NCT02463409

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2015

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 20, 2015

Completed
12 days until next milestone

Study Start

First participant enrolled

June 1, 2015

Completed
3 days until next milestone

First Posted

Study publicly available on registry

June 4, 2015

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2016

Completed
8 months until next milestone

Results Posted

Study results publicly available

May 30, 2017

Completed
Last Updated

June 26, 2017

Status Verified

May 1, 2017

Enrollment Period

1.3 years

First QC Date

May 20, 2015

Results QC Date

April 19, 2017

Last Update Submit

May 30, 2017

Conditions

Pseudohypoparathyroidism Type 1aAlbright Hereditary Osteodystrophy

Outcome Measures

Primary Outcomes (1)

  • Change in Urine cAMP

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

    1 day

Secondary Outcomes (2)

  • Change in Resting Energy Expenditure (REE)

    1 day

  • Change in Apnea Hypopnea Index (AHI)

    1 day

Study Arms (1)

Theophylline

EXPERIMENTAL

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

Drug: Theophylline

Interventions

TheophyllineDRUG

24 hour infusion of IV theophylline

Also known as: Theo
Theophylline

Eligibility Criteria

Age10 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age 10 to 21 years old
  • English proficiency
  • Clinical and genetic diagnosis of PHP1a

You may not qualify if:

  • 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
  • +24 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Vanderbilt Unversity

Nashville, Tennessee, 37232, United States

Location

Related Publications (2)

  • 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.

    PMID: 25925491BACKGROUND

  • 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.

    PMID: 23229731BACKGROUND

Related Links

MeSH Terms

Conditions

Pseudohypoparathyroidism

Interventions

Theophylline

Condition Hierarchy (Ancestors)

Bone Diseases, MetabolicBone DiseasesMusculoskeletal DiseasesMetal Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesCalcium Metabolism Disorders

Intervention Hierarchy (Ancestors)

XanthinesAlkaloidsHeterocyclic CompoundsPurinonesPurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Results Point of Contact

Title
Dr. Ashley Shoemaker
Organization
Vanderbilt University Medical Center
ashley.h.shoemaker@vanderbilt.edu
615-343-8816

Study Officials

  • Ashley H Shoemaker, MD, MSCI

    Vanderbilt University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Assistant Professor

Study Record Dates

First Submitted

May 20, 2015

First Posted

June 4, 2015

Study Start

June 1, 2015

Primary Completion

October 1, 2016

Study Completion

October 1, 2016

Last Updated

June 26, 2017

Results First Posted

May 30, 2017

Record last verified: 2017-05

Data Sharing

IPD Sharing
Will share

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

Locations

US(1)