Effect of Theophylline in Pseudohypoparathyroidism

PHP Ia Clinical Trial

Official title:

Effect of Theophylline in Pseudohypoparathyroidism

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03718403
• Other study ID #: Theophylline in PHP
• Status: Recruiting
• Phase: Phase 4
• Start date: November 11, 2019
• Est. completion date: April 10, 2032

Study information

• Verified date: July 2022
• Source: Massachusetts General Hospital
• Contact: AMITA SHARMA, MD
• Phone: 6177262908
• Email: asharma5[@]mgh.harvard.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.

Description:

It is an open label intervention study for the off-label use of theophylline in psuedohypoparathyroidism (PHP).Theophylline is a is non-specific phosphodiesterase inhibitor which increases the levels of secondary messenger,cAMP. PHP patients have end organ resistance to hormones involving cAMP signaling. Thus, theophylline has a potential to abrogate hormone resistance. Also, it may have a role in bone maturation, neurocognition and metabolism.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: April 10, 2032
• Est. primary completion date: April 10, 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 21 Years

Eligibility

Inclusion Criteria:

• Subjects with known diagnosis of PHP1A and PHP1B

• Age >5 years

Exclusion Criteria:

• • Significant kidney disease, i.e. any CKD stage

• Hypercalciuria

• Kidney stones

• Severe Heart disease

• Uncontrolled seizure disorder

• Peptic ulcer

• Liver dysfunction

• Bleeding disorder

• Allergy to theophylline preparation

• Muscle disorder

• Infection

• Any neurologic disease

• Chronic drugs interacting with theophylline

Related Conditions & MeSH terms

• PHP Ia
• PHP IB
• Php1C
• Pseudohypoparathyroidism

Intervention

Drug:
• Theophylline
Theophylline will be given to maintain a peak level between 10-15 mcg/mL

Locations

Country	Name	City	State
United States	Massachusetts General Hospital	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Massachusetts General Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Effect of Theophylline on Bone Age in PHP	Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks	52 weeks