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APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01960348
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 efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis

Condition Intervention Phase
TTR-mediated Amyloidosis Amyloidosis, Hereditary Amyloid Neuropathies, Familial Familial Amyloid Polyneuropathies Amyloid Neuropathies Amyloidosis, Hereditary, Transthyretin-Related Drug: patisiran (ALN-TTR02) Drug: Sterile Normal Saline (0.9% NaCl) Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)

Resource links provided by NLM:


Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • The difference between the ALN TTR02 and placebo groups in the change from baseline of modified Neuropathy Impairment Score+7 (mNIS+7) [ Time Frame: 18 months ]

Secondary Outcome Measures:
  • The difference between ALN-TTR02 and placebo in the change from baseline in quality of life [ Time Frame: 18 months ]
  • The difference between ALN-TTR02 and placebo in the change from baseline in motor function [ Time Frame: 18 months ]
  • The difference between ALN-TTR02 and placebo in the change from baseline in autonomic function [ Time Frame: 18 months ]

Enrollment: 225
Study Start Date: November 2013
Estimated Study Completion Date: August 2017
Estimated Primary Completion Date: July 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: patisiran (ALN-TTR02) Drug: patisiran (ALN-TTR02)
administered by intravenous (IV) infusion
Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
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:

  • Male or female of 18 to 85 years of age (inclusive);
  • Have a diagnosis of FAP
  • Neuropathy Impairment Score requirement of 5-130
  • Meet Karnofsky performance status requirements
  • Have adequate complete blood counts and liver function tests
  • Have adequate cardiac function
  • Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV)

Exclusion Criteria:

  • Had a prior liver transplant or is planned to undergo liver transplant during the study period;
  • Has untreated hypo- or hyperthyroidism;
  • Has known human immunodeficiency virus (HIV) infection;
  • Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated;
  • Recently received an investigational agent or device
  • Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid
  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: NCT01960348

  Hide Study Locations
Locations
United States, California
Clinical Trial Site
La Mesa, California, United States
Clinical Trial Site
Orange, California, United States
United States, Colorado
Clinical Trial Site
Denver, Colorado, United States
United States, Illinois
Clinical Trial Site
Chicago, Illinois, United States
United States, Maryland
Clinical Trial Site
Baltimore, Maryland, United States
United States, Massachusetts
Clinical Trial Site
Boston, Massachusetts, United States
United States, Michigan
Clinical Trial Site
Detroit, Michigan, United States
United States, Minnesota
Clinical Trial Site
Rochester, Minnesota, United States
United States, Missouri
Clinical Trial Site
St. Louis, Missouri, United States
United States, New York
Clinical Trial Site
New York, New York, United States, 10029
Clinical Trial Site
New York, New York, United States, 10032
United States, North Carolina
Clinical Trial Site
Durham, North Carolina, United States
United States, Oregon
Clinical Trial Site
Portland, Oregon, United States
Argentina
Clinical Trial Site
Buenos Aires, Argentina
Australia
Clinical Trial Site
Westmead, Australia
Brazil
Clinical Trial Site
Ribeirao Preto, Brazil
Clinical Trial Site
Rio de Janeiro, Brazil
Clinical Trial Site
Sao Paulo, Brazil
Bulgaria
Clinical Trial Site
Sofia, Bulgaria
Canada, British Columbia
Clinical Trial Site
Vancouver, British Columbia, Canada
Cyprus
Clinical Trial Site
Nicosia, Cyprus
France
Clinical Trial Site
Creteil, France
Clinical Trial Site
Le Kremlin-bicetre, France
Clinical Trial Site
Lille Cedex, France
Clinical Trial Site
Marseille Cedex, France
Germany
Clinical Trial Site
Heidelberg, Germany
Clinical Trial Site
Muenster, Germany
Clinical Trial Site
Regensburg, Germany
Italy
Clinical Trial Site
Pavia, Italy
Clinical Trial Site
Rome, Italy
Clinical Trial Site
Sicily, Italy
Japan
Clinical Trial Site
Matsumoto, Nagano, Japan
Clinical Trial Site
Aichi, Japan
Clinical Trial Site
Kumamoto, Japan
Korea, Republic of
Clinical Trial Site
Seoul, Korea, Republic of, 135-710
Clinical Trial Site
Seoul, Korea, Republic of, 143-729
Malaysia
Clinical Trial Site
Kuala Lumpur, Malaysia
Mexico
Clinical Trial Site
Mexico City, Mexico
Netherlands
Clinical Trial Site
Groningen, Netherlands
Portugal
Clinical Trial Site
Lisbon, Portugal
Clinical Trial Site
Porto, Portugal
Spain
Clinical Trial Site
Barcelona, Spain
Clinical Trial Site
Huelva, Spain
Clinical Trial Site
Madrid, Spain
Clinical Trial Site
Majorca, Spain
Sweden
Clinical Trial Site
Umeå, Sweden
Taiwan
Clinical Trial Site
Taipai, Taiwan, 11217
Clinical Trial Site
Taipei, Taiwan, 10002
Turkey
Clinical Trial Site
Istanbul, Turkey
United Kingdom
Clinical Trial Site
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Jared Gollob Alnylam Pharmaceuticals
  More Information

Additional Information:
Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01960348     History of Changes
Other Study ID Numbers: ALN-TTR02-004
2013-002987-17 ( EudraCT Number )
Study First Received: October 9, 2013
Last Updated: October 17, 2016

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
FAP
Familial Amyloid Polyneuropathy
TTR
Transthyretin
Amyloidosis

Additional relevant MeSH terms:
Amyloidosis
Polyneuropathies
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Amyloidosis, Familial
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on August 18, 2017