Theophylline Treatment for Pseudohypoparathyroidism

Running, not enrolling · Phase 2 · Has a placebo group

Conditions studied: Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy

In brief

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.

Key facts

Study ID

NCT03029429

Run by

Vanderbilt University Medical Center

People needed

29

Starts

2018-09-01

Expected to finish

2026-12-01

Last updated by the study team

2026-04-29

Who can join

Age: 13 and older, up to 99. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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)
• Active peptic ulcer disease
• Current use of medications known to effect theophylline levels
• History of hypersensitivity to theophylline or other medication components
• History of Major Depressive Disorder in the past 2 years, lifetime history of suicide attempt, history of any suicidal behavior in the past month, history of other sever psychiatric disorders (e.g. schizophrenia, bipolar disorder)
• PHQ-9 score is ≥15 or suicidal ideation of type 4 or 5 (C-SSR) in the past month
• Unable to comply with study procedures in the opinion of the investigator

Where it is running

• Jaclyn Tamaroff — Nashville, Tennessee, United States

Full record on ClinicalTrials.gov

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