylline treatment of patients with PHP.

Study Design The proposed study is a single center, 24-month open label extension clinical trial for patients with PHP who complete the randomized clinical trial.

Conditions

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Pseudohypoparathyroidism Pseudohypoparathyroidism Type 1a Albright Hereditary Osteodystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

This is an open-label extension study
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Open-label theophylline

Oral theophylline - either once daily capsule or q6h elixir.

Group Type EXPERIMENTAL

Theophylline ER

Intervention Type DRUG

Oral theophylline

Interventions

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Theophylline ER

Oral theophylline

Intervention Type DRUG

Other Intervention Names

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Theophylline elixir

Eligibility Criteria

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Inclusion Criteria

* 1\. Successful completion of the randomized clinical trial "Phase 2 Study of Theophylline Treatment of Pseudohypoparathyroidism IND 133103 (11/1/2016)".

Exclusion Criteria

1. History of a seizure disorder unrelated to hypocalcemia
2. History of a cardiac arrhythmia (not including bradycardia)
3. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal)
4. Congestive heart failure
5. Current cigarette use or alcohol abuse
6. Pregnancy or intention to become pregnant during the next year
7. Active peptic ulcer disease
8. Current use of medications known to effect theophylline levels
9. History of hypersensitivity to theophylline or other medication components
10. Unable to comply with study procedures in the opinion of the investigator
Minimum Eligible Age

2 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Ashley Shoemaker

OTHER

Sponsor Role lead

Responsible Party

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Ashley Shoemaker

Assistant Professor

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Ashley Shoemaker, MD

Role: PRINCIPAL_INVESTIGATOR

Vanderbilt University Medical Center

Locations

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Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Countries

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United States

Related Links

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Other Identifiers

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IND 133013 Extension Study

Identifier Type: -

Identifier Source: org_study_id