Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

• Status: Completed
• Trial end date: 2015-05-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Brigham and Women's Hospital
• Treatments: Doxycycline; Taurochenodeoxycholic Acid; Tauroursodeoxycholic acid; Ursodoxicoltaurine

Criteria

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