Phase 2 N=40 Treatment

Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

Amyloidosis; Heart (Manifestation) · Senile Cardiac Amyloidosis

Bottom Line

View on ClinicalTrials.gov: NCT01855360 ↗

Enrolled (actual)

Serious AEs

2.5%

Results posted

Dec 2020

Primary outcome: Primary: Changes in Strain Echocardiography — 9.1 percent LV shortening

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Tauroursodeoxycholic Acid and Doxycycline (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Brigham and Women's Hospital
Primary completion: May 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Changes in Strain Echocardiography	9.1	—
SECONDARY Number of Patients With Adverse Events	7	—

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.

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01855360). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.