Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

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: January 29, 2016
Last verified: January 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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • The difference between ALN-TTR02 and placebo in the change from baseline in quality of life [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • The difference between ALN-TTR02 and placebo in the change from baseline in motor function [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • The difference between ALN-TTR02 and placebo in the change from baseline in autonomic function [ Time Frame: 18 months ] [ Designated as safety issue: No ]

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)
Genders Eligible for Study:   Both
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

  Show 48 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 
Study First Received: October 9, 2013
Last Updated: January 29, 2016
Health Authority: Sweden: Medical Products Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Spain: Agencia Española de Medicamentos y Productos Sanitarios
United States: Food and Drug Administration
South Korea: Ministry of Food and Drug Safety (MFDS)
Portugal: INFARMED, National Authority of Medicines and Health Products, IP
Germany: Federal Institute for Drugs and Medical Devices
Taiwan : Food and Drug Administration
Italy: The Italian Medicines Agency
Bulgaria: Bulgarian Drug Agency
Mexico: Ministry of Health
Japan: Pharmaceuticals and Medical Devices Agency
Turkey: Drug and Medical Device Institution
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Brazil: National Health Surveillance Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Canada: Health Canada
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Cyprus: Cyprus National Bioethics Committee
Individual Participant Data  
Plan to Share IPD: Undecided

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 September 30, 2016