Back to resultsTheophylline for Treatment of Pseudohypoparathyroidism
Primary Purpose
Pseudohypoparathyroidism, Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
Status
Enrolling by invitation
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Theophylline ER
Sponsored by
About this trial
This is an interventional treatment trial for Pseudohypoparathyroidism
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
Sites / Locations
- Vanderbilt University Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Open-label theophylline
Arm Description
Oral theophylline - either once daily capsule or q6h elixir.
Outcomes
Primary Outcome Measures
Adverse Events
Treatment-emergent adverse events will be compared before and during treatment
Secondary Outcome Measures
BMI
Change in BMI
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04240821
Brief Title
Theophylline for Treatment of Pseudohypoparathyroidism
Official Title
Open-Label Extension Study of Theophylline for Treatment of Pseudohypoparathyroidism
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
May 22, 2020 (Actual)
Primary Completion Date
June 30, 2025 (Anticipated)
Study Completion Date
June 30, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Ashley Shoemaker
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pseudohypoparathyroidism, Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
This is an open-label extension study
Masking
None (Open Label)
Allocation
N/A
Enrollment
34 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Open-label theophylline
Arm Type
Experimental
Arm Description
Oral theophylline - either once daily capsule or q6h elixir.
Intervention Type
Drug
Intervention Name(s)
Theophylline ER
Other Intervention Name(s)
Theophylline elixir
Intervention Description
Oral theophylline
Primary Outcome Measure Information:
Title
Adverse Events
Description
Treatment-emergent adverse events will be compared before and during treatment
Time Frame
24 months
Secondary Outcome Measure Information:
Title
BMI
Description
Change in BMI
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
99 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ashley Shoemaker, MD
Organizational Affiliation
Vanderbilt University Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Data will be shared upon request
IPD Sharing Time Frame
After publication
IPD Sharing Access Criteria
University faculty
Links:
URL
http://www.facebook.com/pseudohypoparathyroidism
Description
Research Group Facebook Page
Learn more about this trial
Theophylline for Treatment of Pseudohypoparathyroidism
We'll reach out to this number within 24 hrs