Theophylline for Treatment of Pseudohypoparathyroidism | NCT04240821

Studies
Theophylline for Treatment of Pseudohypoparathyroidism

NCT04240821

Sponsor

Jaclyn Tamaroff

Status

Enrolling by invitation

Last Update Posted

Apr 28, 2026Actual

Enrollment

34Estimated

Phase

Phase 2

Conditions

Pseudohypoparathyroidism
Pseudohypoparathyroidism Type 1a
Albright Hereditary Osteodystrophy

Interventions

Theophylline ER

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

Feb 2026

Overall Recruitment Status or Expanded Access Status

Enrolling by invitation

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

May 22, 2020Actual

Primary Completion Date

Dec 31, 2026Estimated

Completion Date

Dec 31, 2026Estimated

First Submitted Date

Jan 21, 2020

First Submission Date that Met QC Criteria

Jan 21, 2020

First Posted Date

Jan 27, 2020Actual

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

Apr 23, 2026

Last Update Posted Date

Apr 28, 2026Actual

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

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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.

Detailed Description

Trial Objectives

The primary objective of this study is to evaluate the long-term safety of theophylline in patients with PHP.
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.

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 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

34Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Open-label theophylline	Experimental	Oral theophylline - either once daily capsule or q6h elixir.	Drug: Theophylline ER

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Theophylline ER	Drug	Oral theophylline	Open-label theophylline	Theophylline elixir

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Adverse Events	Treatment-emergent adverse events will be compared before and during treatment	24 months

Secondary Outcomes

Measure	Description	Time Frame
BMI	Change in BMI	24 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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:

History of a seizure disorder unrelated to hypocalcemia
History of a cardiac arrhythmia (not including bradycardia)
Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT >3x upper limit of normal)
Congestive heart failure
Current cigarette use or alcohol abuse
Pregnancy or intention to become pregnant during the next year
Active peptic ulcer disease
Current use of medications known to effect theophylline levels
History of hypersensitivity to theophylline or other medication components
Unable to comply with study procedures in the opinion of the investigator

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

2 Years

Maximum Age

99 Years

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Jaclyn Tamaroff, MD	Vanderbilt University Medical Center	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Vanderbilt University Medical Center		Nashville	Tennessee	37212	United States

Label	URL
Research Group Facebook Page	View source

IPD Sharing Statement Module

Plan to Share IPD

Yes

Description

Data will be shared upon request

Types

Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)

Time Frame

After publication

Access Criteria

University faculty

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D011547	Pseudohypoparathyroidism

Ancestor Terms

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

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