Trial Outcomes & Findings for Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis (NCT NCT01617967)

Last Updated: 2024-04-19

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

29 participants

Primary outcome timeframe

Up to 56 days post first dose

Results posted on

2024-04-19

Participant Flow

A total of 29 subjects were enrolled

Participant milestones

Participant milestones
Measure	Patisiran 0.010 mg/kg Q4W Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Overall Study STARTED	4	3	3	7	3	9
Overall Study COMPLETED	3	3	3	6	3	8
Overall Study NOT COMPLETED	1	0	0	1	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Patisiran 0.010 mg/kg Q4W Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.300 mg/kg Q4W Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W Alternative Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Overall Study Protocol Violation	0	1	0
Overall Study Withdrawal by Subject	0	0	1
Overall Study Dosing Suspended by Sponsor	1	0	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patisiran 0.010 mg/kg Q4W n=4 Participants Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 Participants Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 Participants Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=3 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative n=9 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.	Total n=29 Participants Total of all reporting groups
Age, Continuous	65.8 years STANDARD_DEVIATION 8.96 • n=5 Participants	55.7 years STANDARD_DEVIATION 24.83 • n=7 Participants	41.7 years STANDARD_DEVIATION 2.52 • n=5 Participants	58.7 years STANDARD_DEVIATION 16.07 • n=4 Participants	51.0 years STANDARD_DEVIATION 16.09 • n=21 Participants	54.7 years STANDARD_DEVIATION 16.34 • n=10 Participants	55.6 years STANDARD_DEVIATION 15.61 • n=115 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	4 Participants n=4 Participants	1 Participants n=21 Participants	2 Participants n=10 Participants	9 Participants n=115 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	3 Participants n=7 Participants	2 Participants n=5 Participants	3 Participants n=4 Participants	2 Participants n=21 Participants	7 Participants n=10 Participants	20 Participants n=115 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	7 Participants n=4 Participants	3 Participants n=21 Participants	9 Participants n=10 Participants	29 Participants n=115 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants

PRIMARY outcome

Timeframe: Up to 56 days post first dose

Population: Intent-to-treat (ITT) population included all participants, who received at least 1 dose of study drug.

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).

Outcome measures

Outcome measures
Measure	Patisiran 0.010 mg/kg Q4W n=4 Participants Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 Participants Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 Participants Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=3 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative n=9 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation At Least 1 AE	1 Participants	3 Participants	2 Participants	7 Participants	3 Participants	7 Participants
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation At Least 1 SAE	0 Participants	0 Participants	0 Participants	1 Participants	0 Participants	1 Participants
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation Study Drug Discontinuation For Any Reason	1 Participants	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline to Day 21/28 and Day 42/56 depending on dosing regimen (Q3W/Q4W)

Population: ITT population included all participants, who received at least 1 dose of study drug.

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.

Outcome measures

Outcome measures
Measure	Patisiran 0.010 mg/kg Q4W n=4 Participants Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 Participants Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 Participants Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=3 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative n=8 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Percentage Change From Baseline in Serum Transthyretin (TTR) Protein Day 21 or 28	-8.9 Percentage Standard Deviation 9.91	-21.7 Percentage Standard Deviation 1.69	-51.6 Percentage Standard Deviation 22.84	-73.9 Percentage Standard Deviation 8.41	-78.0 Percentage Standard Deviation 7.55	-80.7 Percentage Standard Deviation 10.14
Percentage Change From Baseline in Serum Transthyretin (TTR) Protein Day 42 or 56	-20.3 Percentage Standard Deviation 20.27	-14.6 Percentage Standard Deviation 15.8	-44.7 Percentage Standard Deviation 12.11	-62.8 Percentage Standard Deviation 25.11	-78.7 Percentage Standard Deviation 9.77	-73.3 Percentage Standard Deviation 19.55

SECONDARY outcome

Timeframe: 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)

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Patisiran 0.010 mg/kg Q4W n=4 Participants Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 Participants Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 Participants Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=12 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Pharmacokinetic Parameters of Patisiran - Area Under the Concentration Curve From Time 0 to Last Measurable Time Point (AUC0-last) Day 0	2738 ng*h/mL Standard Deviation 3159	9604 ng*h/mL Standard Deviation 10588	18998 ng*h/mL Standard Deviation 5066	53724 ng*h/mL Standard Deviation 35814	39741 ng*h/mL Standard Deviation 33373	—
Pharmacokinetic Parameters of Patisiran - Area Under the Concentration Curve From Time 0 to Last Measurable Time Point (AUC0-last) Day 21 or Day 28	2799 ng*h/mL Standard Deviation 2645	4884 ng*h/mL Standard Deviation 4652	27748 ng*h/mL Standard Deviation 17081	30013 ng*h/mL Standard Deviation 15935	25958 ng*h/mL Standard Deviation 28460	—

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Patisiran 0.010 mg/kg Q4W n=4 Participants Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 Participants Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 Participants Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=12 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Pharmacokinetic Parameters of Patisiran - Maximum Observed Plasma Concentration (Cmax) Day 0	145 ng/mL Standard Deviation 23.4	672 ng/mL Standard Deviation 473	2560 ng/mL Standard Deviation 295	6053 ng/mL Standard Deviation 1326	4539 ng/mL Standard Deviation 1362	—
Pharmacokinetic Parameters of Patisiran - Maximum Observed Plasma Concentration (Cmax) Day 21 or 28	106 ng/mL Standard Deviation 36.6	683 ng/mL Standard Deviation 391	3243 ng/mL Standard Deviation 986	3782 ng/mL Standard Deviation 1259	3314 ng/mL Standard Deviation 1253	—

SECONDARY outcome

Timeframe: 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

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.

Outcome measures

Outcome measures
Measure	Patisiran 0.010 mg/kg Q4W n=3 Participants Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 Participants Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 Participants Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=11 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Pharmacokinetic Parameters of Patisiran - Beta Elimination Half-life (t1/2 Beta) Day 0	59.5 hour (h) Standard Deviation 59.3	39.6 hour (h) Standard Deviation 34.8	52.2 hour (h) Standard Deviation NA Standard deviation not calculated for 2 participants	48 hour (h) Standard Deviation 23.7	59.3 hour (h) Standard Deviation 17.2	—
Pharmacokinetic Parameters of Patisiran - Beta Elimination Half-life (t1/2 Beta) Day 21 or 28	88.1 hour (h) Standard Deviation NA Standard deviation not calculated for 2 participants	39.4 hour (h) Standard Deviation 34.8	46.9 hour (h) Standard Deviation 6.05	50.2 hour (h) Standard Deviation 32.7	53.8 hour (h) Standard Deviation 15.2	—

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Patisiran 0.010 mg/kg Q4W n=3 Participants Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 Participants Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 Participants Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=11 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Pharmacokinetic Parameters of Patisiran - Systemic Clearance (CL) Day 0	0.0163 L/h/kg Standard Deviation 0.0248	0.123 L/h/kg Standard Deviation 0.205	0.00883 L/h/kg Standard Deviation NA Standard deviation not calculated for 2 participants	0.00851 L/h/kg Standard Deviation 0.00606	0.0130 L/h/kg Standard Deviation 0.0105	—
Pharmacokinetic Parameters of Patisiran - Systemic Clearance (CL) Day 21 or 28	0.0211 L/h/kg Standard Deviation 0.0325	0.0844 L/h/kg Standard Deviation 0.134	0.00658 L/h/kg Standard Deviation 0.00324	0.0190 L/h/kg Standard Deviation 0.0233	0.0222 L/h/kg Standard Deviation 0.0138	—

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Patisiran 0.010 mg/kg Q4W n=3 Participants Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 Participants Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 Participants Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=11 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Pharmacokinetic Parameters of Patisiran - Apparent Volume of Distribution at Steady State (Vss) Day 0	0.169 L/kg Standard Deviation 0.092	0.279 L/kg Standard Deviation 0.0896	0.428 L/kg Standard Deviation NA Standard deviation not calculated for 2 participants	0.360 L/kg Standard Deviation 0.197	0.588 L/kg Standard Deviation 0.305	—
Pharmacokinetic Parameters of Patisiran - Apparent Volume of Distribution at Steady State (Vss) Day 21 or 28	0.264 L/kg Standard Deviation NA Standard deviation not calculated for 2 participants	0.397 L/kg Standard Deviation NA Standard deviation not calculated for 2 participants	0.305 L/kg Standard Deviation 0.163	0.587 L/kg Standard Deviation 0.251	0.881 L/kg Standard Deviation 0.539	—

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Patisiran 0.010 mg/kg Q4W n=3 Participants Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 Participants Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 Participants Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=11 Participants Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Pharmacokinetic Parameters of Patisiran - Renal Clearance (CLR) Day 0	0.0153 mL/h/kg Standard Deviation 0.0307	0.0268 mL/h/kg Standard Deviation 0.0465	0.0129 mL/h/kg Standard Deviation 0.0223	0.0329 mL/h/kg Standard Deviation 0.0500	0.0334 mL/h/kg Standard Deviation 0.0631	—
Pharmacokinetic Parameters of Patisiran - Renal Clearance (CLR) Day 21 or 28	0.0320 mL/h/kg Standard Deviation 0.0555	0.253 mL/h/kg Standard Deviation 0.439	0.0410 mL/h/kg Standard Deviation 0.0710	0.0652 mL/h/kg Standard Deviation 0.0893	0.0563 mL/h/kg Standard Deviation 0.0979	—

Adverse Events

Patisiran 0.010 mg/kg Q4W

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Patisiran 0.050 mg/kg Q4W

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Patisiran 0.150 mg/kg Q4W

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Patisiran 0.300 mg/kg Q4W

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Patisiran 0.300 mg/kg Q3W

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Patisiran 0.300 mg/kg Q3W Alternative

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Patisiran 0.010 mg/kg Q4W n=4 participants at risk Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 participants at risk Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 participants at risk Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 participants at risk Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=3 participants at risk Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative n=9 participants at risk Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Gastrointestinal disorders Nausea	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Gastrointestinal disorders Vomiting	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Sepsis	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Cellulitis	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Urinary tract infection	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.

Other adverse events

Other adverse events
Measure	Patisiran 0.010 mg/kg Q4W n=4 participants at risk Participants received 0.010 mg/kg of patisiran (ALN-TTR02) every four weeks (Q4W).	Patisiran 0.050 mg/kg Q4W n=3 participants at risk Participants received 0.050 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.150 mg/kg Q4W n=3 participants at risk Participants received 0.150 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q4W n=7 participants at risk Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every four weeks.	Patisiran 0.300 mg/kg Q3W n=3 participants at risk Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks (Q3W).	Patisiran 0.300 mg/kg Q3W Alternative n=9 participants at risk Participants received 0.300 mg/kg of patisiran (ALN-TTR02) every three weeks with alternative premedication regimen.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Erythema	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Pharyngitis	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Urinary tract infection	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	22.2% 2/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Vulvovaginal mycotic infection	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Injury, poisoning and procedural complications Traumatic haematoma	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Metabolism and nutrition disorders Hyperglycaemia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	28.6% 2/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Neck pain	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Pain in extremity	25.0% 1/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Temporomandibular joint syndrome	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Nervous system disorders Neuralgia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Nervous system disorders Presyncope	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Psychiatric disorders Anxiety	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Psychiatric disorders Insomnia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Renal and urinary disorders Glycosuria	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Renal and urinary disorders Polyuria	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Renal and urinary disorders Renal impairment	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Blood and lymphatic system disorders Anaemia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Blood and lymphatic system disorders Leukocytosis	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Blood and lymphatic system disorders Neutrophilia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Cardiac disorders Tachyarrhythmia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Cardiac disorders Tachycardia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Cardiac disorders Ventricular arrhythmia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Eye disorders Conjunctival haemorrhage	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Gastrointestinal disorders Abdominal pain	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Gastrointestinal disorders Abdominal pain upper	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Gastrointestinal disorders Diarrhoea	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Gastrointestinal disorders Dry mouth	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Gastrointestinal disorders Dysphagia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Gastrointestinal disorders Nausea	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Gastrointestinal disorders Vomiting	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	22.2% 2/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
General disorders Asthenia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
General disorders Extravasation	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
General disorders Fatigue	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	22.2% 2/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
General disorders Pyrexia	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 3/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
General disorders Vessel puncture site haematoma	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	66.7% 2/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	28.6% 2/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	33.3% 1/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	11.1% 1/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Immune system disorders Infusion related reaction	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	42.9% 3/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Cellulitis	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Herpes virus infection	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Influenza	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Lymphangitis	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.
Infections and infestations Nasopharyngitis	0.00% 0/4 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	14.3% 1/7 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/3 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.	0.00% 0/9 • From first dose of study drug up to Day 56 ITT population was the primary set for safety data and included all participants, who received at least 1 dose of study drug.

Additional Information

Chief Medical Officer

Alnylam Pharmaceuticals, Inc.

Phone: 1-877-256-9526

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60