Clinical Trial: Doxycycline to Upgrade Organ Response in Light Chain (AL) Amyloidosis Trial

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Doxycycline to Upgrade Organ Response in Light Chain (AL) Amyloidosis (DUAL) Trial: A Phase II Open Label Study of Oral Doxycycline Administered as an Adjunct to Plasma Cell Directed Therapy in Light

Brief Summary: In this study the investigators want to find out more about the addition of the antibiotic, doxycycline, to standard anti-amyloid therapy in people with amyloidosis. The investigators want to find out whether doxycycline improves the response to standard anti-amyloid therapy and whether it causes any problems (side effects).

Detailed Summary: Organ response to anti-plasma cell therapy in AL amyloidosis tends to lags behind hematologic response as chemotherapy may not clear pre-formed organ amyloid. Doxycycline has been shown to have inhibitory effects on amyloid fibril formation as well as de-fibrillogenic effects and shown to be beneficial in in vitro, murine models and other preclinical studies. The investigators will prospectively evaluate the safety and efficacy of doxycycline in AL amyloidosis patients when used in conjunction with anti-plasma cell chemotherapy.
Sponsor: Medical College of Wisconsin

Current Primary Outcome: Efficacy measured by amyloid organ response [ Time Frame: 1 year ]

Efficacy will be measured by amyloid organ response at 6 and 12 months.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Hematologic Response [ Time Frame: 1 year ]
    To assess rates of hematologic response.
  • Mortality [ Time Frame: 1 month, 6 months, 1 year ]
    To assess rates of mortality at 1 month, 6 months and 1 year
  • Composite measure of Patient reported Outcomes [ Time Frame: Baseline, 3, 6, 9 and 12 months ]

    We will collect patient-reported health quality of life in this study.

    At baseline, at each subsequent study visit and End of Treatment visti, the study coordinator will ask patients to complete a questionnaire to report on common domains of health-related quality of life using the Patient Reported Outcomes Measurement Informations System Global Health instrument, a 10-item standardized patient-reported outcome measure that provides global ratings of physical function, fatigue, pain, emotional distress

  • Safety [ Time Frame: 1 year ]
    Safety of the combination of doxycycline with anti-plasma cell chemotherapy in AL amyloidosis will be measured by "Number of Participants with Adverse Events", "Number of Participants with Grade III or IV Adverse Events", "Number of Participants discontinuing Doxycycline owing to Adverse Events"


Original Secondary Outcome:

  • Hematologic Response [ Time Frame: 1 year ]
    To assess rates of hematologic response.
  • Mortality [ Time Frame: 1 month, 6 months, 1 year ]
    To assess rates of mortality at 1 month, 6 months and 1 year
  • Composite measure of Patient reported Outcomes [ Time Frame: Baseline, 3, 6, 9 and 12 months ]

    One week following every monthly visit, the study coordinator will call the patient to assess for any early side-effects, monitor and encourage compliance. This patient-reported health quality of life data will be collected in two ways.

    At baseline and then during the regular telephone follow-up calls with patients at 3, 6, and 12 months, the study coordinator will ask patients a small number of open-ended questions on how they are feeling physically, emotionally and socially, and which aspects of their life are most affected by their health at that time

    At the same time points, the study coordinator will ask patients to report on common domains of health-related quality of life using the Patient Reported Outcomes Measurement Informations System Global Health instrument, a 10-item standardized patient-reported outcome measure that provides global ratings of physical function, fatigue, pain, emotional distress

  • Safety [ Time Frame: 1 year ]
    Safety of the combination of doxycycline with anti-plasma cell chemotherapy in AL amyloidosis will be measured by "Number of Participants with Adverse Events", "Number of Participants with Grade III or IV Adverse Events", "Number of Participants discontinuing Doxycycline owing to Adverse Events"


Information By: Medical College of Wisconsin

Dates:
Date Received: July 23, 2014
Date Started: October 2014
Date Completion: September 2019
Last Updated: May 11, 2017
Last Verified: May 2017