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Trial record 2 of 9 for:    "Albrights hereditary osteodystrophy"

Theophylline as a Treatment for Children With Pseudohypoparathyroidism Type 1a (Albright Hereditary Osteodystrophy)

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ClinicalTrials.gov Identifier: NCT02463409
Recruitment Status : Completed
First Posted : June 4, 2015
Results First Posted : May 30, 2017
Last Update Posted : June 26, 2017
Sponsor:
Collaborator:
Massachusetts General Hospital
Information provided by (Responsible Party):
Ashley Shoemaker, Vanderbilt University Medical Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Pseudohypoparathyroidism Type 1a
Albright Hereditary Osteodystrophy
Intervention Drug: Theophylline
Enrollment 6
Recruitment Details  
Pre-assignment Details The screening visit included labs, physical exam and medical history, prior to assignment to the theophylline arm.
Arm/Group Title Theophylline
Hide Arm/Group Description

Patients will receive a 24 hour continuous infusion of intravenous theophylline.

Theophylline: 24 hour infusion of IV theophylline

Period Title: Overall Study
Started 6
Completed 4
Not Completed 2
Reason Not Completed
Withdrawal by Subject             1
Screen failure             1
Arm/Group Title Theophylline
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Patients will receive a 24 hour continuous infusion of intravenous theophylline.

Theophylline: 24 hour infusion of IV theophylline

Overall Number of Baseline Participants 5
Hide Baseline Analysis Population Description
All patients who enrolled in the study, not including screen fails
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 5 participants
14.4  (3.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Female 5
Male 0
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
5
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 5 participants
5
1.Primary Outcome
Title Change in Urine cAMP
Hide Description Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline
Time Frame 1 day
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patients treated with theophylline who maintained appropriate IV access. Only 3 patients had complete data available.
Arm/Group Title Theophylline
Hide Arm/Group Description:

Patients will receive a 24 hour continuous infusion of intravenous theophylline.

Theophylline: 24 hour infusion of IV theophylline

Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: fm/uL
3048  (8369)
2.Secondary Outcome
Title Change in Resting Energy Expenditure (REE)
Hide Description Change in REE before and during treatment with theophylline
Time Frame 1 day
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Theophylline
Hide Arm/Group Description:

Patients will receive a 24 hour continuous infusion of intravenous theophylline.

Theophylline: 24 hour infusion of IV theophylline

Overall Number of Participants Analyzed 4
Mean (Standard Error)
Unit of Measure: kcals per day
-14  (68)
3.Secondary Outcome
Title Change in Apnea Hypopnea Index (AHI)
Hide Description Change in AHI before and during treatment with theophylline
Time Frame 1 day
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Theophylline
Hide Arm/Group Description:

Patients will receive a 24 hour continuous infusion of intravenous theophylline.

Theophylline: 24 hour infusion of IV theophylline

Overall Number of Participants Analyzed 4
Mean (Standard Error)
Unit of Measure: units on a scale
-0.60  (0.67)
Time Frame 3 days
Adverse Event Reporting Description We specifically asked patients about headache, nausea and vomiting at least twice daily. They were monitored continuously via telemetry for arrhythmia. Other adverse events were reported as described by the partiicipant.
 
Arm/Group Title Theophylline
Hide Arm/Group Description

Patients will receive a 24 hour continuous infusion of intravenous theophylline.

Theophylline: 24 hour infusion of IV theophylline. All patients who enrolled in the study are included in this group (excluding screen fails).

All-Cause Mortality
Theophylline
Affected / at Risk (%)
Total   0/5 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Theophylline
Affected / at Risk (%) # Events
Total   0/5 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Theophylline
Affected / at Risk (%) # Events
Total   4/5 (80.00%)    
Cardiac disorders   
Tachycardia  1  1/5 (20.00%)  1
Gastrointestinal disorders   
Nausea  1  3/5 (60.00%)  3
Abdominal pain * 1  1/5 (20.00%)  1
Vomiting  1  1/5 (20.00%)  1
Nervous system disorders   
Headache  1  3/5 (60.00%)  3
dizziness * 1  1/5 (20.00%)  1
1
Term from vocabulary, MedDRA (10.0)
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Ashley Shoemaker
Organization: Vanderbilt University Medical Center
Phone: 615-343-8816 ext 615
Responsible Party: Ashley Shoemaker, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier: NCT02463409     History of Changes
Other Study ID Numbers: IRB 150497
First Submitted: May 20, 2015
First Posted: June 4, 2015
Results First Submitted: April 19, 2017
Results First Posted: May 30, 2017
Last Update Posted: June 26, 2017