HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

• ClinicalTrials.gov Identifier: NCT03569891
• Recruitment Status: Active, not recruiting
• First Posted: June 26, 2018
• Results First Posted: October 10, 2022
• Last Update Posted: March 6, 2023
• Sponsor: CSL Behring
• Information provided by (Responsible Party): CSL Behring

• Study Type: Interventional
• Study Design: Allocation: Non-Randomized; Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Hemophilia B
• Interventions: Genetic: AAV5-hFIXco-Padua; Biological: Factor IX (FIX)
• Enrollment: 67

Participant Flow

Recruitment Details	This is an 18-month post-dose interim analysis due to the completion of the primary endpoint. The study will complete approximately 5 years after post-dose.
Pre-assignment Details	Sixty seven (67) participants were enrolled. Of those, 54 completed the Lead-in and were dosed with AMT-061.

Arm/Group Title	Lead-in Safety Population
Arm/Group Description	This is an 18-month post-dose interim analysis due to the completion of the primary endpoint. The study will complete approximately 5 years after post-dose. The Lead-in Safety Population consisted of all subjects who were enrolled into the lead-in phase. During the lead-in, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of Factor IX (FIX) replacement therapy and bleeding episodes in their dedicated e-diary. AMT-061 was administered after the Lead-in Period.
Period Title: Lead-in
Started	67
Completed	54
Not Completed	13
Reason Not Completed
Ineligible during lead-in	8
Withdrawal by Subject	3
Risks related to COVID-19	2
Period Title: AMT-061
Started	54
Completed	53
Not Completed	1
Reason Not Completed
Adverse Event	1

Baseline Characteristics

Arm/Group Title		Safety Population
Arm/Group Description		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary. After the lead-in phase, subjects received a single-dose of AMT-061.
Overall Number of Baseline Participants		67
Baseline Analysis Population Description		[Not Specified]
Age, Categorical; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	67 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	56 83.6%
	>=65 years	11 16.4%
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	67 participants
		42.8 (16.2)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	67 participants
	Female	0 0.0%
	Male	67 100.0%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	67 participants
	Hispanic or Latino	6 9.0%
	Not Hispanic or Latino	56 83.6%
	Unknown or Not Reported	5 7.5%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	67 participants
	American Indian or Alaska Native	0 0.0%
	Asian	3 4.5%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	2 3.0%
	White	50 74.6%
	More than one race	7 10.4%
	Unknown or Not Reported	5 7.5%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants	Number Analyzed	67 participants
Netherlands		13
Sweden		2
Belgium		6
United States		26
Ireland		3
United Kingdom		7
Italy		5
Germany		2
Denmark		3

Outcome Measures

1. Primary Outcome

Title	Annualized Bleeding Rate (ABR) for All Bleeding Episodes
Description	ABR was calculated as the ratio of the number of bleeds to the number of days in the time interval multiplied by 365.25.
Time Frame	Lead-in period and months 7-18 post-treatment of AMT-061

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS) included all subjects who were enrolled, entered the lead-in phase, were dosed with AMT-061, and provided at least 1 efficacy endpoint assessment for any efficacy endpoint subsequent to AMT-061 dosing. Subjects who were Lead-in Discontinuers were not included in the FAS.

Arm/Group Title	FIX (Lead-in)	AMT-061
Arm/Group Description:	During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed	54	54
Mean (95% Confidence Interval); Unit of Measure: bleeds/year/subject
	4.19; (3.22 to 5.45)	1.51; (0.81 to 2.82)

2. Secondary Outcome

Title	Factor IX Activity Levels After AMT-061 Dosing
Description	[Not Specified]
Time Frame	Baseline and 6,12, and 18 months after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS.

Arm/Group Title		FIX (Lead-in)	AMT-061
Arm/Group Description:		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed		0	54
Mean (Standard Deviation); Unit of Measure: Factor IX activity (%)
Baseline	Number Analyzed	0 participants	54 participants
			1.19 (0.39)
6 months	Number Analyzed	0 participants	51 participants
			38.95 (18.72)
12 months	Number Analyzed	0 participants	50 participants
			41.48 (21.71)
18 months	Number Analyzed	0 participants	50 participants
			36.90 (21.40)

3. Secondary Outcome

Title	Annualized Exogenous Factor IX Consumption
Description	[Not Specified]
Time Frame	Lead-in period and months 0-6, 7-12, and 13-18 after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title		FIX (Lead-in)	AMT-061
Arm/Group Description:		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed		54	54
Mean (Standard Deviation); Unit of Measure: IU/year
Lead-in	Number Analyzed	54 participants	0 participants
		257,338.8 (149,013.1)
Months 0-6	Number Analyzed	0 participants	54 participants
			12,912.9 (37,093.1)
Months 7-12	Number Analyzed	0 participants	54 participants
			8399.1 (29,720.9)
Months 13-18	Number Analyzed	0 participants	54 participants
			8486.6 (28,770.2)

4. Secondary Outcome

Title	Adjusted Annualized Infusion Rate of FIX Replacement Therapy
Description	[Not Specified]
Time Frame	Lead-in period and months 7-18 after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS.

Arm/Group Title		FIX (Lead-in)	AMT-061
Arm/Group Description:		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed		54	54
Mean (95% Confidence Interval); Unit of Measure: Infusions/year
Lead-in	Number Analyzed	54 participants	0 participants
		72.49; (63.52 to 82.71)
Months 7-18	Number Analyzed	0 participants	54 participants
			2.53; (0.92 to 6.96)

5. Secondary Outcome

Title	Percent of Subjects Who Discontinued FIX Prophylaxis and Remained Free of Routine FIX Prophylaxis After AMT-061 Dosing
Description	[Not Specified]
Time Frame	Months 7-18 after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title	AMT-061
Arm/Group Description:	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed	54
Measure Type: Number; Unit of Measure: percentage of participants
	96.3

6. Secondary Outcome

Title	Percentage of Subjects With Trough FIX Activity <12% of Normal
Description	[Not Specified]
Time Frame	Lead-in and 3, 12, and 18 months after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title		FIX (Lead-in)	AMT-061
Arm/Group Description:		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed		54	54
Measure Type: Number; Unit of Measure: percentage of participants
Lead-in	Number Analyzed	54 participants	0 participants
		79.6
3 months	Number Analyzed	0 participants	51 participants
			7.8
12 months	Number Analyzed	0 participants	50 participants
			8
18 months	Number Analyzed	0 participants	50 participants
			6

7. Secondary Outcome

Title	ABR for FIX-treated Bleeding Episodes
Description	[Not Specified]
Time Frame	Lead-in and Months 7-18 after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title	FIX (Lead-in)	AMT-061
Arm/Group Description:	During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed	54	54
Mean (95% Confidence Interval); Unit of Measure: bleeds/year/subject
	3.65; (2.82 to 4.74)	0.84; (0.41 to 1.73)

8. Secondary Outcome

Title	Number of Spontaneous Bleeding Episodes
Description	[Not Specified]
Time Frame	Lead-in period and months 7-18 after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title		FIX (Lead-in)	AMT-061
Arm/Group Description:		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in .
Overall Number of Participants Analyzed		54	54
Measure Type: Number; Unit of Measure: Number of bleeds
Lead-in	Number Analyzed	54 participants	0 participants
		50
Months 7-18	Number Analyzed	0 participants	54 participants
			14

9. Secondary Outcome

Title	Number of Joint Bleeding Episodes
Description	[Not Specified]
Time Frame	Lead-in period and months 7-18 after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title		FIX (Lead-in)	AMT-061
Arm/Group Description:		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed		54	54
Measure Type: Number; Unit of Measure: Number of bleeds
Lead-in	Number Analyzed	54 participants	0 participants
		77
Months 7-18	Number Analyzed	0 participants	54 participants
			19

10. Secondary Outcome

Title	Mean FIX Activity (%) in Subjects With Pre-Existing Neutralizing Antibodies to AAV5 After AMT-061 Dosing
Description	[Not Specified]
Time Frame	Baseline and 6,12, and 18 months after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title		FIX (Lead-in)	AMT-061
Arm/Group Description:		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed		0	21
Mean (Standard Deviation); Unit of Measure: Factor IX activity (%)
Baseline	Number Analyzed	0 participants	21 participants
			1.24 (0.44)
6 months	Number Analyzed	0 participants	18 participants
			35.91 (19.02)
12 months	Number Analyzed	0 participants	18 participants
			35.54 (17.84)
18 months	Number Analyzed	0 participants	17 participants
			31.14 (13.75)

11. Secondary Outcome

Title	Mean FIX Activity (%) in Subjects Without Pre-Existing Neutralizing Antibodies to AAV5 After AMT-061 Dosing
Description	[Not Specified]
Time Frame	Baseline and 6,12, and 18 months after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title		FIX (Lead-in)	AMT-061
Arm/Group Description:		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed		0	33
Mean (Standard Deviation); Unit of Measure: Factor IX activity (%)
Baseline	Number Analyzed	0 participants	33 participants
			1.15 (0.36)
6 months	Number Analyzed	0 participants	33 participants
			40.61 (18.64)
12 months	Number Analyzed	0 participants	32 participants
			44.82 (23.21)
18 months	Number Analyzed	0 participants	33 participants
			39.87 (24.08)

12. Secondary Outcome

Title	Number of New Target Joints and the Number of New Target Joints Resolved.
Description	A target joint was defined as 3 or more spontaneous bleeding episodes into a single joint within a consecutive 6-month period prior to the dosing visit and which was not resolved by the time of dosing. An identified target joint with ≤2 spontaneous bleeding episodes within a consecutive 12-month period was considered resolved.
Time Frame	Up to 18 months after AT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title	FIX (Lead-in)	AMT-061
Arm/Group Description:	During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed	0	54
Measure Type: Number; Unit of Measure: Joints
New target joints		1
New target joints resolved		0

13. Secondary Outcome

Title	Percent of Participants With Zero Bleeding Episodes During the 52 Weeks Following Stable FIX Expression (6 to 18 Months) After AMT-061 Dosing
Description	[Not Specified]
Time Frame	Lead-in period and months 7-18 post-treatment of AMT-061

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title	FIX (Lead-in)	AMT-061
Arm/Group Description:	During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed	54	54
Measure Type: Number; Unit of Measure: percentage of participants
	25.9	63.0

14. Secondary Outcome

Title	International Physical Activity Questionnaire (iPAQ) Overall Score
Description	The iPAQ was designed to provide an evaluation of daily physical activities in metabolic equivalent of task (MET) minutes/week. To calculate MET minutes a week multiply the MET value given (walking = 3.3, moderate activity = 4, vigorous activity = 8) by the minutes the activity was carried out and again by the number of days that that activity was undertaken. A higher score is considered to be more favorable.
Time Frame	Lead-in period and up to 12 months after AT-01 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title	FIX (Lead-in)	AMT-061
Arm/Group Description:	During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed	54	54
Least Squares Mean (Standard Error); Unit of Measure: MET minutes/week
	4548.1 (512.38)	3826.9 (480.44)

15. Secondary Outcome

Title	EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) VAS Overall Score
Description	The EQ-5D-5L descriptive system of health-related QoL states consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). The EQ-5D-5L VAS overall score ranges from 0 to 100. A higher score is considered to be more favorable.
Time Frame	Lead-in period and up to 12 months after AMT-061 dosing

Outcome Measure Data

Analysis Population Description

FAS

Arm/Group Title	FIX (Lead-in)	AMT-061
Arm/Group Description:	During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.	Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline after lead-in.
Overall Number of Participants Analyzed	54	54
Least Squares Mean (Standard Error); Unit of Measure: score on a scale
	80.9 (2.20)	81.0 (2.15)

16. Secondary Outcome

Title	Number of Adverse Events
Description	Follow up and assess any adverse events reported for safety
Time Frame	5 years

Outcome Measure Data Not Reported

Adverse Events

Time Frame	Up to 2 years per subject (up to 6 months Lead-in and up to an additional 18 months post-dose)
Adverse Event Reporting Description	This is an 18-month post-dose interim analysis due to the completion of the primary endpoint. The study will complete approximately 5 years after post-dose. The Safety Population consisted of all subjects who were enrolled in either the Lead-in Safety Population (consisted of all subjects who were enrolled into the lead-in phase) or the Post-treatment Safety Population (consisted of all subjects who received AMT-061, irrespective of any protocol deviations).
Arm/Group Title	Lead-In (Including Discontinuers)		Lead-In (Not Including Discontinuers)		AMT-061
Arm/Group Description	During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.		During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.		Single infusion of AMT-061 after lead-in.
All-Cause Mortality
	Lead-In (Including Discontinuers)		Lead-In (Not Including Discontinuers)		AMT-061
	Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)
Total	0/67 (0.00%)		0/54 (0.00%)		1/54 (1.85%)
Serious Adverse Events
	Lead-In (Including Discontinuers)		Lead-In (Not Including Discontinuers)		AMT-061
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	5/67 (7.46%)		4/54 (7.41%)		13/54 (24.07%)
Cardiac disorders
Acute myocardial infarction †	1/67 (1.49%)	1	0/54 (0.00%)	0	0/54 (0.00%)	0
Atrial fibrillation †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Cardiogenic shock †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Gastrointestinal disorders
Gastrointestinal haemorrhage †	1/67 (1.49%)	1	1/54 (1.85%)	1	0/54 (0.00%)	0
Lower gastrointestinal haemorrhage †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Upper gastrointestinal haemorrhage †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
General disorders
Complication Associated With Device †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Infections and infestations
COVID-19 †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Cellulitis †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Injury, poisoning and procedural complications
Jaw fracture †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Musculoskeletal and connective tissue disorders
Haemarthrosis †	1/67 (1.49%)	1	1/54 (1.85%)	1	1/54 (1.85%)	1
Haemophilic arthropathy †	1/67 (1.49%)	1	1/54 (1.85%)	1	0/54 (0.00%)	0
Muscle haemorrhage †	1/67 (1.49%)	1	1/54 (1.85%)	1	0/54 (0.00%)	0
Pseudarthrosis †	1/67 (1.49%)	1	1/54 (1.85%)	1	0/54 (0.00%)	0
Musculoskeletal chest pain †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatocellular carcinoma †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Nervous system disorders
Epilepsy †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Transient ischaemic attack †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
Renal and urinary disorders
Acute kidney injury †	1/67 (1.49%)	1	0/54 (0.00%)	0	0/54 (0.00%)	0
Nephrolithiasis †	0/67 (0.00%)	0	0/54 (0.00%)	0	1/54 (1.85%)	1
† Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Lead-In (Including Discontinuers)		Lead-In (Not Including Discontinuers)		AMT-061
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	27/67 (40.30%)		24/54 (44.44%)		53/54 (98.15%)
Blood and lymphatic system disorders
Anaemia †	2/67 (2.99%)	2	1/54 (1.85%)	1	4/54 (7.41%)	4
Iron deficiency anaemia †	2/67 (2.99%)	2	2/54 (3.70%)	2	3/54 (5.56%)	3
Gastrointestinal disorders
Nausea †	2/67 (2.99%)	2	2/54 (3.70%)	2	6/54 (11.11%)	6
Diarrhoea †	1/67 (1.49%)	1	1/54 (1.85%)	1	6/54 (11.11%)	6
Toothache †	2/67 (2.99%)	2	2/54 (3.70%)	2	4/54 (7.41%)	7
Abdominal pain upper †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	4
Haemorrhoids †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	3
General disorders
Fatigue †	0/67 (0.00%)	0	0/54 (0.00%)	0	14/54 (25.93%)	16
Influenza like illness †	1/67 (1.49%)	1	1/54 (1.85%)	1	7/54 (12.96%)	11
Pain †	2/67 (2.99%)	2	1/54 (1.85%)	1	4/54 (7.41%)	4
Malaise †	0/67 (0.00%)	0	0/54 (0.00%)	0	5/54 (9.26%)	7
Chest pain †	0/67 (0.00%)	0	0/54 (0.00%)	0	4/54 (7.41%)	4
Chills †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	3
Pyrexia †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	4
Infections and infestations
Nasopharyngitis †	8/67 (11.94%)	8	8/54 (14.81%)	8	15/54 (27.78%)	20
Cystitis †	2/67 (2.99%)	2	2/54 (3.70%)	2	3/54 (5.56%)	3
COVID-19 †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	3
Influenza †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	3
Injury, poisoning and procedural complications
Ligament sprain †	1/67 (1.49%)	1	1/54 (1.85%)	1	4/54 (7.41%)	4
Infusion related reaction †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	3
Limb injury †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	4
Investigations
Alanine aminotransferase increased †	0/67 (0.00%)	0	0/54 (0.00%)	0	11/54 (20.37%)	12
Blood creatine phosphokinase increased †	0/67 (0.00%)	0	0/54 (0.00%)	0	8/54 (14.81%)	10
Aspartate aminotransferase increased †	0/67 (0.00%)	0	0/54 (0.00%)	0	7/54 (12.96%)	8
C-reactive protein increased †	0/67 (0.00%)	0	0/54 (0.00%)	0	4/54 (7.41%)	4
Musculoskeletal and connective tissue disorders
Arthralgia †	5/67 (7.46%)	5	4/54 (7.41%)	4	18/54 (33.33%)	31
Back pain †	1/67 (1.49%)	1	1/54 (1.85%)	1	7/54 (12.96%)	10
Pain in extremity †	1/67 (1.49%)	1	1/54 (1.85%)	1	6/54 (11.11%)	7
Myalgia †	1/67 (1.49%)	1	1/54 (1.85%)	1	4/54 (7.41%)	4
Arthritis †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	3
Nervous system disorders
Headache †	0/67 (0.00%)	0	0/54 (0.00%)	0	16/54 (29.63%)	31
Dizziness †	0/67 (0.00%)	0	0/54 (0.00%)	0	4/54 (7.41%)	4
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain †	3/67 (4.48%)	3	2/54 (3.70%)	2	7/54 (12.96%)	7
Cough †	1/67 (1.49%)	1	0/54 (0.00%)	0	6/54 (11.11%)	6
Rhinorrhoea †	0/67 (0.00%)	0	0/54 (0.00%)	0	3/54 (5.56%)	3
Vascular disorders
Hypertension †	1/67 (1.49%)	1	1/54 (1.85%)	1	4/54 (7.41%)	4
† Indicates events were collected by systematic assessment

Limitations and Caveats

This is an 18-month post-dose interim analysis due to the completion of the primary endpoint. The study will complete approximately 5 years after post-dose.

More Information

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: Study Director
• Organization: CSL Behring
• Phone: 610-878-4000
• EMail: clinicaltrials@cslbehring.com

• Responsible Party: CSL Behring
• ClinicalTrials.gov Identifier: NCT03569891
• Other Study ID Numbers: CSL222_3001 (CT-AMT-061-02)
• First Submitted: June 14, 2018
• First Posted: June 26, 2018
• Results First Submitted: August 15, 2022
• Results First Posted: October 10, 2022
• Last Update Posted: March 6, 2023