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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02463409
• Date of registration: 20/05/2015
• Prospective Registration: Yes
• Primary sponsor: Vanderbilt University Medical Center
• Public title: Theophylline as a Treatment for Children With Pseudohypoparathyroidism Type 1a (Albright Hereditary Osteodystrophy)
• Scientific title: Effects of Theophylline on cAMP Signaling in Children With Pseudohypoparathyroidism Type 1a
• Date of first enrolment: June 2015
• Target sample size: 6
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02463409
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Ashley H Shoemaker, MD, MSCI
• Affiliation: Vanderbilt University

Key inclusion & exclusion criteria

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



Age minimum: 10 Years
Age maximum: 21 Years
Gender: All

Health Condition(s) or Problem(s) studied

Albright Hereditary Osteodystrophy

Pseudohypoparathyroidism Type 1a

Intervention(s)

Drug: Theophylline

Primary Outcome(s)

Change in Urine cAMP [Time Frame: 1 day]

Secondary Outcome(s)

Change in Apnea Hypopnea Index (AHI) [Time Frame: 1 day]

Change in Resting Energy Expenditure (REE) [Time Frame: 1 day]

Secondary ID(s)

IRB 150497

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Massachusetts General Hospital

Results

• Results available: Yes
• Date Posted: 30/05/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02463409