Theophylline for Treatment of Pseudohypoparathyroidism

Phase 2Enrolling By InvitationNCT04240821

Ashley Shoemaker34 enrolled

Overview

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.

Trial Objectives 1. The primary objective of this study is to evaluate the long-term safety of theophylline in patients with PHP. 2. The secondary objectives of this study are to evaluate changes in BMI, hormone resistance and epiphyseal closure associated with theophylline treatment of patients with PHP. Study Design The proposed study is a single center, 24-month open label extension clinical trial for patients with PHP who complete the randomized clinical trial.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Pseudohypoparathyroidism Pseudohypoparathyroidism Type 1a Albright Hereditary Osteodystrophy

Interventions

Theophylline ERDRUG

Oral theophylline

Eligibility

Sex: ALLMin age: 2 YearsMax age: 99 YearsHealthy volunteers:

Medical Language ↔ Plain English

Inclusion Criteria: * 1\. Successful completion of the randomized clinical trial "Phase 2 Study of Theophylline Treatment of Pseudohypoparathyroidism IND 133103 (11/1/2016)". Exclusion Criteria: 1. History of a seizure disorder unrelated to hypocalcemia 2. History of a cardiac arrhythmia (not including bradycardia) 3. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal) 4. Congestive heart failure 5. Current cigarette use or alcohol abuse 6. Pregnancy or intention to become pregnant during the next year 7. Active peptic ulcer disease 8. Current use of medications known to effect theophylline levels 9. History of hypersensitivity to theophylline or other medication components 10. Unable to comply with study procedures in the opinion of the investigator

Locations (1)

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Outcomes

Primary Outcomes

Adverse Events

Treatment-emergent adverse events will be compared before and during treatment

Time frame: 24 months

Secondary Outcomes

BMI

Change in BMI

Time frame: 24 months

Data from ClinicalTrials.gov

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