Theophylline as a Treatment for Children With Pseudohypoparathyroidism Type 1a (Albright Hereditary Osteodystrophy)

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-06-30

Study Completion Date

2016-10-31

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Pseudohypoparathyroidism Type 1a Albright Hereditary Osteodystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Theophylline

Patients will receive a 24 hour continuous infusion of intravenous theophylline.

Group Type EXPERIMENTAL

Theophylline

Intervention Type DRUG

24 hour infusion of IV theophylline

Interventions

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Theophylline

24 hour infusion of IV theophylline

Intervention Type DRUG

Other Intervention Names

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Theo

Eligibility Criteria

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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

Minimum Eligible Age

10 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No