Using Theophylline to Treat Pseudohypoparathyroidism
Phase 2 Study of Theophylline for the Treatment of Psuedohypoparathyroidism
['FUNDING_R01'] · VANDERBILT UNIVERSITY MEDICAL CENTER · NIH-11003699
This study is looking at whether the medication theophylline can help kids with pseudohypoparathyroidism by improving how their hormones work, which might help them grow better and manage their weight.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | VANDERBILT UNIVERSITY MEDICAL CENTER (nih funded) |
| Locations | 1 site (NASHVILLE, UNITED STATES) |
| Trial ID | NIH-11003699 on ClinicalTrials.gov |
What this research studies
This research investigates the use of theophylline, a phosphodiesterase inhibitor, to improve hormonal signaling in children with pseudohypoparathyroidism (PHP). PHP is a rare genetic disorder that leads to hormone deficiencies and associated issues like obesity and short stature. The study aims to enhance the function of Gsα-dependent receptors by increasing cyclic adenosine monophosphate (cAMP) levels, which may help reduce body mass index (BMI) and improve overall health outcomes. Participants will be monitored for changes in BMI, growth rates, and hormone resistance during the treatment period.
Who could benefit from this research
Good fit: Ideal candidates for this research are children diagnosed with pseudohypoparathyroidism who are experiencing early-onset obesity and short stature.
Not a fit: Patients without a diagnosis of pseudohypoparathyroidism or those who do not exhibit obesity or growth issues may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option for managing obesity and growth issues in children with pseudohypoparathyroidism.
How similar studies have performed: While the use of theophylline in this context is novel, similar approaches targeting hormonal signaling pathways have shown promise in other conditions.
Where this research is happening
NASHVILLE, UNITED STATES
- VANDERBILT UNIVERSITY MEDICAL CENTER — NASHVILLE, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: SHOEMAKER, ASHLEY HALL — VANDERBILT UNIVERSITY MEDICAL CENTER
- Study coordinator: SHOEMAKER, ASHLEY HALL
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.