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The Study of an Investigational Drug, ALN-TTR02 (Patisiran), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01961921
First received: October 9, 2013
Last updated: October 17, 2016
Last verified: October 2016
  Purpose
The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

Condition Intervention Phase
TTR-mediated Amyloidosis
Drug: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients With Familial Amyloidotic Polyneuropathy Who Have Previously Received ALN-TTR02

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • Safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in ATTR patients [ Time Frame: Up to 56 days post last dose ]

Secondary Outcome Measures:
  • Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in neurologic impairment associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in quality of life and disability associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in motor function associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in nutritional status associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]

Enrollment: 27
Study Start Date: October 2013
Study Completion Date: August 2016
Primary Completion Date: July 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ALN-TTR02 (patisiran) Drug: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion

  Eligibility

Ages Eligible for Study:   18 Years to 85 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Previously received and tolerated ALN-TTR02 (patisiran) in Study ALN-TTR02-002.
  • Adequate Karnofsky performance status, liver function, and renal function.

Exclusion Criteria:

  • Pregnant or nursing.
  • Has had a liver transplant.
  • Has a New York Heart Association heart failure classification >2.
  • Has unstable angina.
  • Has uncontrolled clinically significant cardiac arrhythmia.
  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: NCT01961921

Locations
United States, Massachusetts
Clinical Trial Site
Boston, Massachusetts, United States
Brazil
Clinical Trial Site
Rio de Janeiro, Brazil
France
Clinical Trial Site
Le Kremlin-bicetre, France
Clinical Trial Site
Marseille Cedex, France
Germany
Clinical Trial Site
Munster, Germany
Portugal
Clinical Trial Site
Lisbon, Portugal
Clinical Trial Site
Porto, Portugal
Spain
Clinical Trial Site
Majorca, Spain
Sweden
Clinical Trial Site
Umeå, Sweden
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
  More Information

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01961921     History of Changes
Other Study ID Numbers: ALN-TTR02-003
2013-001644-65 ( EudraCT Number )
Study First Received: October 9, 2013
Last Updated: October 17, 2016

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on April 28, 2017