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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 Description
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Brief Summary:
This study will test an investigational drug, theophylline, in children with pseudohypoparathyroidism type 1a (PHP1a). This study involves a 3 day visit to the Vanderbilt Clinical Research Center.

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

Detailed Description:
Study measures will be done at baseline and during a 24h infusion of IV theophylline. Theophylline levels will be drawn to ensure therapeutic dosing and to monitor for toxicity. Measures include laboratory values, response to PTH infusion and resting energy expenditure.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Effects of Theophylline on cAMP Signaling in Children With Pseudohypoparathyroidism Type 1a
Study Start Date : June 2015
Actual Primary Completion Date : October 2016
Actual Study Completion Date : October 2016

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Theophylline
Patients will receive a 24 hour continuous infusion of intravenous theophylline.
 Drug: Theophylline
24 hour infusion of IV theophylline
Other Name: Theo


Outcome Measures
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Primary Outcome Measures :
  1. Change in Urine cAMP [ Time Frame: 1 day ]
    Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline


Secondary Outcome Measures :
  1. Change in Resting Energy Expenditure (REE) [ Time Frame: 1 day ]
    Change in REE before and during treatment with theophylline

  2. Change in Apnea Hypopnea Index (AHI) [ Time Frame: 1 day ]
    Change in AHI before and during treatment with theophylline


Eligibility Criteria
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Ages Eligible for Study:   10 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age 10 to 21 years old
  2. English proficiency
  3. Clinical and genetic diagnosis of PHP1a

Exclusion Criteria:

  1. Use of a PDE inhibitor in the past 30 days
  2. History of a seizure disorder unrelated to hypocalcemia
  3. History of a cardiac arrhythmia (not including bradycardia)
  4. History of hepatic insufficiency
  5. AST or ALT >2x upper limit of normal
  6. Total bilirubin >1.5 x upper limit of normal (unless patient has a diagnosis of Gilbert's syndrome and no other causes leading to hyperbilirubinemia are identified)
  7. Congestive heart failure
  8. Cigarette use in the past 30 days
  9. Alcohol use within the past 24 hours
  10. Current pregnancy
  11. Untreated hypothyroidism (defined as free T4 level < 0.6 ng/dL or TSH >10 mcU/mL)
  12. Active peptic ulcer disease
  13. Fever >101 degrees in the past 24 hours
  14. Current use of medications known to effect theophylline levels (listed below)
  15. Severe sleep apnea requiring BiPAP

Drugs with clinically significant drug interactions with theophylline:

  • Allopurinol
  • Cimetidine
  • Ciprofloxacin
  • Clarithromycin
  • Enoxacin
  • Ephedrine
  • Erythromycin
  • Estrogen
  • Flurazepam
  • Fluvoxamine
  • Lithium
  • Lorazepam
  • Methotrexate
  • Midazolam
  • Pentoxifylline
  • Propranolol
  • Rifampin
  • Sulfinpyrazone
  • Tacrine
  • Thiabendazole
  • Ticlopidine
  • Troleandomycin
  • Verapamil
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): NCT02463409


Locations
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United States, Tennessee
Vanderbilt Unversity
Nashville, Tennessee, United States, 37232
Sponsors and Collaborators
Vanderbilt University Medical Center
Massachusetts General Hospital
Investigators
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Principal Investigator: Ashley H Shoemaker, MD, MSCI Vanderbilt University
More Information
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Additional Information:

Publications:

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Responsible Party: Ashley Shoemaker, Assistant Professor, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier: NCT02463409    
Other Study ID Numbers: IRB 150497
First Posted: June 4, 2015    Key Record Dates
Results First Posted: May 30, 2017
Last Update Posted: June 26, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Investigators may contact Dr. Shoemaker for data after our primary analysis is completed and published (if applicable).
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