Theophylline for Treatment of Pseudohypoparathyroidism

Pseudohypoparathyroidism Clinical Trial

Official title:

Open-Label Extension Study of Theophylline for Treatment of Pseudohypoparathyroidism

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04240821
• Other study ID #: IND 133013 Extension Study
• Status: Enrolling by invitation
• Phase: Phase 2
• Start date: May 22, 2020
• Est. completion date: June 30, 2026

Study information

• Verified date: April 2024
• Source: Vanderbilt University Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 34
• Est. completion date: June 30, 2026
• Est. primary completion date: June 30, 2026
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 2 Years to 99 Years

Eligibility

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

Related Conditions & MeSH terms

• Albright Hereditary Osteodystrophy
• Pseudohypoparathyroidism
• Pseudohypoparathyroidism Type 1a

Intervention

Drug:
• Theophylline ER
Oral theophylline

Locations

Country	Name	City	State
United States	Vanderbilt University Medical Center	Nashville	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
Ashley Shoemaker

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events	Treatment-emergent adverse events will be compared before and during treatment	24 months
Secondary	BMI	Change in BMI	24 months

External Links

•   Research Group Facebook Page