Active — Not RecruitingPhase 2

Theophylline Treatment for Pseudohypoparathyroidism

United States29 participantsStarted 2018-09-01

Plain-language summary

Pseudohypoparathyroidism is a genetic disorder with limited treatment options. Patients have early-onset obesity, short stature and increased risk of type 2 diabetes. This phase 2 clinical trial will test the efficacy of theophylline, a phosphodiesterase inhibitor, in pseudohypoparathyroidism. The investigators hypothesize that theophylline will cause weight loss, improve glucose tolerance and slow growth plate closure in children and young adults.

Who can participate

Age range13 Years – 99 Years

SexALL

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Inclusion criteria

✓. Age 13 years and above
✓. Clinical diagnosis of PHP (per the EuroPHP network classification guidelines1): Presence of PTH resistance or ectopic classification OR brachydactyly type E plus 2 minor criteria (TSH resistance, other hormonal resistance, developmental delay, intrauterine or post-natal growth retardation, obesity/overweight, specific facial features)
✓. Obesity (BMI \>95th percentile for age/gender and/or ≥30 kg/m2)

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 IDNCT03029429

SponsorVanderbilt University Medical Center

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-12-01

View on ClinicalTrials.gov More Pseudohypoparathyroidism trials