Phase 2 N=6 Treatment

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

Pseudohypoparathyroidism Type 1a · Albright Hereditary Osteodystrophy

Bottom Line

View on ClinicalTrials.gov: NCT02463409 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

May 2017

Primary outcome: Primary: Change in Urine cAMP — 3048 fm/uL

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Theophylline (Drug)
Age: Pediatric, Adult · 10+ yrs
Sex: All
Sponsor: Vanderbilt University Medical Center
Primary completion: Oct 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in Urine cAMP	3048	—
SECONDARY Change in Resting Energy Expenditure (REE)	-14	—
SECONDARY Change in Apnea Hypopnea Index (AHI)	-0.60	—

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.

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 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02463409). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.