ACTIVE_NOT_RECRUITING

Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

Conditions

Pseudohypoparathyroidism Albright Hereditary Osteodystrophy Pseudohypoparathyroidism Type 1a PHP AHO

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Phase 2 Study of Theophylline Treatment for Pseudohypoparathyroidism

Quick Facts

Study Start:2020-07-13

Study Completion:2026-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT04551170

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:2 Years to 12 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
* Age 2 to 12 years old * Clinical diagnosis of PHP (per the EuroPHP network classification guidelines5): Presence of PTH resistance and/or ectopic ossification 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)	1. Use of a PDE inhibitor in the past 30 days 2. History of a seizure disorder unrelated to hypocalcemia 3. History of a cardiac arrhythmia (not including bradycardia) 4. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal) 5. Congestive heart failure 6. Current cigarette use or alcohol abuse 7. Pregnancy or intention to become pregnant during the next year 8. Untreated hypothyroidism (defined as free thyroxine below the lower limit of normal) 9. Active peptic ulcer disease 10. Current use of medications known to effect theophylline levels (see protection of human subjects) 11. History of hypersensitivity to theophylline or other medication components 12. 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) 13. PHQ-9 score is ≥15 or suicidal ideation of type 4 or 5 (C-SSR) in the past month 14. Untreated hypothyroidism or uncontrolled PTH resistance (PTH \>2x upper limit of normal), or treatment of these disorders by medications other than calcitriol or levothyroxine (such as Cytomel or Armour thyroid) 15. Unable to comply with study procedures in the opinion of the investigator

Inclusion Criteria

Exclusion Criteria

* Age 2 to 12 years old
* Clinical diagnosis of PHP (per the EuroPHP network classification guidelines5): Presence of PTH resistance and/or ectopic ossification 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)

1. Use of a PDE inhibitor in the past 30 days
2. History of a seizure disorder unrelated to hypocalcemia
3. History of a cardiac arrhythmia (not including bradycardia)
4. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal)
5. Congestive heart failure
6. Current cigarette use or alcohol abuse
7. Pregnancy or intention to become pregnant during the next year
8. Untreated hypothyroidism (defined as free thyroxine below the lower limit of normal)
9. Active peptic ulcer disease
10. Current use of medications known to effect theophylline levels (see protection of human subjects)
11. History of hypersensitivity to theophylline or other medication components
12. 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)
13. PHQ-9 score is ≥15 or suicidal ideation of type 4 or 5 (C-SSR) in the past month
14. Untreated hypothyroidism or uncontrolled PTH resistance (PTH \>2x upper limit of normal), or treatment of these disorders by medications other than calcitriol or levothyroxine (such as Cytomel or Armour thyroid)
15. Unable to comply with study procedures in the opinion of the investigator

Contacts and Locations

Principal Investigator

Ashley Shoemaker, MD

PRINCIPAL_INVESTIGATOR

Vanderbilt University Medical Center

Study Locations (Sites)

Vanderbilt University Medical Center

Nashville, Tennessee, 37212

United States

Collaborators and Investigators

Sponsor: Vanderbilt University Medical Center

Ashley Shoemaker, MD, PRINCIPAL_INVESTIGATOR, Vanderbilt University Medical Center

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2020-07-13

Study Completion Date2026-12-31

Study Record Updates

Study Start Date2020-07-13

Study Completion Date2026-12-31

Terms related to this study

Keywords Provided by Researchers

Pseudohypoparathyroidism
PHP
AHO
Albright Hereditary Osteodystrophy

Additional Relevant MeSH Terms

Pseudohypoparathyroidism
Albright Hereditary Osteodystrophy
Pseudohypoparathyroidism Type 1a