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

Primary Purpose

Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Theophylline

About this trial

This is an interventional treatment trial for Pseudohypoparathyroidism Type 1a

Eligibility Criteria

10 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Vanderbilt Unversity

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Theophylline

Arm Description

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

Outcomes

Primary Outcome Measures

Change in Urine cAMP

Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline

Secondary Outcome Measures

Change in Resting Energy Expenditure (REE)

Change in REE before and during treatment with theophylline

Change in Apnea Hypopnea Index (AHI)

Change in AHI before and during treatment with theophylline

Full Information

NCT ID

NCT02463409

First Posted

May 20, 2015

Last Updated

May 30, 2017

Sponsor

Vanderbilt University Medical Center

Collaborators

Massachusetts General Hospital

1. Study Identification

Unique Protocol Identification Number

NCT02463409

Brief Title

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

Official Title

Effects of Theophylline on cAMP Signaling in Children With Pseudohypoparathyroidism Type 1a

Study Type

Interventional

2. Study Status

Record Verification Date

May 2017

Overall Recruitment Status

Completed

Study Start Date

June 2015 (undefined)

Primary Completion Date

October 2016 (Actual)

Study Completion Date

October 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Vanderbilt University Medical Center

Collaborators

Massachusetts General Hospital

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

Detailed Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

6 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Theophylline

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Theophylline

Other Intervention Name(s)

Theo

Intervention Description

24 hour infusion of IV theophylline

Primary Outcome Measure Information:

Title

Change in Urine cAMP

Description

Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline

Time Frame

1 day

Secondary Outcome Measure Information:

Title

Change in Resting Energy Expenditure (REE)

Description

Change in REE before and during treatment with theophylline

Time Frame

1 day

Title

Change in Apnea Hypopnea Index (AHI)

Description

Change in AHI before and during treatment with theophylline

Time Frame

1 day

10. Eligibility

Sex

All

Minimum Age & Unit of Time

10 Years

Maximum Age & Unit of Time

21 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Ashley H Shoemaker, MD, MSCI

Organizational Affiliation

Vanderbilt University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Vanderbilt Unversity

City

Nashville

State/Province

Tennessee

ZIP/Postal Code

37232

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Investigators may contact Dr. Shoemaker for data after our primary analysis is completed and published (if applicable).

Citations:

PubMed Identifier

25925491

Citation

Landreth H, Malow BA, Shoemaker AH. Increased Prevalence of Sleep Apnea in Children with Pseudohypoparathyroidism Type 1a. Horm Res Paediatr. 2015;84(1):1-5. doi: 10.1159/000381452. Epub 2015 Apr 23.

Results Reference

background

PubMed Identifier

23229731

Citation

Shoemaker AH, Lomenick JP, Saville BR, Wang W, Buchowski MS, Cone RD. Energy expenditure in obese children with pseudohypoparathyroidism type 1a. Int J Obes (Lond). 2013 Aug;37(8):1147-53. doi: 10.1038/ijo.2012.200. Epub 2012 Dec 11.

Results Reference

background

Links:

URL

http://www.facebook.com/pseudohypoparathyroidism

Description

Vanderbilt PHP Research Page

Learn more about this trial

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

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