Old asthma drug could help kids with rare bone and hormone disorder

NCT ID NCT04551170

Ongoing Disease control Sponsor: Vanderbilt University Medical Center Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tests theophylline, a drug used for asthma, in 34 children aged 2 to 12 with pseudohypoparathyroidism, a genetic condition causing obesity, short stature, and hormone resistance. The study aims to see if theophylline can help with weight loss, slow bone growth plate closure, and reduce hormone resistance. Results are not yet available.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: theophylline
What this could lead to: If it works, this could point toward a new treatment to help children with pseudohypoparathyroidism lose weight and reduce hormone resistance.
What could go wrong: This is a small early-phase trial with only 34 children, so results may not apply to everyone. Theophylline can cause side effects like nausea or heart issues, and it may not prove effective.

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Conditions

The condition(s) this trial relates to.

pseudohypoparathyroidism pseudohypoparathyroidism type 1A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ALBRIGHT HEREDITARY OSTEODYSTROPHY PSEUDOHYPOPARATHYROIDISM PSEUDOHYPOPARATHYROIDISM TYPE 1A

Contacts and locations

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Locations

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

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