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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
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 belowSelect...
I am considered obese based on my BMI.
Select...
I am between 2 and 12 years old.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My thyroid condition is untreated or not well-controlled, and I am not on calcitriol or levothyroxine.
Select...
I have a history of seizures not caused by low calcium.
Select...
My liver is not working well (my liver tests are high).
Select...
I have been diagnosed with congestive heart failure.
Select...
I have an active stomach or intestinal ulcer.
Select...
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
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 • NCT0226172732%
COPD Exacerbation - not hospitalised
16%
COPD Exacerbation - Hospitalised
7%
Upper Respiratory Tract Infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
Low-dose Theophylline Arm
Placebo
Theophylline and Prednisone Arm
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
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.
Find a Location
Who is running the clinical trial?
Vanderbilt University Medical CenterLead Sponsor
894 Previous Clinical Trials
934,505 Total Patients Enrolled
4 Trials studying Pseudohypoparathyroidism
93 Patients Enrolled for Pseudohypoparathyroidism
Ashley Shoemaker, MDPrincipal Investigator - Vanderbilt University Medical Center
Monroe Carell Jr. Children's Hospital, Vanderbilt University
4 Previous Clinical Trials
3,107 Total Patients Enrolled
3 Trials studying Pseudohypoparathyroidism
107 Patients Enrolled for Pseudohypoparathyroidism
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- Your PHQ-9 score is 15 or higher, or you have had serious thoughts about harming yourself in the past month.My thyroid condition is untreated or not well-controlled, and I am not on calcitriol or levothyroxine.I have been diagnosed with PHP, showing specific symptoms like hormone resistance or specific hand bone changes.I have a history of seizures not caused by low calcium.I have taken a PDE inhibitor medication in the last 30 days.I have not had major depression, suicide attempts, or severe psychiatric disorders recently.My liver is not working well (my liver tests are high).I have been diagnosed with congestive heart failure.You have not been treated for low thyroid levels.I have an active stomach or intestinal ulcer.I am considered obese based on my BMI.You currently smoke cigarettes or abuse alcohol.I am not taking medications that affect theophylline levels.You have had a bad reaction to theophylline or any of the other ingredients in the medication.I have had a heart rhythm problem that is not slow heart rate.I am between 2 and 12 years old.
Research Study Groups:
This trial has the following groups:- Group 1: Theophylline
- Group 2: Placebo
Awards:
This trial has 2 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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