Clincosm LogoSign in Get a demo

Theophylline for Treatment of Pseudohypoparathyroidism

Sponsor
Ashley Shoemaker (Other)
Overall Status
Enrolling by invitation
CT.gov ID
NCT04240821
Collaborator
(none)
34
Enrollment
1
Location
1
Arm
61.3
Anticipated Duration (Months)
0.6
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Theophylline ER
 Phase 2

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.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
34 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Intervention Model Description:
This is an open-label extension studyThis is an open-label extension study
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Open-Label Extension Study of Theophylline for Treatment of Pseudohypoparathyroidism
Actual Study Start Date :
May 22, 2020
Anticipated Primary Completion Date :
Jun 30, 2025
Anticipated Study Completion Date :
Jun 30, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: Open-label theophylline

Oral theophylline - either once daily capsule or q6h elixir.

Drug: Theophylline ER
Oral theophylline
Other Names:
  • Theophylline elixir

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Adverse Events [24 months]

      Treatment-emergent adverse events will be compared before and during treatment

    Secondary Outcome Measures

    1. BMI [24 months]

      Change in BMI

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    2 Years to 99 Years
    Sexes Eligible for Study:
    All
    Accepts 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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Vanderbilt University Medical Center Nashville Tennessee United States 37212

    Sponsors and Collaborators

    • Ashley Shoemaker

    Investigators

    • Principal Investigator: Ashley Shoemaker, MD, Vanderbilt University Medical Center

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    Ashley Shoemaker, Assistant Professor, Vanderbilt University Medical Center
    ClinicalTrials.gov Identifier:
    NCT04240821
    Other Study ID Numbers:
    • IND 133013 Extension Study
    First Posted:
    Jan 27, 2020
    Last Update Posted:
    Aug 16, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Pseudohypoparathyroidism
    Pseudopseudohypoparathyroidism
    Theophylline

    Study Results

    No Results Posted as of Aug 16, 2022