What side effects are associated with this type of intervention?

Common treatments for Primary Hyperparathyroidism include surgical removal of the overactive parathyroid gland(s) and medications such as bisphosphonates, calcimimetics, and hormone replacement therapy. Side effects of these treatments can vary. Surgical treatment may lead to complications such as hypocalcemia, infection, or damage to surrounding tissues. Bisphosphonates can cause gastrointestinal issues, musculoskeletal pain, and rarely, osteonecrosis of the jaw. Calcimimetics may result in nausea, vomiting, and hypocalcemia. Hormone replacement therapy can increase the risk of blood clots, stroke, and certain cancers. While Theophylline is not a standard treatment for Primary Hyperparathyroidism, its side effects include gastrointestinal disturbances, nervousness, and cardiovascular issues such as tachycardia.

What prior FDA approvals exist?

Primary Hyperparathyroidism is typically treated with surgical removal of the overactive parathyroid gland(s). For patients who are not surgical candidates, the FDA has approved medications such as cinacalcet, which is a calcimimetic agent that lowers parathyroid hormone levels by increasing the sensitivity of calcium-sensing receptors on the parathyroid gland. While theophylline, which acts through phosphodiesterase inhibition and adenosine receptor antagonism, is not specifically approved for Primary Hyperparathyroidism, it is used in other conditions like COPD. There are no FDA-approved treatments for Primary Hyperparathyroidism that work through the same mechanisms as theophylline.

What other types of research exist?

As of 2024, promising novel alternatives to Theophylline for Primary Hyperparathyroidism may include newer phosphodiesterase inhibitors or adenosine receptor antagonists that are currently under research. Additionally, treatments targeting the calcium-sensing receptor (CaSR) such as calcimimetics (e.g., etelcalcetide) and novel gene therapies or monoclonal antibodies that modulate parathyroid hormone (PTH) secretion could be considered. It is essential to consult with a healthcare provider to evaluate the most current and suitable treatment options based on individual patient needs and the latest clinical evidence.

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Methylxanthine

Theophylline for Pseudohypoparathyroidism

Phase 4

Recruiting

Led By AMITA SHARMA, MD

Research Sponsored by Massachusetts General Hospital

Eligibility Criteria Checklist

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

Must have

Subjects with known diagnosis of PHP1A and PHP1B

Age >5 years

Must not have

Muscle disorder

Liver dysfunction

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Drug Has Already Been Approved

Summary

This trial tests a medication in patients with a condition where the body doesn't respond well to hormones to help their bodies respond better.

Who is the study for?

This trial is for people over the age of 5 with a confirmed diagnosis of Pseudohypoparathyroidism types PHP1A and PHP1B. It's not suitable for those with muscle disorders, peptic ulcers, severe heart conditions, uncontrolled seizures, liver issues, bleeding problems, kidney stones or diseases, allergies to theophylline, infections or other neurological diseases.

What is being tested?

The study is testing the effects of a drug called Theophylline on individuals with Pseudohypoparathyroidism. A total of 100 participants will be involved to see how this medication influences their condition.

What are the potential side effects?

Possible side effects from taking Theophylline may include stomach discomforts like nausea and vomiting; restlessness; sleep disturbances; faster heartbeat; occasional headaches and irritability.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with PHP1A or PHP1B.

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I am older than 5 years.

Exclusion Criteria

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

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I have a muscle disorder.

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I have liver problems.

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I have severe heart disease.

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I am not taking any medications that interact with theophylline.

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I have kidney stones.

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I currently have an infection.

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I have a neurological condition.

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I have kidney disease.

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I have a peptic ulcer.

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I have seizures that are not controlled by medication.

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I have a bleeding disorder.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, 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

Effect of Theophylline on Bone Age in PHP

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

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

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

Drug Has Already Been Approved

The FDA has already approved this drug, and is just seeking more data.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Single arm open labeled intervention studyExperimental Treatment1 Intervention

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Theophylline

2008

Completed Phase 4

~2950

0 / 13Eligibility 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

Primary Hyperparathyroidism (PHPT) is commonly treated through surgical and pharmacologic approaches. The primary surgical treatment is parathyroidectomy, which involves the removal of the overactive parathyroid gland(s) to normalize calcium levels. Pharmacologic treatments include calcimimetics like cinacalcet, which enhance the sensitivity of the calcium-sensing receptors on the parathyroid gland, thereby reducing parathyroid hormone (PTH) secretion. Bisphosphonates and denosumab are used to manage bone density by inhibiting bone resorption. These treatments are crucial for PHPT patients as they help manage hypercalcemia and prevent complications such as osteoporosis, kidney stones, and cardiovascular issues.