What side effects are associated with this type of intervention?

The most common treatments for pseudohypoparathyroidism include management of calcium and phosphate levels, often using calcium supplements and active vitamin D analogs. Theophylline, a phosphodiesterase inhibitor being studied for this condition, may cause side effects such as nausea, vomiting, diarrhea, headache, insomnia, and palpitations. Other phosphodiesterase inhibitors can also lead to similar adverse effects, including gastrointestinal disturbances and cardiovascular symptoms. It is important to monitor patients for these potential side effects during treatment.

What prior FDA approvals exist?

There is no specific information provided about prior FDA approvals for the treatment of pseudohypoparathyroidism or related drugs similar to theophylline, a phosphodiesterase inhibitor. Generally, treatment options for pseudohypoparathyroidism are limited and focus on managing symptoms such as obesity, short stature, and glucose intolerance. Theophylline is currently being studied in a phase 2 clinical trial for its potential benefits in this condition.

What other types of research exist?

Currently, there are no specific novel alternatives to Theophylline mentioned for Pseudohypoparathyroidism in the provided context. However, ongoing research and clinical trials may reveal new treatments targeting the underlying genetic and metabolic pathways involved in this condition. Patients should consult with their healthcare providers for the latest treatment options and clinical trial opportunities.

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Phosphodiesterase Inhibitor

Theophylline for Pseudohypoparathyroidism

Phase 2

Recruiting

Led By Ashley Shoemaker, MD

Research Sponsored by Vanderbilt University Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Obesity (BMI >95th percentile for age/gender and/or ≥30 kg/m2)

Age 13 years and above

Must not have

Congestive heart failure

Active peptic ulcer disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline and 52 weeks

Awards & highlights

All Individual Drugs Already Approved

Approved for 5 Other Conditions

Summary

This trial tests theophylline, a medication that helps important body signals last longer, in children and young adults with pseudohypoparathyroidism (PHP). These patients often struggle with obesity and short stature, which current treatments do not effectively address. Theophylline aims to improve their hormone response, potentially reducing weight and improving growth.

Who is the study for?

This trial is for individuals aged 13 and above with a clinical diagnosis of pseudohypoparathyroidism, who are significantly overweight. They must not have liver problems, heart arrhythmias (except slow heartbeat), untreated low thyroid hormone levels, active stomach ulcers, severe psychiatric disorders including recent major depression or suicidal behavior, heart failure, pregnancy plans within a year, or be using certain other medications.

What is being tested?

The study is testing theophylline's effectiveness in treating pseudohypoparathyroidism by comparing it to a placebo. The goal is to see if theophylline can help with weight loss, improve blood sugar control and affect bone growth in children and young adults.

What are the potential side effects?

Potential side effects of theophylline include insomnia, gastrointestinal discomforts like nausea or diarrhea, increased heart rate palpitations or irregularities; nervousness; headaches; and potentially more serious effects if levels get too high.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am considered obese based on my BMI.

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I am 13 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been diagnosed with congestive heart failure.

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I have an active stomach ulcer.

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I have a history of seizures not caused by low calcium.

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I have a history of irregular heartbeats, not including slow heartbeat.

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I am taking medication that affects theophylline levels.

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My liver is not working well (AST or ALT levels are high).

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline and 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline and 52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in body mass index

Secondary study objectives

Change in insulinogenic index

change in calcitriol dose

change in levothyroxine dose

Side effects data

From 2018 Phase 4 trial • 1670 Patients • NCT02261727

32%

COPD Exacerbation - not hospitalised

15%

COPD Exacerbation - Hospitalised

Upper Respiratory Tract Infection

100%

80%

60%

40%

20%

Study treatment Arm

Theophylline and Prednisone Arm

Low-dose Theophylline Arm

Placebo

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: TheophyllineExperimental Treatment1 Intervention

Theophylline capsules by mouth once daily or Theophylline elixir by mouth q6h (dose determined by serum drug levels)

Group II: PlacebosPlacebo Group1 Intervention

Theophylline capsule by mouth once daily or Theophylline elixir by mouth q6h

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Theophylline

FDA approved

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Theophylline, a phosphodiesterase inhibitor, works by increasing the levels of cyclic AMP (cAMP) within cells. This elevation in cAMP can lead to various beneficial effects, such as weight loss, improved glucose tolerance, and delayed growth plate closure, which are particularly relevant for pseudohypoparathyroidism patients who often suffer from early-onset obesity, short stature, and increased risk of type 2 diabetes. By targeting these specific symptoms, theophylline may offer a promising therapeutic approach for managing the metabolic and growth-related complications associated with pseudohypoparathyroidism.