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ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

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ClinicalTrials.gov Identifier: NCT02319005
Recruitment Status : Completed
First Posted : December 18, 2014
Results First Posted : July 18, 2018
Last Update Posted : July 18, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Conditions Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
Amyloidosis, Hereditary
Amyloid Neuropathies, Familial
Amyloid Neuropathies
Amyloidosis, Hereditary, Transthyretin-Related
Familial Transthyretin Cardiac Amyloidosis
Interventions Drug: Revusiran (ALN-TTRSC)
Drug: Sterile Normal Saline (0.9% NaCl)
Enrollment 206
Recruitment Details A total of 206 patients with hereditary amyloid transthyretin (hATTR) cardiac amyloidosis were enrolled and randomized in the study.
Pre-assignment Details  
Arm/Group Title Revusiran (ALN-TTRSC) Placebo
Hide Arm/Group Description All patients who received at least 1 dose of revusiran All patients who received at least 1 dose of placebo
Period Title: Overall Study
Started 140 66
Completed 92 51
Not Completed 48 15
Arm/Group Title Revusiran Placebo Total
Hide Arm/Group Description All patients who received at least 1 dose of revusiran All patients who received at least 1 dose of placebo Total of all reporting groups
Overall Number of Baseline Participants 140 66 206
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 140 participants 66 participants 206 participants
68.6  (9.28) 66.2  (9.52) 67.8  (9.40)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 140 participants 66 participants 206 participants
Female
35
  25.0%
13
  19.7%
48
  23.3%
Male
105
  75.0%
53
  80.3%
158
  76.7%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 140 participants 66 participants 206 participants
Hispanic or Latino
5
   3.6%
0
   0.0%
5
   2.4%
Not Hispanic or Latino
129
  92.1%
65
  98.5%
194
  94.2%
Unknown or Not Reported
6
   4.3%
1
   1.5%
7
   3.4%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 140 participants 66 participants 206 participants
American Indian or Alaska Native
0
   0.0%
1
   1.5%
1
   0.5%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
68
  48.6%
36
  54.5%
104
  50.5%
White
66
  47.1%
29
  43.9%
95
  46.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
6
   4.3%
0
   0.0%
6
   2.9%
1.Primary Outcome
Title 6 Minute Walk Distance (6-MWD)
Hide Description The difference between revusiran and placebo group in change from baseline to 18 months in the total distance walked in 6 minutes
Time Frame 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study drug was discontinued early due to an imbalance in mortality observed between patients treated with revusiran and placebo. No patients had an 18-month visit, therefore, the endpoints cannot be calculated as data were not collected.
Arm/Group Title Revusiran Placebo
Hide Arm/Group Description:
All patients who received at least 1 dose of revusiran
All patients who received at least 1 dose of placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
2.Primary Outcome
Title Serum TTR Levels
Hide Description The difference between revusiran (ALN-TTRSC) and placebo group in the percent reduction in serum TTR levels over 18 months
Time Frame 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study drug was discontinued early due to an imbalance in mortality observed between patients treated with revusiran and placebo. No patients had an 18-month visit, therefore, the endpoints cannot be calculated as data were not collected.
Arm/Group Title Revusiran Placebo
Hide Arm/Group Description:
All patients who received at least 1 dose of revusiran
All patients who received at least 1 dose of placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Composite Cardiovascular (CV) Mortality and Cardiovascular (CV) Hospitalization
Hide Description Number of cardiovascular-related deaths and cardiovascular-related hospitalizations in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
Time Frame 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study drug was discontinued early due to an imbalance in mortality observed between patients treated with revusiran and placebo. No patients had an 18-month visit, therefore, the endpoints cannot be calculated as data were not collected.
Arm/Group Title Revusiran Placebo
Hide Arm/Group Description:
All patients who received at least 1 dose of revusiran
All patients who received at least 1 dose of placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title New York Heart Association (NYHA) Class
Hide Description The difference between revusiran (ALN-TTRSC) and placebo group in the change from baseline to 18 months in the NYHA class
Time Frame 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study drug was discontinued early due to an imbalance in mortality observed between patients treated with revusiran and placebo. No patients had an 18-month visit, therefore, the endpoints cannot be calculated as data were not collected.
Arm/Group Title Revusiran Placebo
Hide Arm/Group Description:
All patients who received at least 1 dose of revusiran
All patients who received at least 1 dose of placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Kansas City Cardiomyopathy Questionnaire (KCCQ)
Hide Description The difference between revusiran (ALN-TTRSC) and placebo group in the change from Baseline to 18 months in the Kansas City Cardiomyopathy Questionnaire
Time Frame 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study drug was discontinued early due to an imbalance in mortality observed between patients treated with revusiran and placebo. No patients had an 18-month visit, therefore, the endpoints cannot be calculated as data were not collected.
Arm/Group Title Revusiran Placebo
Hide Arm/Group Description:
All patients who received at least 1 dose of revusiran
All patients who received at least 1 dose of placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
6.Secondary Outcome
Title Cardiovascular (CV) Mortality
Hide Description Number of cardiovascular-related deaths in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
Time Frame 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study drug was discontinued early due to an imbalance in mortality observed between patients treated with revusiran and placebo. No patients had an 18-month visit, therefore, the endpoints cannot be calculated as data were not collected.
Arm/Group Title Revusiran Placebo
Hide Arm/Group Description:
All patients who received at least 1 dose of revusiran
All patients who received at least 1 dose of placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Cardiovascular (CV) Hospitalization
Hide Description Number of cardiovascular-related hospitalizations in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
Time Frame 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study drug was discontinued early due to an imbalance in mortality observed between patients treated with revusiran and placebo. No patients had an 18-month visit, therefore, the endpoints cannot be calculated as data were not collected.
Arm/Group Title Revusiran Placebo
Hide Arm/Group Description:
All patients who received at least 1 dose of revusiran
All patients who received at least 1 dose of placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
8.Secondary Outcome
Title All-cause Mortality
Hide Description Total number of deaths in the placebo group compared to the revusiran (ALN-TTRSC) treatment group
Time Frame 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
Study drug was discontinued early due to an imbalance in mortality observed between patients treated with revusiran and placebo. No patients had an 18-month visit, therefore, the endpoints cannot be calculated as data were not collected.
Arm/Group Title Revusiran Placebo
Hide Arm/Group Description:
All patients who received at least 1 dose of revusiran
All patients who received at least 1 dose of placebo
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame All adverse events (AEs) that occurred after the start of study drug administration on Day 0 (Baseline) up to 90 days post modified early termination visit (End of Study); Median duration of collection was 10 months
Adverse Event Reporting Description The same event may appear as both an AE and a serious adverse event (SAE). However, what is presented are distinct events. An event may be categorized as serious in one subject and as nonserious in another subject, or one subject may have experienced both a serious and nonserious event during the study
 
Arm/Group Title Revusiran Placebo
Hide Arm/Group Description All patients who received at least 1 dose of revusiran All patients who received at least 1 dose of placebo
All-Cause Mortality
Revusiran Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   23/140 (16.43%)      7/66 (10.61%)    
Show Serious Adverse Events Hide Serious Adverse Events
Revusiran Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   83/140 (59.29%)      34/66 (51.52%)    
Blood and lymphatic system disorders     
Pancytopenia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Anaemia  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Heparin-induced thrombocytopenia  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Spleen disorders  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Cardiac disorders     
Cardiac failure  1  25/140 (17.86%)  35 9/66 (13.64%)  13
Cardiac failure acute  1  15/140 (10.71%)  20 9/66 (13.64%)  12
Cardiac failure congestive  1  9/140 (6.43%)  12 4/66 (6.06%)  6
Atrial fibrillation  1  7/140 (5.00%)  8 2/66 (3.03%)  3
Atrial flutter  1  2/140 (1.43%)  2 4/66 (6.06%)  4
Cardiac failure chronic  1  4/140 (2.86%)  4 2/66 (3.03%)  2
Cardiac arrest  1  3/140 (2.14%)  3 1/66 (1.52%)  1
Ventricular tachycardia  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Atrioventricular block complete  1  1/140 (0.71%)  1 1/66 (1.52%)  1
Cardiogenic shock  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Cardiac amyloidosis  1  1/140 (0.71%)  1 1/66 (1.52%)  1
Arrhythmia supraventricular  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Cardiomyopathy  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Cardiorenal syndrome  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Atrial Tachycardia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Tachycardia  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Pericardial effusion  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Ventricular fibrillation  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Low cardiac output syndrome  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Ear and labyrinth disorders     
Vertigo positional  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Gastrointestinal disorders     
Impaired gastric emptying  1  6/140 (4.29%)  6 0/66 (0.00%)  0
Diarrhoea  1  2/140 (1.43%)  2 1/66 (1.52%)  1
Vomiting  1  2/140 (1.43%)  2 1/66 (1.52%)  1
Dysphagia  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Constipation  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Gastrointestinal amyloidosis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Pancreatitis acute  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Large intestinal obstruction  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Nausea  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Pancreatitis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Small intestinal obstruction  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Ascites  1  1/140 (0.71%)  1 0/66 (0.00%)  0
General disorders     
Death  1  1/140 (0.71%)  1 1/66 (1.52%)  1
Asthenia  1  1/140 (0.71%)  1 1/66 (1.52%)  1
Chest pain  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Hypothermia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Malaise  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Systematic inflammatory response syndrome  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Peripheral swelling  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Medical device complication  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Fatigue  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Non-cardiac chest pain  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Hepatobiliary disorders     
Cholecystitis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Cholangitis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Pneumobilia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Cholelithiasis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Hepatic function abnormal  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Hepatitis cholestatic  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Infections and infestations     
Pneumonia  1  3/140 (2.14%)  3 0/66 (0.00%)  0
Sepsis  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Urinary tract infection  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Nocardiosis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Epididymitis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Infective tenosynovitis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Cellulitis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Diverticulitis  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Gastroenteritis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Infectious pleural effusion  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Urosepsis  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Bronchopneumonia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Herpes zoster  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Escherichia infection  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Injury, poisoning and procedural complications     
Craniocerebral injury  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Fall  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Drug administration error  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Investigations     
Weight decreased  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Liver function test abnormal  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Hepatic enzyme abnormal  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Hepatic enzyme increased  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Metabolism and nutrition disorders     
Dehydration  1  3/140 (2.14%)  3 3/66 (4.55%)  3
Malnutrition  1  4/140 (2.86%)  4 0/66 (0.00%)  0
Hyponatraemia  1  2/140 (1.43%)  3 1/66 (1.52%)  1
Failure to thrive  1  2/140 (1.43%)  2 1/66 (1.52%)  1
Hypokalaemia  1  1/140 (0.71%)  1 2/66 (3.03%)  2
Cachexia  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Hyperglycaemic hyperosmolar nonketotic syndrome  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Hyperkalaemia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Diabetes mellitus inadequate control  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Lactic acidosis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Hypervolaemia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Osteoarthritis  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Lumbar spinal stenosis  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Muscular weakness  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Pain in extremity  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Foot deformity  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Prostate cancer  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Breast cancer  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Neuroendocrine tumor  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Cholangiocarcinoma  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Metastases to liver  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Pancreatic carcinoma  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Gallbladder cancer  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Metastases to biliary tract  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Clear cell renal cell carcinoma  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Nervous system disorders     
Neuropathy peripheral  1  7/140 (5.00%)  7 0/66 (0.00%)  0
Syncope  1  4/140 (2.86%)  4 3/66 (4.55%)  3
Presyncope  1  0/140 (0.00%)  0 2/66 (3.03%)  4
Subarachnoid hemorrhage  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Cerebrovascular accident  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Convulsion  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Amnesia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Central nervous system lesions  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Cerebral infarction  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Ischaemic Stroke  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Psychiatric disorders     
Mental status changes  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Renal and urinary disorders     
Renal failure acute  1  5/140 (3.57%)  7 2/66 (3.03%)  2
Renal failure  1  3/140 (2.14%)  3 1/66 (1.52%)  1
Urinary retention  1  1/140 (0.71%)  1 2/66 (3.03%)  2
Obstructive uropathy  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Haematuria  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Renal impairment  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Renal failure chronic  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Reproductive system and breast disorders     
Benign prostatic hyperplasia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Pleural effusion  1  1/140 (0.71%)  1 1/66 (1.52%)  2
Acute respiratory failure  1  2/140 (1.43%)  2 0/66 (0.00%)  0
Chronic obstructive pulmonary disease  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Dyspnoea  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Pleuritic pain  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Hypoxia  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Respiratory failure  1  1/140 (0.71%)  1 0/66 (0.00%)  0
Epistaxis  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Skin and subcutaneous tissue disorders     
Cutaneous Vasculitis  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Surgical and medical procedures     
Lipoma excision  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Vascular disorders     
Hypotension  1  3/140 (2.14%)  3 2/66 (3.03%)  2
Peripheral artery aneurysm  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Hypertensive crisis  1  0/140 (0.00%)  0 1/66 (1.52%)  1
Orthostatic hypotensions  1  0/140 (0.00%)  0 1/66 (1.52%)  1
1
Term from vocabulary, MedDRA (17.1)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Revusiran Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   136/140 (97.14%)      62/66 (93.94%)    
Cardiac disorders     
Cardiac failure  1  10/140 (7.14%)  14 3/66 (4.55%)  3
Atrial fibrillation  1  16/140 (11.43%)  20 2/66 (3.03%)  3
Cardiac failure congestive  1  4/140 (2.86%)  4 6/66 (9.09%)  9
Gastrointestinal disorders     
Constipation  1  20/140 (14.29%)  23 11/66 (16.67%)  13
Nausea  1  20/140 (14.29%)  28 5/66 (7.58%)  7
Vomiting  1  9/140 (6.43%)  18 5/66 (7.58%)  7
Abdominal pain  1  7/140 (5.00%)  10 3/66 (4.55%)  5
Diarrhoea  1  9/140 (6.43%)  9 5/66 (7.58%)  5
Abdominal pain upper  1  9/140 (6.43%)  9 1/66 (1.52%)  1
General disorders     
Injection Site Pain  1  23/140 (16.43%)  101 4/66 (6.06%)  13
Injection site erythema  1  16/140 (11.43%)  64 0/66 (0.00%)  0
Fatigue  1  14/140 (10.00%)  16 8/66 (12.12%)  32
Asthenia  1  15/140 (10.71%)  18 2/66 (3.03%)  2
Infections and infestations     
Nasopharyngitis  1  11/140 (7.86%)  11 5/66 (7.58%)  5
Bronchitis  1  10/140 (7.14%)  11 3/66 (4.55%)  3
Sinusitis  1  7/140 (5.00%)  8 3/66 (4.55%)  4
Upper respiratory tract infection  1  8/140 (5.71%)  8 4/66 (6.06%)  4
Injury, poisoning and procedural complications     
Fall  1  10/140 (7.14%)  16 5/66 (7.58%)  9
Investigations     
Weight decreased  1  12/140 (8.57%)  14 4/66 (6.06%)  5
Blood lactic acid increased  1  15/140 (10.71%)  16 3/66 (4.55%)  3
Blood bilirubin increased  1  10/140 (7.14%)  11 4/66 (6.06%)  4
Liver function test abnormal  1  12/140 (8.57%)  12 1/66 (1.52%)  1
Metabolism and nutrition disorders     
Hypokalaemia  1  6/140 (4.29%)  10 8/66 (12.12%)  12
Decreased appetite  1  13/140 (9.29%)  16 6/66 (9.09%)  6
Gout  1  3/140 (2.14%)  4 7/66 (10.61%)  16
Fluid overload  1  6/140 (4.29%)  6 4/66 (6.06%)  6
Musculoskeletal and connective tissue disorders     
Pain in extremity  1  14/140 (10.00%)  19 8/66 (12.12%)  9
Arthralgia  1  9/140 (6.43%)  10 6/66 (9.09%)  8
Muscle spasms  1  9/140 (6.43%)  11 2/66 (3.03%)  3
Nervous system disorders     
Dizziness  1  18/140 (12.86%)  19 13/66 (19.70%)  15
Neuropathy peripheral  1  20/140 (14.29%)  21 6/66 (9.09%)  6
Hypoaesthesia  1  12/140 (8.57%)  21 4/66 (6.06%)  5
Psychiatric disorders     
Insomnia  1  8/140 (5.71%)  8 2/66 (3.03%)  2
Renal and urinary disorders     
Renal failure acute  1  9/140 (6.43%)  9 1/66 (1.52%)  3
Respiratory, thoracic and mediastinal disorders     
Cough  1  25/140 (17.86%)  29 10/66 (15.15%)  14
Dyspnoea  1  16/140 (11.43%)  22 9/66 (13.64%)  9
Productive cough  1  9/140 (6.43%)  9 1/66 (1.52%)  1
Skin and subcutaneous tissue disorders     
Pruritus  1  9/140 (6.43%)  9 2/66 (3.03%)  2
Vascular disorders     
Hypotension  1  11/140 (7.86%)  15 4/66 (6.06%)  4
1
Term from vocabulary, MedDRA (17.1)
Indicates events were collected by systematic assessment
While dosing of patients was discontinued early, a protocol amendment allowed additional safety follow-up visits. Per the new assessment schedule, this study did not meet the criteria of a Terminated study, as additional follow-up visits occurred.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Chief Medical Officer
Organization: Alnylam Pharmaceuticals Inc
Phone: 866-330-0326
Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02319005     History of Changes
Other Study ID Numbers: ALN-TTRSC-004
First Submitted: December 12, 2014
First Posted: December 18, 2014
Results First Submitted: March 27, 2018
Results First Posted: July 18, 2018
Last Update Posted: July 18, 2018