Clinical Trial: Safety and Efficacy Study of Doxycycline/UrsoDeoxyCholicAcid on Disease Progression in ATTR Amyloidosis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase II Multicenter Pilot Study of the Safety and Efficacy of Doxycycline/UrsoDeoxyCholicAcid on Disease Progression in ATTR Amyloidosis

Brief Summary: The primary objective for this study is to evaluate the efficacy of doxycycline + ursodeoxycholic acid (UDCA) on disease progression in Transthyretin Amyloidosis (ATTR) subjects with cardiomyopathy with or without neuropathy.

Detailed Summary:
Sponsor: Umeå University

Current Primary Outcome: The efficacy on serum N terminal proBNP (NT-proBNP) [ Time Frame: At 12 month treatment ]

The primary endpoint of the study is the response rate to doxycycline + UDCA treatment at month 12. A responder is an ATTR subject with:

- a reduction of, or an increase in serum NT-proBNP concentration of less than 30% of pre-treatment level will be regarded as consistent with treatment efficacy



Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Modified Body Mass Index (mBMI) reduction [ Time Frame: 12 month ]
    mBMI-reduction of less than 10%
  • Increase of septum thickness [ Time Frame: 12 month ]
    Increase of septum thickness ≤ 2 mm
  • Neurologic Kumamoto Scale [ Time Frame: 6, 12 and 18 month ]
    To assess the change from baseline in the neurologic Kumamoto Scale
  • Number of patients with adverse events [ Time Frame: During 12 month treatment and during 6 month follow-up ]

    To assess the tolerability and safety of the treatment, the number of patients with adverse reactions will be recorded.

    Monthly phone contacts will be performed for monitoring of the treatment safety.

    The safety profile of doxycycline + UDCA will be assessed through the recording, reporting and analysis of baseline medical conditions, physical examination findings including vital signs and laboratory tests. These will be compared to analysis results observed during the study.

  • Blood work for potential drug-related adverse events [ Time Frame: 18 months ]

    To assess the tolerability and safety of the treatment, blood work [e.g.complete blood count, creatinine and aspartate transaminase (AST), alkaline phosphatase(ALT)] for potential drug-related adverse events will be drawn at 1, 3, 6, 9, 12 and 18 month.

    The safety profile of doxycycline + UDCA will be assessed through the recording, reporting and analysis of baseline medical conditions, physical examination findings including vital signs and laboratory tests. These will be compared to analysis results observed during the study.



Original Secondary Outcome: Same as current

Information By: Umeå University

Dates:
Date Received: April 19, 2012
Date Started: February 2012
Date Completion:
Last Updated: May 11, 2016
Last Verified: May 2016