Clinical Trial: Tolerability and Efficacy of a Combination of Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: An 18 Month, Open Label Study of the Tolerability and Efficacy of a Combination of Doxycycline and Tauroursodeoxycholic Acid (TUDCA) in Patients With Transthyretin Amyloid Cardiomyopathy.

Brief Summary: The objective of the study is to determine whether the combination of the bile acid TUDCA, and doxycycline will slow the progression of familial and senile amyloidosis.

Detailed Summary:
Sponsor: Brigham and Women's Hospital

Current Primary Outcome: The rate of progression of transthyretin cardiac amyloidosis, as measured by changes in strain echocardiography, in a group of patients taking a combination of doxycycline and TUDCA. [ Time Frame: Time Frame: * (FDAAA) outcome measure is assessed every 6 months by serial echocardiography, with final measurement 18 months after enrollment. ]

Outcome measure, namely changes in longitudinal echocardiographic strain, will be compared to previously determined changes derived from a cohort of patients with TTR cardiac amyloidosis who were not receiving specific therapy for amyloid deposition.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of patients with adverse events to the medications over the period of therapy (18 months) [ Time Frame: 18 months ]
    Measurements of kidney function (BUN and creatinine) and blood counts will be done every 3 months.
  • To evaluate general and health related quality of life (QoL) in SSA and ATTR subjects. [ Time Frame: Measurements made every 6 months for 18 months. ]


Original Secondary Outcome: Same as current

Information By: Brigham and Women's Hospital

Dates:
Date Received: March 12, 2013
Date Started: June 2013
Date Completion:
Last Updated: January 11, 2016
Last Verified: January 2016