Effect of Theophylline in Pseudohypoparathyroidism

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ClinicalTrials.gov Identifier: NCT03718403

Recruitment Status : Not yet recruiting

First Posted : October 24, 2018

Last Update Posted : October 24, 2018

See Contacts and Locations

Sponsor:

Information provided by (Responsible Party):

Amita Sharma, Massachusetts General Hospital

Study Description

Brief Summary:

The study evaluates the effect of theophylline in 40 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.

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	40 participants
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
Estimated Study Start Date :	April 10, 2019
Estimated Primary Completion Date :	April 10, 2030
Estimated Study Completion Date :	April 10, 2032

Resource links provided by the National Library of Medicine

Drug Information available for: Theophylline

Genetic and Rare Diseases Information Center resources: Pseudohypoparathyroidism Pseudopseudohypoparathyroidism Albright's Hereditary Osteodystrophy Pseudohypoparathyroidism Type 1A

U.S. FDA Resources

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

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

Eligibility Criteria

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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

Contacts and Locations

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

Sponsors and Collaborators

Massachusetts General Hospital

Investigators

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Principal Investigator:	AMITA SHARMA, MD	Massachusetts General Hospital

More Information

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Responsible Party:	Amita Sharma, Medical Director, Division of Pediatric Nephrology, Massachusetts General Hospital
ClinicalTrials.gov Identifier:	NCT03718403 History of Changes
Other Study ID Numbers:	Theophylline in PHP
First Posted:	October 24, 2018 Key Record Dates
Last Update Posted:	October 24, 2018
Last Verified:	October 2018

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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

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