Pseudohypoparathyroidism, Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
Conditions
Brief summary
Pseudohypoparathyroidism is a genetic disorder with limited treatment options, characterized by early-onset obesity, short stature, hormone resistance and cognitive impairment. This phase 2 clinical trial will test the efficacy of theophylline, a phosphodiesterase inhibitor, in pseudohypoparathyroidism. We hypothesize that theophylline will cause weight loss, improve glucose tolerance and decrease hormone resistance in children and young adults.
Detailed description
Trial Objectives 1. The primary objective of this study is to evaluate the long-term safety of theophylline in patients with PHP. 2. The secondary objectives of this study are to evaluate changes in BMI, hormone resistance and epiphyseal closure associated with theophylline treatment of patients with PHP. Study Design The proposed study is a single center, 24-month open label extension clinical trial for patients with PHP who complete the randomized clinical trial.
Interventions
DRUGTheophylline ER
Oral theophylline
Sponsors
Ashley Shoemaker
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This is an open-label extension study
Eligibility
Sex/Gender
ALL
Age
2 Years to 99 Years
Healthy volunteers
Yes
Inclusion criteria
* 1\. Successful completion of the randomized clinical trial "Phase 2 Study of Theophylline Treatment of Pseudohypoparathyroidism IND 133103 (11/1/2016)".
Exclusion criteria
1. History of a seizure disorder unrelated to hypocalcemia 2. History of a cardiac arrhythmia (not including bradycardia) 3. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal) 4. Congestive heart failure 5. Current cigarette use or alcohol abuse 6. Pregnancy or intention to become pregnant during the next year 7. Active peptic ulcer disease 8. Current use of medications known to effect theophylline levels 9. History of hypersensitivity to theophylline or other medication components 10. Unable to comply with study procedures in the opinion of the investigator
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | 24 months | Treatment-emergent adverse events will be compared before and during treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| BMI | 24 months | Change in BMI |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORAshley Shoemaker, MD
Vanderbilt University Medical Center
Outcome results
None listed