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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01855360
Recruitment Status : Completed
First Posted : May 16, 2013
Last Update Posted : June 20, 2017
Information provided by (Responsible Party):
Rodney H. Falk, MD, Brigham and Women's Hospital

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.

Condition or disease Intervention/treatment Phase
Amyloidosis; Heart (Manifestation) Senile Cardiac Amyloidosis Drug: Tauroursodeoxycholic Acid and Doxycycline Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 38 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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.
Actual Study Start Date : June 2013
Actual Primary Completion Date : May 31, 2015
Actual Study Completion Date : May 31, 2015

Arm Intervention/treatment
Experimental: TUDCA and Doxycycline
TUDCA taken orally, 250 mg three times daily. Doxycycline taken orally, 100 mg twice daily
Drug: Tauroursodeoxycholic Acid and Doxycycline

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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. ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01855360

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United States, Massachusetts
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Brigham and Women's Hospital
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Principal Investigator: Rodney H Falk, MD Brigham and Women's Hospital, Boston MA
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Responsible Party: Rodney H. Falk, MD, Director, Brigham and Women's Hospital Cardiac Amyloidosis Program, Brigham and Women's Hospital Identifier: NCT01855360    
Other Study ID Numbers: BWHAMY1
First Posted: May 16, 2013    Key Record Dates
Last Update Posted: June 20, 2017
Last Verified: June 2017
Keywords provided by Rodney H. Falk, MD, Brigham and Women's Hospital:
cardiac amyloidosis
senile amyloidosis
familial amyloidosis
transthyretin amyloidosis
Additional relevant MeSH terms:
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Heart Diseases
Cardiovascular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Tauroursodeoxycholic acid
Anti-Bacterial Agents
Anti-Infective Agents
Antiprotozoal Agents
Antiparasitic Agents
Antiviral Agents
Cholagogues and Choleretics
Gastrointestinal Agents