What side effects are associated with this type of intervention?

Theophylline, a phosphodiesterase inhibitor being studied for pseudohypoparathyroidism, can cause side effects such as nausea, vomiting, diarrhea, headaches, insomnia, and palpitations. Other common treatments for pseudohypoparathyroidism, such as calcium and vitamin D supplementation, may lead to hypercalcemia, hypercalciuria, and gastrointestinal discomfort. It is important to monitor patients closely to manage these potential adverse effects.

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 treating this condition. Generally, treatment for pseudohypoparathyroidism focuses on managing symptoms and hormone resistance, but specific FDA-approved drugs for this purpose are not detailed in the provided information.

What other types of research exist?

Promising novel alternatives to Theophylline for Pseudohypoparathyroidism patients may include therapies targeting cAMP signaling pathways, such as vasopressin V2 receptor antagonists, or other hormone analogs that modulate related signaling pathways. Further research into these areas could provide new treatment options by 2024.

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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 2 to 12 years old

Must not have

Untreated hypothyroidism or uncontrolled PTH resistance (PTH >2x upper limit of normal), or treatment of these disorders by medications other than calcitriol or levothyroxine (such as Cytomel or Armour thyroid)

History of a seizure disorder unrelated to hypocalcemia

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline and 52 weeks

Awards & highlights

Approved for 5 Other Conditions

All Individual Drugs Already Approved

Summary

This trial tests theophylline, a long-used medication, in children with pseudohypoparathyroidism (PHP) who have obesity and growth issues. Theophylline helps keep important body signals active longer by blocking an enzyme. Researchers hope this will help with weight loss and growth in these children.

Who is the study for?

This trial is for children aged 2-12 with pseudohypoparathyroidism, characterized by obesity and hormone resistance. They must meet specific criteria including a BMI >95th percentile or ≥30 kg/m2 and have certain symptoms of the disorder. Kids can't join if they have untreated thyroid issues, liver problems, heart failure, are pregnant or planning to be, have had severe psychiatric disorders including depression in the last 2 years, smoke cigarettes or abuse alcohol.

What is being tested?

The study tests Theophylline against a placebo to see if it helps with weight loss and reduces hormone resistance in kids with pseudohypoparathyroidism. It's a phase 2 trial where some kids will get the real drug while others will get an inactive substance without knowing which one they're taking.

What are the potential side effects?

Possible side effects of Theophylline include stomach upset, increased heart rate (arrhythmia), nervousness, trouble sleeping (insomnia), headache and dizziness. Severe reactions might involve seizures or irregular heartbeat.

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 between 2 and 12 years old.

Exclusion Criteria

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

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My thyroid condition is untreated or not well-controlled, and I am not on calcitriol or levothyroxine.

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

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

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I have been diagnosed with congestive heart failure.

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I have an active stomach or intestinal ulcer.

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

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

Calcitriol

Change in epiphyseal closure

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

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

All Individual Drugs Already Approved

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

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: PlaceboPlacebo Group1 Intervention

Placebo capsule by mouth once daily or Placebo 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

Pseudohypoparathyroidism is characterized by resistance to parathyroid hormone (PTH), leading to symptoms such as hypocalcemia and hyperphosphatemia. Theophylline, a phosphodiesterase inhibitor, is being studied for its potential to treat this condition. Phosphodiesterase inhibitors work by preventing the breakdown of cyclic AMP (cAMP), a molecule that mediates the effects of PTH. By increasing cAMP levels, these inhibitors can enhance the cellular response to PTH, potentially alleviating the symptoms of hormone resistance. This mechanism is crucial for patients with Pseudohypoparathyroidism as it directly targets the underlying issue of hormone resistance, offering a potential therapeutic pathway to manage the disease more effectively.