Theophylline for Treatment of Pseudohypoparathyroidism

Part of paid clinical trials in Nashville, Tennessee.

Sponsor
Jaclyn Tamaroff
Study ID
NCT04240821
Phase
PHASE2
Status
Enrolling By Invitation

Conditions

  • Albright Hereditary Osteodystrophy
  • Pseudohypoparathyroidism
  • Pseudohypoparathyroidism Type 1a

Eligibility Criteria

Sex
ALL
Age
2 Years - 99 Years
Healthy Volunteers
Accepted

Interventions

  • Theophylline ER — DRUG
    Oral theophylline

Study Details

Pseudohypoparathyroidism is a genetic disorder with limited treatment options, characterized by early-onset obesity, short stature, hormone resistance and cognitive impairment. This phase 2 clinical trial will test the efficacy of theophylline, a phosphodiesterase inhibitor, in pseudohypoparathyroidism. We hypothesize that theophylline will cause weight loss, improve glucose tolerance and decrease hormone resistance in children and young adults.

Key Dates

Start date
May 22, 2020
Status verified
Feb 2026
Primary completion
Dec 31, 2026
Completion
Dec 31, 2026

Study Design

Enrollment
34 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Open-label theophylline
    Oral theophylline - either once daily capsule or q6h elixir.

Primary Outcome Measure

Adverse Events [ Time Frame: 24 months ]

Locations (1)

FacilityCityStateZIPSite coordinators
Vanderbilt University Medical CenterNashvilleTennessee37212-

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By research site
Vanderbilt University Medical Center· Nashville, TN

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