Trial record 3 of 21 for:    "transthyretin amyloidosis"

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2013 by Brigham and Women's Hospital
Sponsor:
Information provided by (Responsible Party):
Rodney H. Falk, MD, Brigham and Women's Hospital
ClinicalTrials.gov Identifier:
NCT01855360
First received: March 12, 2013
Last updated: November 19, 2013
Last verified: May 2013
  Purpose

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 Intervention Phase
Amyloidosis; Heart (Manifestation)
Senile Cardiac Amyloidosis
Drug: Tauroursodeoxycholic Acid and Doxycycline
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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.

Resource links provided by NLM:


Further study details as provided by Brigham and Women's Hospital:

Primary Outcome Measures:
  • 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. ] [ Designated as safety issue: No ]
    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:
  • Number of patients with adverse events to the medications over the period of therapy (18 months) [ Time Frame: 18 months ] [ Designated as safety issue: Yes ]
    Measurements of kidney function (BUN and creatinine) and blood counts will be done every 3 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. ] [ Designated as safety issue: No ]

Estimated Enrollment: 40
Study Start Date: June 2013
Estimated Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: TUDCA and Doxycycline
TUDCA taken orally, 250 mg three times daily. Doxycycline taken orally, 100 mg twice daily
Drug: Tauroursodeoxycholic Acid and Doxycycline

  Eligibility

Ages Eligible for Study:   18 Years to 90 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01855360

Contacts
Contact: Rodney H Falk, MD 617 421 6094 rfalk@partners.org

Locations
United States, Massachusetts
Brigham and Women's Hospital/ Harvard Vanguard Medical Associates Recruiting
Boston, Massachusetts, United States, 02115
Contact: Rodney H Falk, MD    617-421-6094    rfalk@partners.org   
Principal Investigator: Rodney H Falk, MD         
Sponsors and Collaborators
Brigham and Women's Hospital
Investigators
Principal Investigator: Rodney H Falk, MD Brigham and Women's Hospital, Boston MA
  More Information

No publications provided

Responsible Party: Rodney H. Falk, MD, Director, Brigham and Women's Hospital Cardiac Amyloidosis Program, Brigham and Women's Hospital
ClinicalTrials.gov Identifier: NCT01855360     History of Changes
Other Study ID Numbers: BWHAMY1
Study First Received: March 12, 2013
Last Updated: November 19, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Brigham and Women's Hospital:
amyloid
amyloidosis
cardiac amyloidosis
senile amyloidosis
familial amyloidosis
transthyretin amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Cardiomyopathies
Proteostasis Deficiencies
Metabolic Diseases
Heart Diseases
Cardiovascular Diseases
Doxycycline
Tauroursodeoxycholic acid
Taurochenodeoxycholic Acid
Anti-Bacterial Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions
Antimalarials
Antiprotozoal Agents
Antiparasitic Agents
Antiviral Agents
Cholagogues and Choleretics
Gastrointestinal Agents

ClinicalTrials.gov processed this record on August 28, 2014