Theophylline for Treatment of Pseudohypoparathyroidism

Phase 2

• Conditions: Pseudohypoparathyroidism Type 1a; Pseudohypoparathyroidism; Albright Hereditary Osteodystrophy
• Interventions: Drug: Theophylline ER

• Registration Number: NCT04240821
• Lead Sponsor: Ashley Shoemaker

Brief 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.

Detailed Description

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 Timeline

• Study First Submitted: 2020-01-21
• Study First Submitted QC: 2020-01-21
• Study First Posted: 2020-01-27
• Study Start: 2020-05-22
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-29
• Last Update Posted: 2024-11-01
• Primary Completion: 2026-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 34

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open-label theophylline	Theophylline ER	Oral theophylline - either once daily capsule or q6h elixir.

Primary Outcome Measures

Name	Time	Method
Adverse Events	24 months	Treatment-emergent adverse events will be compared before and during treatment

Secondary Outcome Measures

Name	Time	Method
BMI	24 months	Change in BMI

Trial Locations

Locations (1)

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States