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Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

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ClinicalTrials.gov Identifier: NCT01617967
Recruitment Status : Completed
First Posted : June 13, 2012
Results First Posted : November 21, 2018
Last Update Posted : November 21, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition TTR-mediated Amyloidosis
Intervention Drug: Patisiran
Enrollment 29
Recruitment Details A total of 29 subjects were enrolled
Pre-assignment Details  
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W Patisiran 0.300 mg/kg Q3W Patisiran 0.300 mg/kg Q3W Alternative
Hide Arm/Group Description Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W). Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks. Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks. Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks. Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W). Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Period Title: Overall Study
Started 4 3 3 7 3 9
Completed 3 3 3 6 3 8
Not Completed 1 0 0 1 0 1
Reason Not Completed
Protocol Violation             0             0             0             1             0             0
Withdrawal by Subject             0             0             0             0             0             1
Dosing Suspended by Sponsor             1             0             0             0             0             0
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W Patisiran 0.300 mg/kg Q3W Patisiran 0.300 mg/kg Q3W Alternative Total
Hide Arm/Group Description Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W). Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks. Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks. Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks. Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W). Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen. Total of all reporting groups
Overall Number of Baseline Participants 4 3 3 7 3 9 29
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 3 participants 3 participants 7 participants 3 participants 9 participants 29 participants
65.8  (8.96) 55.7  (24.83) 41.7  (2.52) 58.7  (16.07) 51.0  (16.09) 54.7  (16.34) 55.6  (15.61)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 3 participants 3 participants 7 participants 3 participants 9 participants 29 participants
Female
1
  25.0%
0
   0.0%
1
  33.3%
4
  57.1%
1
  33.3%
2
  22.2%
9
  31.0%
Male
3
  75.0%
3
 100.0%
2
  66.7%
3
  42.9%
2
  66.7%
7
  77.8%
20
  69.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 3 participants 3 participants 7 participants 3 participants 9 participants 29 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
4
 100.0%
3
 100.0%
3
 100.0%
7
 100.0%
3
 100.0%
9
 100.0%
29
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation
Hide Description The number of participants experiencing at least one adverse event (AE), at least one serious adverse event (SAE) and study drug discontinuation (due to any reason).
Time Frame Up to 56 days post first dose
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-treat (ITT) population included all participants, who received at least 1 dose of study drug.
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W Patisiran 0.300 mg/kg Q3W Patisiran 0.300 mg/kg Q3W Alternative
Hide Arm/Group Description:
Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).
Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Overall Number of Participants Analyzed 4 3 3 7 3 9
Measure Type: Count of Participants
Unit of Measure: Participants
At Least 1 AE
1
  25.0%
3
 100.0%
2
  66.7%
7
 100.0%
3
 100.0%
7
  77.8%
At Least 1 SAE
0
   0.0%
0
   0.0%
0
   0.0%
1
  14.3%
0
   0.0%
1
  11.1%
Study Drug Discontinuation For Any Reason
1
  25.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
  11.1%
2.Secondary Outcome
Title Percentage Change From Baseline in Serum Transthyretin (TTR) Protein
Hide Description Percentage change of TTR relative to pretreatment/baseline levels is reported. For arms with a dosing regimen of Q4W TTR protein samples were measured on Days 28 and 56. For the arms with a dosing regimen of Q3W TTR protein samples were measured on Days 21 and 42.
Time Frame Baseline to Day 21/28 and Day 42/56 depending on dosing regimen (Q3W/Q4W)
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants, who received at least 1 dose of study drug.
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W Patisiran 0.300 mg/kg Q3W Patisiran 0.300 mg/kg Q3W Alternative
Hide Arm/Group Description:
Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).
Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Overall Number of Participants Analyzed 4 3 3 7 3 8
Mean (Standard Deviation)
Unit of Measure: Percentage
Day 21 or 28 Number Analyzed 4 participants 3 participants 3 participants 7 participants 3 participants 8 participants
-8.9  (9.91) -21.7  (1.69) -51.6  (22.84) -73.9  (8.41) -78.0  (7.55) -80.7  (10.14)
Day 42 or 56 Number Analyzed 3 participants 3 participants 3 participants 6 participants 3 participants 8 participants
-20.3  (20.27) -14.6  (15.8) -44.7  (12.11) -62.8  (25.11) -78.7  (9.77) -73.3  (19.55)
3.Secondary Outcome
Title Pharmacokinetic Parameters of Patisiran - Area Under the Concentration Curve From Time 0 to Last Measurable Time Point (AUC0-last)
Hide Description Pharmacokinetic profiles for patisiran (ALN-TTR02) were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.
Time Frame Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W)
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W All Patisiran 0.300 mg/kg Q3W
Hide Arm/Group Description:
Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).
Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.
All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.
Overall Number of Participants Analyzed 4 3 3 7 12
Mean (Standard Deviation)
Unit of Measure: ng*h/mL
Day 0 Number Analyzed 4 participants 3 participants 3 participants 7 participants 12 participants
2738  (3159) 9604  (10588) 18998  (5066) 53724  (35814) 39741  (33373)
Day 21 or Day 28 Number Analyzed 3 participants 3 participants 3 participants 6 participants 11 participants
2799  (2645) 4884  (4652) 27748  (17081) 30013  (15935) 25958  (28460)
4.Secondary Outcome
Title Pharmacokinetic Parameters of Patisiran - Maximum Observed Plasma Concentration (Cmax)
Hide Description Pharmacokinetic profiles for patisiran (ALN-TTR02) were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.
Time Frame Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing regimen (Q3W/Q4W)
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W All Patisiran 0.300 mg/kg Q3W
Hide Arm/Group Description:
Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).
Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.
All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.
Overall Number of Participants Analyzed 4 3 3 7 12
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 0 Number Analyzed 4 participants 3 participants 3 participants 7 participants 12 participants
145  (23.4) 672  (473) 2560  (295) 6053  (1326) 4539  (1362)
Day 21 or 28 Number Analyzed 3 participants 3 participants 3 participants 6 participants 11 participants
106  (36.6) 683  (391) 3243  (986) 3782  (1259) 3314  (1253)
5.Secondary Outcome
Title Pharmacokinetic Parameters of Patisiran - Beta Elimination Half-life (t1/2 Beta)
Hide Description Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.
Time Frame Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W All Patisiran 0.300 mg/kg Q3W
Hide Arm/Group Description:
Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).
Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.
All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.
Overall Number of Participants Analyzed 3 3 3 7 11
Mean (Standard Deviation)
Unit of Measure: hour (h)
Day 0 Number Analyzed 3 participants 3 participants 2 participants 7 participants 11 participants
59.5  (59.3) 39.6  (34.8) 52.2 [1]   (NA) 48  (23.7) 59.3  (17.2)
Day 21 or 28 Number Analyzed 2 participants 3 participants 3 participants 6 participants 9 participants
88.1 [1]   (NA) 39.4  (34.8) 46.9  (6.05) 50.2  (32.7) 53.8  (15.2)
[1]
Standard deviation not calculated for 2 participants
6.Secondary Outcome
Title Pharmacokinetic Parameters of Patisiran - Systemic Clearance (CL)
Hide Description Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.
Time Frame Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W All Patisiran 0.300 mg/kg Q3W
Hide Arm/Group Description:
Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).
Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.
All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.
Overall Number of Participants Analyzed 3 3 3 7 11
Mean (Standard Deviation)
Unit of Measure: L/h/kg
Day 0 Number Analyzed 3 participants 3 participants 2 participants 7 participants 11 participants
0.0163  (0.0248) 0.123  (0.205) 0.00883 [1]   (NA) 0.00851  (0.00606) 0.0130  (0.0105)
Day 21 or 28 Number Analyzed 3 participants 3 participants 3 participants 6 participants 11 participants
0.0211  (0.0325) 0.0844  (0.134) 0.00658  (0.00324) 0.0190  (0.0233) 0.0222  (0.0138)
[1]
Standard deviation not calculated for 2 participants
7.Secondary Outcome
Title Pharmacokinetic Parameters of Patisiran - Apparent Volume of Distribution at Steady State (Vss)
Hide Description Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.
Time Frame Predose, end of infusion and post-infusion at 5 minutes (min), 10 min, 30 min, 1 hour (h), 2 h, 4 h, 6 h, 24 h and 48 h on Day 0 and Day 21/28 depending on dosing frequency
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W All Patisiran 0.300 mg/kg Q3W
Hide Arm/Group Description:
Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).
Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.
All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.
Overall Number of Participants Analyzed 3 3 3 7 11
Mean (Standard Deviation)
Unit of Measure: L/kg
Day 0 Number Analyzed 3 participants 3 participants 2 participants 7 participants 11 participants
0.169  (0.092) 0.279  (0.0896) 0.428 [1]   (NA) 0.360  (0.197) 0.588  (0.305)
Day 21 or 28 Number Analyzed 2 participants 2 participants 3 participants 5 participants 9 participants
0.264 [1]   (NA) 0.397 [1]   (NA) 0.305  (0.163) 0.587  (0.251) 0.881  (0.539)
[1]
Standard deviation not calculated for 2 participants
8.Secondary Outcome
Title Pharmacokinetic Parameters of Patisiran - Renal Clearance (CLR)
Hide Description Pharmacokinetic profiles for patisiran were determined based on dosage and dosing frequency only, not premedication regimens. For arms with a dosing regimen of Q4W PK samples were taken on Days 0 and 28. For the arm with a dosing regimen of Q3W PK samples were taken on Days 0 and 21.
Time Frame Predose (within 1 h of planned dosing start) and post-infusion at 0-6 h (pooled) on Day 0 and Day 21/28 depending on dosing frequency
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic (PK) population included all participants, who received at least one dose of patisiran and had adequate data to determine a full pharmacokinetic profile. For PK outcome measures the two arms for patisiran 0.300 mg/kg at a dosing frequency of Q3W were combined and reported irrespective of premedication regimen.
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W All Patisiran 0.300 mg/kg Q3W
Hide Arm/Group Description:
Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).
Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.
Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.
All participants, who received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks irrespective of premedication regimen.
Overall Number of Participants Analyzed 3 3 3 7 11
Mean (Standard Deviation)
Unit of Measure: mL/h/kg
Day 0 Number Analyzed 3 participants 3 participants 3 participants 7 participants 11 participants
0.0153  (0.0307) 0.0268  (0.0465) 0.0129  (0.0223) 0.0329  (0.0500) 0.0334  (0.0631)
Day 21 or 28 Number Analyzed 3 participants 3 participants 3 participants 5 participants 9 participants
0.0320  (0.0555) 0.253  (0.439) 0.0410  (0.0710) 0.0652  (0.0893) 0.0563  (0.0979)
Time Frame From first dose of study drug up to Day 56
Adverse Event Reporting Description ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
 
Arm/Group Title Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W Patisiran 0.300 mg/kg Q3W Patisiran 0.300 mg/kg Q3W Alternative
Hide Arm/Group Description Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W). Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks. Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks. Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks. Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W). Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
All-Cause Mortality
Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W Patisiran 0.300 mg/kg Q3W Patisiran 0.300 mg/kg Q3W Alternative
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/3 (0.00%)   0/3 (0.00%)   0/7 (0.00%)   0/3 (0.00%)   0/9 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W Patisiran 0.300 mg/kg Q3W Patisiran 0.300 mg/kg Q3W Alternative
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/3 (0.00%)   0/3 (0.00%)   1/7 (14.29%)   0/3 (0.00%)   1/9 (11.11%) 
Gastrointestinal disorders             
Nausea  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Vomiting  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Infections and infestations             
Sepsis  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Cellulitis  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Urinary tract infection  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
1
Term from vocabulary, MedDRA (15.0)
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%
Patisiran 0.010 mg/kg Q4W Patisiran 0.050 mg/kg Q4W Patisiran 0.150 mg/kg Q4W Patisiran 0.300 mg/kg Q4W Patisiran 0.300 mg/kg Q3W Patisiran 0.300 mg/kg Q3W Alternative
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/4 (25.00%)   3/3 (100.00%)   2/3 (66.67%)   7/7 (100.00%)   3/3 (100.00%)   7/9 (77.78%) 
Blood and lymphatic system disorders             
Anaemia  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Leukocytosis  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  1/3 (33.33%)  0/9 (0.00%) 
Neutrophilia  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  1/3 (33.33%)  0/9 (0.00%) 
Cardiac disorders             
Tachyarrhythmia  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Tachycardia  1  0/4 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  0/9 (0.00%) 
Ventricular arrhythmia  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Eye disorders             
Conjunctival haemorrhage  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  1/3 (33.33%)  0/9 (0.00%) 
Gastrointestinal disorders             
Abdominal pain  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Abdominal pain upper  1  0/4 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  0/9 (0.00%) 
Diarrhoea  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  1/9 (11.11%) 
Dry mouth  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Dysphagia  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  1/3 (33.33%)  1/9 (11.11%) 
Nausea  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Vomiting  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  2/9 (22.22%) 
General disorders             
Asthenia  1  0/4 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  1/9 (11.11%) 
Extravasation  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Fatigue  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  2/9 (22.22%) 
Pyrexia  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  3/9 (33.33%) 
Vessel puncture site haematoma  1  0/4 (0.00%)  1/3 (33.33%)  2/3 (66.67%)  2/7 (28.57%)  1/3 (33.33%)  1/9 (11.11%) 
Immune system disorders             
Infusion related reaction  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  3/7 (42.86%)  0/3 (0.00%)  0/9 (0.00%) 
Infections and infestations             
Cellulitis  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Herpes virus infection  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Influenza  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Lymphangitis  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Nasopharyngitis  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Pharyngitis  1  0/4 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/7 (0.00%)  1/3 (33.33%)  0/9 (0.00%) 
Urinary tract infection  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  1/3 (33.33%)  2/9 (22.22%) 
Vulvovaginal mycotic infection  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Injury, poisoning and procedural complications             
Traumatic haematoma  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Metabolism and nutrition disorders             
Hyperglycaemia  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Musculoskeletal and connective tissue disorders             
Back pain  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  2/7 (28.57%)  0/3 (0.00%)  1/9 (11.11%) 
Neck pain  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Pain in extremity  1  1/4 (25.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  0/9 (0.00%) 
Temporomandibular joint syndrome  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  1/3 (33.33%)  0/9 (0.00%) 
Nervous system disorders             
Neuralgia  1  0/4 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Presyncope  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Psychiatric disorders             
Anxiety  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  1/9 (11.11%) 
Insomnia  1  0/4 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  0/9 (0.00%) 
Renal and urinary disorders             
Glycosuria  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Polyuria  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/7 (14.29%)  0/3 (0.00%)  0/9 (0.00%) 
Renal impairment  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Respiratory, thoracic and mediastinal disorders             
Oropharyngeal pain  1  0/4 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  0/9 (0.00%) 
Skin and subcutaneous tissue disorders             
Erythema  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
Pruritus  1  0/4 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/7 (0.00%)  0/3 (0.00%)  1/9 (11.11%) 
1
Term from vocabulary, MedDRA (15.0)
Indicates events were collected by systematic assessment
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: 1-877-256-9526
Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01617967     History of Changes
Other Study ID Numbers: ALN-TTR02-002
2012-000467-24 ( EudraCT Number )
First Submitted: June 7, 2012
First Posted: June 13, 2012
Results First Submitted: August 30, 2018
Results First Posted: November 21, 2018
Last Update Posted: November 21, 2018