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Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

NCT04551170 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 34

Last updated 2026-04-29

No results posted yet for this study

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.

Conditions

  • Pseudohypoparathyroidism
  • Albright Hereditary Osteodystrophy
  • Pseudohypoparathyroidism Type 1a

Interventions

DRUG

Theophylline

oral theophylline

DRUG

Placebo

Placebo capsule or elixir

Sponsors & Collaborators

Principal Investigators

  • Jaclyn Tamaroff, MD · Vanderbilt University Medical Center

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-07-13
Primary Completion
2026-12-31
Completion
2026-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Companies

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 NCT04551170 on ClinicalTrials.gov