Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

• Register: ClinicalTrials.gov
• Last refreshed on: 11 November 2024
• Main ID: NCT04551170
• Date of registration: 09/09/2020
• Prospective Registration: No
• Primary sponsor: Vanderbilt University Medical Center
• Public title: Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old
• Scientific title: Phase 2 Study of Theophylline Treatment for Pseudohypoparathyroidism
• Date of first enrolment: July 13, 2020
• Target sample size: 34
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04551170
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Ashley Shoemaker, MD
• Affiliation: Vanderbilt University Medical Center

• Name: Jenny Leshko, RN

• Name: Ashley Shoemaker, M.D.

Key inclusion & exclusion criteria

Inclusion Criteria:

- Age 2 to 12 years old

- Clinical diagnosis of PHP (per the EuroPHP network classification guidelines5):
Presence of PTH resistance and/or ectopic ossification OR brachydactyly type E plus
2 minor criteria (TSH resistance, other hormonal resistance, developmental delay,
intrauterine or post-natal growth retardation, obesity/overweight, specific facial
features)

- Obesity (BMI >95th percentile for age/gender and/or =30 kg/m2)

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. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT >3x upper
limit of normal)

5. Congestive heart failure

6. Current cigarette use or alcohol abuse

7. Pregnancy or intention to become pregnant during the next year

8. Untreated hypothyroidism (defined as free thyroxine below the lower limit of normal)

9. Active peptic ulcer disease

10. Current use of medications known to effect theophylline levels (see protection of
human subjects)

11. History of hypersensitivity to theophylline or other medication components

12. History of Major Depressive Disorder in the past 2 years, lifetime history of
suicide attempt, history of any suicidal behavior in the past month, history of
other sever psychiatric disorders (e.g. schizophrenia, bipolar disorder)

13. PHQ-9 score is =15 or suicidal ideation of type 4 or 5 (C-SSR) in the past month

14. Untreated hypothyroidism or uncontrolled PTH resistance (PTH >2x upper limit of
normal), or treatment of these disorders by medications other than calcitriol or
levothyroxine (such as Cytomel or Armour thyroid)

15. Unable to comply with study procedures in the opinion of the investigator


Age minimum: 2 Years
Age maximum: 12 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pseudohypoparathyroidism

Pseudohypoparathyroidism Type 1a

Albright Hereditary Osteodystrophy

Intervention(s)

Drug: Placebo

Drug: Theophylline

Primary Outcome(s)

Change in body mass index [Time Frame: baseline and 52 weeks]

Secondary Outcome(s)

Change in epiphyseal closure [Time Frame: baseline and 52 weeks]

Change in calcitriol dose [Time Frame: baseline and 52 weeks]

Change in levothyroxine dose [Time Frame: baseline and 52 weeks]

Secondary ID(s)

IND 133103 R01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors