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

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ClinicalTrials.gov Identifier: NCT01960348
Recruitment Status : Completed
First Posted : October 10, 2013
Last Update Posted : January 29, 2018
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis

Condition or disease Intervention/treatment 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  (Clinical Trial)
Actual Enrollment : 225 participants
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)
Study Start Date : November 2013
Primary Completion Date : August 2017
Study Completion Date : August 2017

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. 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 :
  1. The difference between ALN-TTR02 and placebo in the change from baseline in quality of life [ Time Frame: 18 months ]
  2. The difference between ALN-TTR02 and placebo in the change from baseline in motor function [ Time Frame: 18 months ]
  3. The difference between ALN-TTR02 and placebo in the change from baseline in autonomic function [ Time Frame: 18 months ]

Information from the National Library of Medicine

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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01960348

  Show 52 Study Locations
Sponsors and Collaborators
Alnylam Pharmaceuticals
Study Director: Jared Gollob Alnylam Pharmaceuticals

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01960348     History of Changes
Other Study ID Numbers: ALN-TTR02-004
2013-002987-17 ( EudraCT Number )
First Posted: October 10, 2013    Key Record Dates
Last Update Posted: January 29, 2018
Last Verified: January 2018

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
Familial Amyloid Polyneuropathy

Additional relevant MeSH terms:
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