ACTIVE_NOT_RECRUITING

Theophylline Treatment for Pseudohypoparathyroidism

Conditions

Pseudohypoparathyroidism Albright Hereditary Osteodystrophy 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. 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.

Official Title

Phase 2 Study of Theophylline Treatment for Pseudohypoparathyroidism

Quick Facts

Study Start:2018-09-01

Study Completion:2026-12-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT03029429

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:13 Years to 99 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Age 13 years and above 2. 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) 3. 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 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. Unable to comply with study procedures in the opinion of the investigator

Inclusion Criteria

Exclusion Criteria

1. Age 13 years and above
2. 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)
3. 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
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. 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)

Ashley Shoemaker

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 Date2018-09-01

Study Completion Date2026-12-01

Study Record Updates

Study Start Date2018-09-01

Study Completion Date2026-12-01

Terms related to this study

Keywords Provided by Researchers

pseudohypoparathyroidism
AHO
Albright Hereditary Osteodystrophy

Additional Relevant MeSH Terms

Pseudohypoparathyroidism
Albright Hereditary Osteodystrophy