Tolerability and Efficacy of a Combination of Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy
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.
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
TUDCA taken orally, 250 mg three times daily. Doxycycline taken orally, 100 mg twice daily
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.
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.
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
|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|
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