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Theophylline for Treatment of Pseudohypoparathyroidism

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04240821
Recruitment Status : Enrolling by invitation
First Posted : January 27, 2020
Last Update Posted : August 16, 2022
Sponsor:
Information provided by (Responsible Party):
Ashley Shoemaker, Vanderbilt University Medical Center

Study Description
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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.

Condition or disease Intervention/treatment Phase
Pseudohypoparathyroidism Pseudohypoparathyroidism Type 1a Albright Hereditary Osteodystrophy Drug: Theophylline ER Phase 2

Detailed Description:

Trial Objectives

  1. The primary objective of this study is to evaluate the long-term safety of theophylline in patients with PHP.
  2. 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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 34 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: This is an open-label extension study
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-Label Extension Study of Theophylline for Treatment of Pseudohypoparathyroidism
Actual Study Start Date : May 22, 2020
Estimated Primary Completion Date : June 30, 2025
Estimated Study Completion Date : June 30, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Open-label theophylline
Oral theophylline - either once daily capsule or q6h elixir.
 Drug: Theophylline ER
Oral theophylline
Other Name: Theophylline elixir


Outcome Measures
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Primary Outcome Measures :
  1. Adverse Events [ Time Frame: 24 months ]
    Treatment-emergent adverse events will be compared before and during treatment


Secondary Outcome Measures :
  1. BMI [ Time Frame: 24 months ]
    Change in BMI


Eligibility Criteria
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Ages Eligible for Study:   2 Years to 99 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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:

  1. History of a seizure disorder unrelated to hypocalcemia
  2. History of a cardiac arrhythmia (not including bradycardia)
  3. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT >3x upper limit of normal)
  4. Congestive heart failure
  5. Current cigarette use or alcohol abuse
  6. Pregnancy or intention to become pregnant during the next year
  7. Active peptic ulcer disease
  8. Current use of medications known to effect theophylline levels
  9. History of hypersensitivity to theophylline or other medication components
  10. Unable to comply with study procedures in the opinion of the investigator
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04240821


Locations
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United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37212
Sponsors and Collaborators
Ashley Shoemaker
Investigators
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Principal Investigator: Ashley Shoemaker, MD Vanderbilt University Medical Center
More Information
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Additional Information:

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Responsible Party: Ashley Shoemaker, Assistant Professor, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier: NCT04240821    
Other Study ID Numbers: IND 133013 Extension Study
First Posted: January 27, 2020    Key Record Dates
Last Update Posted: August 16, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Data will be shared upon request
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Time Frame: After publication
Access Criteria: University faculty

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pseudohypoparathyroidism
Pseudopseudohypoparathyroidism
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Calcium Metabolism Disorders
Theophylline
Bronchodilator Agents
Autonomic Agents
 Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Phosphodiesterase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Vasodilator Agents
Purinergic P1 Receptor Antagonists
Purinergic Antagonists
Purinergic Agents
Neurotransmitter Agents