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Effect of Theophylline in 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03718403
Recruitment Status : Recruiting
First Posted : October 24, 2018
Last Update Posted : July 22, 2022
Sponsor:
Information provided by (Responsible Party):
Amita Sharma, Massachusetts General Hospital

Brief Summary:
The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.

Condition or disease Intervention/treatment Phase
PHP Ia PHP IB Php1C Drug: Theophylline Phase 4

Detailed Description:
It is an open label intervention study for the off-label use of theophylline in psuedohypoparathyroidism (PHP).Theophylline is a is non-specific phosphodiesterase inhibitor which increases the levels of secondary messenger,cAMP. PHP patients have end organ resistance to hormones involving cAMP signaling. Thus, theophylline has a potential to abrogate hormone resistance. Also, it may have a role in bone maturation, neurocognition and metabolism.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Open label intervention study, off label use of theophylline
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Effect of Theophylline in Pseudohypoparathyroidism
Actual Study Start Date : November 11, 2019
Estimated Primary Completion Date : April 10, 2030
Estimated Study Completion Date : April 10, 2032


Arm Intervention/treatment
Experimental: Single arm open labeled intervention study
Subjects with PHP will be given theophylline to decrease the end organ resistance by increasing levels of cAMP, a second messenger. Theophylline will be dosed twice a day for a period of 52 weeks.
Drug: Theophylline
Theophylline will be given to maintain a peak level between 10-15 mcg/mL




Primary Outcome Measures :
  1. Effect of Theophylline on Bone Age in PHP [ Time Frame: 52 weeks ]
    Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks



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Ages Eligible for Study:   5 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects with known diagnosis of PHP1A and PHP1B
  • Age >5 years

Exclusion Criteria:

  • • Significant kidney disease, i.e. any CKD stage

    • Hypercalciuria
    • Kidney stones
    • Severe Heart disease
    • Uncontrolled seizure disorder
    • Peptic ulcer
    • Liver dysfunction
    • Bleeding disorder
    • Allergy to theophylline preparation
    • Muscle disorder
    • Infection
    • Any neurologic disease
    • Chronic drugs interacting with theophylline

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): NCT03718403


Contacts
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Contact: AMITA SHARMA, MD 6177262908 asharma5@mgh.harvard.edu

Locations
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United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Amita Sharma    617-726-2908    asharma5@mgh.harvard.edu   
Sponsors and Collaborators
Massachusetts General Hospital
Investigators
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Principal Investigator: AMITA SHARMA, MD Massachusetts General Hospital
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Responsible Party: Amita Sharma, Medical Director, Division of Pediatric Nephrology, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT03718403    
Other Study ID Numbers: Theophylline in PHP
First Posted: October 24, 2018    Key Record Dates
Last Update Posted: July 22, 2022
Last Verified: July 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Amita Sharma, Massachusetts General Hospital:
PHP
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