Using Theophylline to Treat Pseudohypoparathyroidism
Effect of Theophylline in Pseudohypoparathyroidism
This study is testing if theophylline can help people with Pseudohypoparathyroidism improve their hormone response and possibly benefit their bone health, brain function, and metabolism.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | 5 Years to 21 Years |
| Sex | All |
| Sponsor | Massachusetts General Hospital Academic / other |
| Locations | 1 site (Boston, Massachusetts) |
| Trial ID | NCT03718403 on ClinicalTrials.gov |
What this trial studies
This study evaluates the effects of theophylline, a non-specific phosphodiesterase inhibitor, in patients diagnosed with Pseudohypoparathyroidism (PHP). It is an open-label intervention involving 100 subjects, focusing on the potential of theophylline to enhance cAMP signaling and reduce hormone resistance in PHP patients. The study aims to explore additional benefits of theophylline on bone maturation, neurocognition, and metabolism in this patient population.
Who should consider this trial
Good fit: Ideal candidates include individuals aged over 5 years with a confirmed diagnosis of PHP1A or PHP1B.
Not a fit: Patients with significant kidney disease, severe heart disease, or other specified exclusions may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve hormone responsiveness and overall health outcomes for patients with Pseudohypoparathyroidism.
How similar studies have performed: While the use of theophylline in this context is novel, similar studies exploring phosphodiesterase inhibitors have shown promise in related conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Subjects with known diagnosis of PHP1A and PHP1B * Age \>5 years Exclusion Criteria: * • Significant kidney disease, i.e. any CKD stage * Hypercalciuria * Kidney stones * Severe Heart disease * Uncontrolled seizure disorder * Peptic ulcer * Liver dysfunction * Bleeding disorder * Allergy to theophylline preparation * Muscle disorder * Infection * Any neurologic disease * Chronic drugs interacting with theophylline
Where this trial is running
Boston, Massachusetts
- Massachusetts General Hospital — Boston, Massachusetts, United States (Recruiting)
Study contacts
- Principal investigator: Amita Sharma, Md — Massachusetts General Hospital
- Study coordinator: Amita Sharma, Md
- Email: asharma5@mgh.harvard.edu
- Phone: 6177262908
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.