Theophylline for Treatment of Pseudohypoparathyroidism

NCT04240821 · Status: ENROLLING_BY_INVITATION · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 34

Last updated 2026-04-28

No results posted yet for this study

Summary

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.

Conditions

  • Pseudohypoparathyroidism
  • Pseudohypoparathyroidism Type 1a
  • Albright Hereditary Osteodystrophy

Interventions

DRUG

Theophylline ER

Oral theophylline

Sponsors & Collaborators

  • Jaclyn Tamaroff

    lead OTHER

Principal Investigators

  • Jaclyn Tamaroff, MD · Vanderbilt University Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2020-05-22
Primary Completion
2026-12-31
Completion
2026-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04240821 on ClinicalTrials.gov