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

Arm | Intervention/treatment |
---|---|
Experimental: Theophylline
Patients will receive a 24 hour continuous infusion of intravenous theophylline.
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Drug: Theophylline
24 hour infusion of IV theophylline
Other Name: Theo |
- Change in Urine cAMP [ Time Frame: 1 day ]Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline
- Change in Resting Energy Expenditure (REE) [ Time Frame: 1 day ]Change in REE before and during treatment with theophylline
- Change in Apnea Hypopnea Index (AHI) [ Time Frame: 1 day ]Change in AHI before and during treatment with theophylline

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Ages Eligible for Study: | 10 Years to 21 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age 10 to 21 years old
- English proficiency
- Clinical and genetic diagnosis of PHP1a
Exclusion Criteria:
- Use of a PDE inhibitor in the past 30 days
- History of a seizure disorder unrelated to hypocalcemia
- History of a cardiac arrhythmia (not including bradycardia)
- History of hepatic insufficiency
- AST or ALT >2x upper limit of normal
- 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)
- Congestive heart failure
- Cigarette use in the past 30 days
- Alcohol use within the past 24 hours
- Current pregnancy
- Untreated hypothyroidism (defined as free T4 level < 0.6 ng/dL or TSH >10 mcU/mL)
- Active peptic ulcer disease
- Fever >101 degrees in the past 24 hours
- Current use of medications known to effect theophylline levels (listed below)
- 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

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
United States, Tennessee | |
Vanderbilt Unversity | |
Nashville, Tennessee, United States, 37232 |
Principal Investigator: | Ashley H Shoemaker, MD, MSCI | Vanderbilt University |
Publications:
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). |
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 |