Effect of Theophylline in Pseudohypoparathyroidism
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ClinicalTrials.gov Identifier: NCT03718403 |
Recruitment Status :
Recruiting
First Posted : October 24, 2018
Last Update Posted : July 22, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
PHP Ia PHP IB Php1C | Drug: Theophylline | Phase 4 |
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.
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Drug: Theophylline
Theophylline will be given to maintain a peak level between 10-15 mcg/mL |
- 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 |
Inclusion Criteria:
- Subjects with known diagnosis of PHP1A and PHP1B
- Age >5 years
Exclusion Criteria:
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• 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

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
Contact: AMITA SHARMA, MD | 6177262908 | asharma5@mgh.harvard.edu |
United States, Massachusetts | |
Massachusetts General Hospital | Recruiting |
Boston, Massachusetts, United States, 02114 | |
Contact: Amita Sharma 617-726-2908 asharma5@mgh.harvard.edu |
Principal Investigator: | AMITA SHARMA, MD | Massachusetts General Hospital |
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 |
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 |
PHP |
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 |