Active — Not RecruitingPhase 2

Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

United States34 participantsStarted 2020-07-13

Plain-language summary

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 participate

Age range2 Years – 12 Years

SexALL

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Exclusion criteria

✕. Use of a PDE inhibitor in the past 30 days
✕. History of a seizure disorder unrelated to hypocalcemia
✕. History of a cardiac arrhythmia (not including bradycardia)
✕. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal)
✕. Congestive heart failure
✕. Current cigarette use or alcohol abuse
✕. Pregnancy or intention to become pregnant during the next year
✕. Untreated hypothyroidism (defined as free thyroxine below the lower limit of normal)

What they're measuring

Change in body mass index

Timeframe: baseline and 52 weeks

Trial details

NCT IDNCT04551170

SponsorVanderbilt University Medical Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-12-31

View on ClinicalTrials.gov More Pseudohypoparathyroidism trials