Theophylline for Treatment of Pseudohypoparathyroidism

Ashley Shoemaker

Status and phase: Invitation-only; Phase 2

Conditions: Pseudohypoparathyroidism Type 1a; Pseudohypoparathyroidism; Albright Hereditary Osteodystrophy

Treatments: Drug: Theophylline ER

Study type: Interventional

Funder types: Other

Identifiers

NCT04240821

IND 133013 Extension Study

Details and patient eligibility

About

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.

Full 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.

Enrollment: 34 estimated patients

Sex: All

Ages: 2 to 99 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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34 participants in 1 patient group

Open-label theophylline

Experimental group

Description:

Oral theophylline - either once daily capsule or q6h elixir.

Treatment:

Drug: Theophylline ER