Effect of Theophylline in Pseudohypoparathyroidism | NCT03718403

Studies
Effect of Theophylline in Pseudohypoparathyroidism

NCT03718403

Sponsor

Massachusetts General Hospital

Status

Recruiting

Last Update Posted

Jul 22, 2022Actual

Enrollment

100Estimated

Phase

Phase 4

Conditions

PHP Ia
PHP IB
Php1C

Interventions

Theophylline

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jul 2022

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 11, 2019Actual

Primary Completion Date

Apr 10, 2030Estimated

Completion Date

Apr 10, 2032Estimated

First Submitted Date

Sep 8, 2017

First Submission Date that Met QC Criteria

Oct 22, 2018

First Posted Date

Oct 24, 2018Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 20, 2022

Last Update Posted Date

Jul 22, 2022Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Amita Sharma, Medical Director, Division of Pediatric Nephrology, Massachusetts General HospitalPrincipal Investigator

Lead Sponsor

Massachusetts General HospitalOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Yes

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 4

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

100Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Single arm open labeled intervention study	Experimental	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.	Drug: Theophylline

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Theophylline	Drug	Theophylline will be given to maintain a peak level between 10-15 mcg/mL	Single arm open labeled intervention study

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

5 Years

Maximum Age

21 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
AMITA SHARMA, MD	Contact	6177262908		asharma5@mgh.harvard.edu

Overall Officials

Name	Affiliation	Role
AMITA SHARMA, MD	Massachusetts General Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Massachusetts General Hospital	Recruiting	Boston	Massachusetts	02114	United States

Condition Browse Module

MeSH Terms

ID	Term
D011547	Pseudohypoparathyroidism
C548076	Pseudohypoparathyroidism Type 1C

Ancestor Terms

ID	Term
D001851	Bone Diseases, Metabolic
D001847	Bone Diseases
D009140	Musculoskeletal Diseases
D008664	Metal Metabolism, Inborn Errors

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
D013806	Theophylline

Ancestor Terms

ID	Term
D014970	Xanthines
D000470	Alkaloids
D006571	Heterocyclic Compounds
D011688	Purinones

Browse Leaves

Not provided

Browse Branches

Not provided