search
Back to results

Effect of Theophylline in Pseudohypoparathyroidism

Primary Purpose

PHP Ia, PHP IB, Php1C

Status
Recruiting
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Theophylline
Sponsored by
Massachusetts General Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for PHP Ia focused on measuring PHP

Eligibility Criteria

5 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Massachusetts General HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Single arm open labeled intervention study

Arm Description

Subjects with PHP will be given theophylline to decrease the end organ resistance by increasing levels of cAMP, a second messenger. Theophylline will be dosed twice a day for a period of 52 weeks.

Outcomes

Primary Outcome Measures

Effect of Theophylline on Bone Age in PHP
Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks

Secondary Outcome Measures

Full Information

First Posted
September 8, 2017
Last Updated
July 20, 2022
Sponsor
Massachusetts General Hospital
search

1. Study Identification

Unique Protocol Identification Number
NCT03718403
Brief Title
Effect of Theophylline in Pseudohypoparathyroidism
Official Title
Effect of Theophylline in Pseudohypoparathyroidism
Study Type
Interventional

2. Study Status

Record Verification Date
July 2022
Overall Recruitment Status
Recruiting
Study Start Date
November 11, 2019 (Actual)
Primary Completion Date
April 10, 2030 (Anticipated)
Study Completion Date
April 10, 2032 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Massachusetts General Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
PHP Ia, PHP IB, Php1C
Keywords
PHP

7. Study Design

Primary Purpose
Other
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Model Description
Open label intervention study, off label use of theophylline
Masking
None (Open Label)
Allocation
N/A
Enrollment
100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Single arm open labeled intervention study
Arm Type
Experimental
Arm Description
Subjects with PHP will be given theophylline to decrease the end organ resistance by increasing levels of cAMP, a second messenger. Theophylline will be dosed twice a day for a period of 52 weeks.
Intervention Type
Drug
Intervention Name(s)
Theophylline
Intervention Description
Theophylline will be given to maintain a peak level between 10-15 mcg/mL
Primary Outcome Measure Information:
Title
Effect of Theophylline on Bone Age in PHP
Description
Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks
Time Frame
52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
AMITA SHARMA, MD
Phone
6177262908
Email
asharma5@mgh.harvard.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
AMITA SHARMA, MD
Organizational Affiliation
Massachusetts General Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Amita Sharma
Phone
617-726-2908
Email
asharma5@mgh.harvard.edu

12. IPD Sharing Statement

Learn more about this trial

Effect of Theophylline in Pseudohypoparathyroidism

We'll reach out to this number within 24 hrs