What side effects are associated with this type of intervention?

Phosphodiesterase inhibitors, such as theophylline, are being studied for the treatment of pseudohypoparathyroidism. Common side effects of phosphodiesterase inhibitors include gastrointestinal disturbances (nausea, vomiting, diarrhea), central nervous system effects (headache, dizziness, insomnia), and cardiovascular issues (tachycardia, palpitations). It is important for patients to discuss potential side effects with their healthcare provider to manage and mitigate any adverse effects effectively.

What prior FDA approvals exist?

There are no specific FDA-approved treatments for pseudohypoparathyroidism mentioned in the provided context. Theophylline, a phosphodiesterase inhibitor, is currently being studied for its potential benefits in this condition, including weight loss, improved glucose tolerance, and decreased hormone resistance. Broadly, treatment options for pseudohypoparathyroidism are limited and often focus on managing symptoms rather than targeting the underlying genetic disorder.

What other types of research exist?

Currently, there are no specific novel alternatives to Theophylline mentioned for the treatment of Pseudohypoparathyroidism in the provided context. Patients should consult their healthcare provider for the latest treatment options and ongoing clinical trials that may offer new therapeutic approaches.

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Phosphodiesterase Inhibitor

Theophylline for Pseudohypoparathyroidism

Phase 2

Waitlist Available

Led By Ashley Shoemaker, MD

Research Sponsored by Ashley Shoemaker

Eligibility Criteria Checklist

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

Must have

Must not have

Congestive heart failure

Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT >3x upper limit of normal)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

No Placebo-Only Group

All Individual Drugs Already Approved

Approved for 5 Other Conditions

Summary

This trial will test theophylline, a medication that helps relax muscles, in children and young adults with a genetic disorder called pseudohypoparathyroidism. The goal is to see if it can help them lose weight, handle sugar better, and respond better to hormones.

Who is the study for?

This trial is for children and young adults who have completed a previous theophylline study for pseudohypoparathyroidism. It's not open to those with seizure disorders, liver issues, active ulcers, heart failure, arrhythmias (except bradycardia), or those on certain meds. Pregnant individuals, smokers and heavy drinkers are also excluded.

What is being tested?

The trial is testing if Theophylline ER can help with weight loss, glucose tolerance and hormone resistance in patients with pseudohypoparathyroidism. This phase 2 trial follows up from an earlier study to further evaluate the drug's effectiveness.

What are the potential side effects?

Potential side effects of Theophylline may include nervousness, trouble sleeping, increased urination, stomach upset and occasionally more serious effects like irregular heartbeat or seizures.

Eligibility Criteria

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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My liver is not working well (my liver tests are high).

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I have had a heart rhythm problem that is not slow heart rate.

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I am taking medication that affects theophylline levels.

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I have had seizures not caused by low calcium levels.

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I currently have an active stomach ulcer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Adverse Events

Secondary study objectives

BMI

Side effects data

From 2023 Phase 3 trial • 100 Patients • NCT03984188

28%

COPD Exacerbation

14%

Gastritis

Arthalgia/Myalgia

Hospitalization for Respiratory Failure

Change in urinary frequency

Hospitalization for Stroke

Hospitalization for failure to thrive

100%

80%

60%

40%

20%

Study treatment Arm

Placebo Group

Low-dose Theophylline Group

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

1Treatment groups

Experimental Treatment

Group I: Open-label theophyllineExperimental Treatment1 Intervention

Oral theophylline - either once daily capsule or q6h elixir.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Theophylline

FDA approved

0 / 6Eligibility 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

Phosphodiesterase inhibitors like Theophylline work by preventing the breakdown of cyclic AMP (cAMP), which is crucial for hormone signaling. By increasing cAMP levels, these inhibitors can enhance the cellular response to hormones, potentially reducing the hormone resistance seen in pseudohypoparathyroidism. This is significant for patients as it directly addresses the core issue of hormone resistance, potentially improving symptoms such as hypocalcemia and hyperphosphatemia.