๐Ÿ›๏ธ Verified NIH Record โ€ข Updated 2026-04-23

Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old (NCT04551170)

Phase 2 Study of Theophylline Treatment for Pseudohypoparathyroidism

๐Ÿ”ต Active, Not Recruiting Phase2 ๐Ÿ’Š Investigational Drug / Biologic Sponsor: Vanderbilt University Medical Center

๐Ÿฉบ Plain-English Summary (8th-Grade Level)

Pseudohypoparathyroidism is a genetic disorder with limited treatment options, characterized by early-onset obesity, short stature and resistance to multiple hormones. This phase 2 clinical trial and open-label extension study will test the efficacy of theophylline, a phosphodiesterase inhibitor, in pseudohypoparathyroidism. We hypothesize that theophylline will cause weight loss, slow the rate of growth plate closure and decrease hormone resistance in children.

Who can join: Review the eligibility criteria below.

Location: Vanderbilt University Medical Center โ€” Nashville, Tennessee

Age range: 2 Years to 12 Years

๐Ÿงช Interventions in This Study

๐Ÿ“‹ "Do I Qualify?" โ€” 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria โ†—

๐Ÿ“ All Participating Trial Locations (1 Sites)

  • Vanderbilt University Medical Center โ€” Nashville, Tennessee

๐Ÿ’ฐ Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Pseudohypoparathyroidism

๐Ÿ’ก How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
๐Ÿ›๏ธ View Official NIH Record โ†—