Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

NCT01855360 | Completed Phase 1 Results

• 1 other identifier: BWHAMY1
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Amyloidosis; Heart (Manifestation); Senile Cardiac Amyloidosis

Keywords

amyloid; amyloidosis; cardiac amyloidosis; senile amyloidosis; familial amyloidosis; transthyretin amyloidosis

Outcome Measures

Primary Outcomes (1)

• Changes in Strain Echocardiography

  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.

  Time Frame: * (FDAAA) outcome measure is assessed every 6 months by serial echocardiography, with final measurement 18 months after enrollment, change at 12 months reported as pre-defined primary endpoint.

Secondary Outcomes (1)

• Number of Patients With Adverse Events

  18 months

Study Arms (1)

TUDCA and Doxycycline

EXPERIMENTAL

INTERVENTION: Patients meeting study criteria were prescribed TUDCA taken orally, 250 mg three times daily. and doxycycline taken orally, 100 mg twice daily.

Drug: Tauroursodeoxycholic Acid and Doxycycline

Interventions

Tauroursodeoxycholic Acid and Doxycycline DRUG

Also known as: Doxycycline:Vibramycin, Monodox, Acticlate, Atridox, Avidoxy, Doxy, Doxycin, Doryx, Oracea, Periostat, Adoxa, Ocudox, and Doryx MPC.

TUDCA and Doxycycline

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Brigham and Women's Hospital: lead

Study Sites (1)

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Amyloidosis; Amyloid Neuropathies, Familial; Alzheimer Disease; Amyloidosis, Familial; Amyloidosis, Hereditary, Transthyretin-Related

Interventions

ursodoxicoltaurine; Doxycycline

Condition Hierarchy (Ancestors)

Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Dementia; Brain Diseases; Central Nervous System Diseases; Tauopathies; Neurocognitive Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Tetracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Results Point of Contact

• Title: Rodney H Falk
• Organization: Brigham and WQomen's Hospital

rfalk@bwh.harvard.edu

6175257053

Study Officials

• Rodney H Falk, MD

  Brigham and Women's Hospital, Boston MA

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Brigham and Women's Hospital Cardiac Amyloidosis Program

Study Record Dates

• First Submitted: March 12, 2013
• First Posted: May 16, 2013
• Study Start: June 1, 2013
• Primary Completion: May 31, 2015
• Study Completion: May 31, 2015
• Last Updated: December 4, 2020
• Results First Posted: December 4, 2020

Record last verified: 2018-11

Locations

US (1)