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

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ClinicalTrials.gov Identifier: NCT01855360
Recruitment Status : Completed
First Posted : May 16, 2013
Last Update Posted : June 20, 2017
Sponsor:
Information provided by (Responsible Party):
Rodney H. Falk, MD, Brigham and Women's Hospital

Tracking Information
First Submitted Date  ICMJE March 12, 2013
First Posted Date  ICMJE May 16, 2013
Last Update Posted Date June 20, 2017
Actual Study Start Date  ICMJE June 2013
Actual Primary Completion Date May 31, 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 14, 2013)
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 Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01855360 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 14, 2013)
  • 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 Measures  ICMJE Same as current
Current Other Outcome Measures  ICMJE Not Provided
Original Other Outcome Measures  ICMJE Not Provided
 
Descriptive Information
Brief Title  ICMJE Tolerability and Efficacy of a Combination of Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy
Official Title  ICMJE 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 Description Not Provided
Study Type  ICMJE Interventional
Study Phase Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Amyloidosis; Heart (Manifestation)
  • Senile Cardiac Amyloidosis
Intervention  ICMJE Drug: Tauroursodeoxycholic Acid and Doxycycline
Study Arms Experimental: TUDCA and Doxycycline
TUDCA taken orally, 250 mg three times daily. Doxycycline taken orally, 100 mg twice daily
Intervention: Drug: Tauroursodeoxycholic Acid and Doxycycline
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 16, 2017)
38
Original Estimated Enrollment  ICMJE
 (submitted: May 14, 2013)
40
Actual Study Completion Date May 31, 2015
Actual Primary Completion Date May 31, 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Documented transthyretin cardiac amyloidosis by biopsy and staining using immunohistochemistry or mass spectrometry
  • Echocardiographic appearance of left ventricular wall thickness of 13mm or more in the absence of hypertensive heart disease
  • Confirmed ATTR or SSA by genetic testing
  • Age 18-90
  • Male or non-pregnant, non-lactating females
  • Willingness to return to the treatment center for follow-up

Exclusion Criteria:

  • Prior liver transplantation or liver transplantation anticipated in less than 6 months
  • Alanine Transaminase and/or Aspartate Transaminase ≥2 x upper normal limit (UNL)
  • Alkaline Phosphatase ≥2 x UNL
  • Creatinine clearance <20 mL/min
  • Any other lab values that in the opinion of the investigator might place the subject at unacceptable risk for participation in the study
  • History of poor compliance
  • History of hypersensitivity to any of the ingredients of the study therapies
  • Any investigational drug within 4 weeks prior to study entry or during the study
  • Current use of diflunisal for therapy of amyloidosis
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 90 Years   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01855360
Other Study ID Numbers  ICMJE BWHAMY1
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Rodney H. Falk, MD, Brigham and Women's Hospital
Study Sponsor  ICMJE Brigham and Women's Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Rodney H Falk, MD Brigham and Women's Hospital, Boston MA
PRS Account Brigham and Women's Hospital
Verification Date June 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP