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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 : December 20, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
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 Design

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 : July 2017
Study Completion Date : August 2017


Arms and Interventions

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


Outcome Measures

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 ]

Eligibility Criteria

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
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

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
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 )
First Posted: October 10, 2013    Key Record Dates
Last Update Posted: December 20, 2017
Last Verified: December 2017

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