This trial is active, not recruiting.

Conditions amyloidosis; heart (manifestation), senile cardiac amyloidosis
Treatment tauroursodeoxycholic acid and doxycycline
Phase phase 1/phase 2
Sponsor Brigham and Women's Hospital
Start date June 2013
End date July 2016
Trial size 40 participants
Trial identifier NCT01855360, BWHAMY1


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
TUDCA taken orally, 250 mg three times daily. Doxycycline taken orally, 100 mg twice daily
tauroursodeoxycholic acid and doxycycline

Primary Outcomes

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.

Secondary Outcomes

Number of patients with adverse events to the medications over the period of therapy (18 months)
time frame: 18 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.

Eligibility Criteria

Male or female participants from 18 years up to 90 years old.

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

Additional Information

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.
Principal investigator Rodney H Falk, MD
Trial information was received from ClinicalTrials.gov and was last updated in January 2016.
Information provided to ClinicalTrials.gov by Brigham and Women's Hospital.