Ignite Creation Date:
2024-05-06 @ 11:28 AM
Last Modification Date:
2024-10-26 @ 12:46 PM
Study NCT ID:
NCT03528902
Status:
COMPLETED
Last Update Posted:
2023-08-01
First Post:
2018-03-21
Brief Title:
Tamoxifen Therapy to Treat Pulmonary Arterial Hypertension
Sponsor:
Vanderbilt University Medical Center
Organization:
Vanderbilt University Medical Center
Study Overview
Official Title:
Tamoxifen Therapy to Treat Pulmonary Arterial Hypertension
Status:
COMPLETED
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
T3PAH
Brief Summary:
The main purpose of this clinical trial is to examine the feasibility and effects of tamoxifen in subjects with pulmonary arterial hypertension PAH The study will evaluate how well the drug is tolerated and its impact on functional condition and selected biomarkers Changes in tricuspid annular plane systolic excursion TAPSE and other parameters determined by transthoracic echocardiography will be evaluated as well as changes in additional metrics such as six minute walk test distance quality of life assessments and hormone levels
Detailed Description:
Pulmonary arterial hypertension PAH is characterized by progressive loss of function by the pulmonary vascular bed due to a variety of factors including obliterative vascular lesions vasoconstriction and thrombotic occlusion of the pulmonary arteries Ultimately right-sided heart failure ensues with severe limitation of exercise and eventual progression to death or lung transplantation While there are multiple FDA-approved therapies for PAH representing 3 major pathways of interest no treatments are curative and have additional limitations including high expense multiple side effects and dosing inconveniences
The strongest established risk factor for the progressively fatal disease pulmonary arterial hypertension PAH is female sex 31 femalemale ratio We and others have found higher circulating estrogen levels and enhanced estrogen signaling in PAH patients Preclinical work by our group and others supports the concept that anti-estrogen therapy is effective for both prevention and treatment in PAH Recent and ongoing clinical studies are underway to assess these approaches in humans including a recent study demonstrating the safety of estrogen reduction in postmenopausal women
Tamoxifen is the most commonly used selective estrogen receptor modulator SERM Due to its extensive use in humans for over three decades it has an excellent safety profile and its long-term sequelae are well characterized Furthermore it is a generic drug which has been FDA-approved for treatment and prevention of breast cancer particularly those with estrogen receptor-positive neoplasms
To help to determine whether tamoxifen may be a safe and effective treatment for PAH in women and men we will conduct a single-center randomized double-blind placebo-controlled Phase II study of subjects with PAH All subjects will also be treated with background standard of care therapy at the discretion of their PAH care physician
The strongest established risk factor for the progressively fatal disease pulmonary arterial hypertension PAH is female sex 31 femalemale ratio We and others have found higher circulating estrogen levels and enhanced estrogen signaling in PAH patients Preclinical work by our group and others supports the concept that anti-estrogen therapy is effective for both prevention and treatment in PAH Recent and ongoing clinical studies are underway to assess these approaches in humans including a recent study demonstrating the safety of estrogen reduction in postmenopausal women
Tamoxifen is the most commonly used selective estrogen receptor modulator SERM Due to its extensive use in humans for over three decades it has an excellent safety profile and its long-term sequelae are well characterized Furthermore it is a generic drug which has been FDA-approved for treatment and prevention of breast cancer particularly those with estrogen receptor-positive neoplasms
To help to determine whether tamoxifen may be a safe and effective treatment for PAH in women and men we will conduct a single-center randomized double-blind placebo-controlled Phase II study of subjects with PAH All subjects will also be treated with background standard of care therapy at the discretion of their PAH care physician
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None