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Clinical Trial Results:
A multinational, open-label, randomised, controlled trial to investigate efficacy and safety of NNC0365-3769 (Mim8) in adults and adolescents with haemophilia A with or without inhibitors.

Summary
EudraCT number	2020-001048-24
Trial protocol	IE SK DK DE BE AT LV LT FR NL PL PT IT
Global end of trial date	17 Dec 2024

Results information
Results version number	v1(current)
This version publication date	03 Jul 2025
First version publication date	03 Jul 2025
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

Top of page

Sponsor protocol code

NN7769-4514

ISRCTN number

-

US NCT number

NCT05053139

WHO universal trial number (UTN)

U1111-1249-4378

Other trial identifiers

Japanese trial registration number: jRCT2031210643

Sponsor organisation name

Novo Nordisk A/S

Sponsor organisation address

Novo Alle, Bagsvaerd, Denmark, 2880

Public contact

Clinical Reporting Office (2834), Novo Nordisk A/S, clinicaltrials@novonordisk.com

Scientific contact

Clinical Reporting Office (2834), Novo Nordisk A/S, clinicaltrials@novonordisk.com

Is trial part of an agreed paediatric investigation plan (PIP)

Yes

EMA paediatric investigation plan number(s)

EMEA-002762-PIP02-20

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

No

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Yes

Analysis stage

Final

Date of interim/final analysis

06 Feb 2024

Is this the analysis of the primary completion data?

No

Global end of trial reached?

Yes

Global end of trial date

17 Dec 2024

Was the trial ended prematurely?

No

Main objective of the trial

To confirm the haemostatic effect of Mim8 as treatment prophylaxis for adult and adolescent patients with haemophilia A with or without inhibitors. This will be done by: - Demonstrating superiority in number of bleeding episodes when treated with Mim8 once-weekly versus no prophylaxis for subjects on no prophylaxis treatment prior to enrolment (Comparing Arm 1 and Arm 2 main treatment period) - Demonstrating non-inferiority in number of bleeding episodes when treated with either Mim8 once-weekly or once-monthly versus treatment with coagulation factor prophylaxis during run-in for subjects on prophylaxis treatment prior to enrolment (Comparing Arm 3 and Arm 4 run-in with main treatment period)

Protection of trial subjects

The trial was conducted in accordance with the Declaration of Helsinki (Oct 2013) and ICH Good Clinical Practice, including archiving of essential documents (May 1996) and EN ISO 14155 Part 1 and 2 and FDA 21 CFR 312.120.

Background therapy

-

Evidence for comparator

-

Actual start date of recruitment

02 Dec 2021

Long term follow-up planned

No

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Austria: 5

Country: Number of subjects enrolled

Belgium: 3

Country: Number of subjects enrolled

Canada: 3

Country: Number of subjects enrolled

Switzerland: 6

Country: Number of subjects enrolled

China: 36

Country: Number of subjects enrolled

Germany: 16

Country: Number of subjects enrolled

Denmark: 6

Country: Number of subjects enrolled

Spain: 6

Country: Number of subjects enrolled

France: 10

Country: Number of subjects enrolled

United Kingdom: 15

Country: Number of subjects enrolled

India: 12

Country: Number of subjects enrolled

Ireland: 1

Country: Number of subjects enrolled

Israel: 4

Country: Number of subjects enrolled

Italy: 9

Country: Number of subjects enrolled

Japan: 12

Country: Number of subjects enrolled

Korea, Republic of: 18

Country: Number of subjects enrolled

Lithuania: 5

Country: Number of subjects enrolled

Latvia: 3

Country: Number of subjects enrolled

Mexico: 2

Country: Number of subjects enrolled

Malaysia: 6

Country: Number of subjects enrolled

Netherlands: 9

Country: Number of subjects enrolled

Poland: 25

Country: Number of subjects enrolled

Portugal: 7

Country: Number of subjects enrolled

Romania: 5

Country: Number of subjects enrolled

Russian Federation: 3

Country: Number of subjects enrolled

Saudi Arabia: 7

Country: Number of subjects enrolled

Serbia: 2

Country: Number of subjects enrolled

Slovakia: 4

Country: Number of subjects enrolled

Türkiye: 11

Country: Number of subjects enrolled

Taiwan: 4

Country: Number of subjects enrolled

United States: 20

Country: Number of subjects enrolled

South Africa: 6

Worldwide total number of subjects

281

EEA total number of subjects

114

Number of subjects enrolled per age group

In utero

0

Preterm newborn - gestational age < 37 wk

0

Newborns (0-27 days)

0

Infants and toddlers (28 days-23 months)

0

Children (2-11 years)

0

Adolescents (12-17 years)

87

Adults (18-64 years)

190

From 65 to 84 years

4

85 years and over

0

Subject disposition

Top of page

Recruitment details

This study was conducted at 100 sites that enrolled subjects in 32 countries. The study was conducted in 2 parts: main part (26 weeks) and extension part (26 weeks), With 5 randomized arms (Arm 1, 2a, 2b, 3 and 4).

Screening details

Subjects on coagulation factor prophylaxis entered run-in period (26 weeks) in Arm 3 and 4 to collect high-quality bleeding, treatment data and randomized to main part. Subjects with no prophylaxis (Arm 1, 2a and 2b) was randomized to main part directly. After main part of study, subjects continued in the extension part of the study.

Period 1 title

Main Phase (26 weeks)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

Yes

Arm1: No PPX - Mim8 PPX QW/QM

Arm description

Subjects on no prophylaxis treatment did not enter the run-in period and continued no prophylaxis (on-demand treatment with their standard of care products) treatment in the main part of the study (26 weeks). After the main part, subjects continued in the extension part of the study (26 weeks) to receive either once-weekly (QW) loading dose of 24 milligram (mg) (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 4 mg (body weight 30-<45 kg) or 9 mg (body weight >=45 kg) respectively, or once-monthly (QM) loading dose of 40 mg (body weight 30-<45 kg) or 92 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 20 mg (body weight 30 - <45 kg) or 46 mg (body weight >=45 kg) respectively, based on agreement with the investigator.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once weekly or once monthly in Arm 1 during extension phase as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing.

Arm 2a: Mim8 PPX QW

Arm description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once weekly dose in Arm 2a as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing

Arm 2b: Mim8 PPX QM

Arm description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once monthly dose in Arm 2b as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing

Arm 3: PPX - Mim8 PPX QW

Arm description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered the main part of the study, to receive loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once weekly dose in Arm 3 as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing

Arm 4: PPX- Mim8 PPX QM

Arm description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered main part of the study, to receive loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once monthly dose in Arm 4 as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing

Number of subjects in period 1	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW	Arm 4: PPX- Mim8 PPX QM
Started	18	22	21	111	109
Completed	18	21	21	105	108
Not completed	0	1	0	6	1
Physician decision	-	1	-	2	1
Consent withdrawn by subject	-	-	-	3	-
Withdrawal by Parent/Guardian	-	-	-	1	-

Period 2 title

Extension Phase (26 weeks)

Is this the baseline period?

No

Allocation method

Randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

Yes

Arm1: No PPX - Mim8 PPX QW/QM

Arm description

Subjects on no prophylaxis treatment did not enter the run-in period and continued no prophylaxis (on-demand treatment with their standard of care products) treatment in the main part of the study (26 weeks). After the main part, subjects continued in the extension part of the study (26 weeks) to receive either once-weekly (QW) loading dose of 24 milligram (mg) (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 4 mg (body weight 30-<45 kg) or 9 mg (body weight >=45 kg) respectively, or once-monthly (QM) loading dose of 40 mg (body weight 30-<45 kg) or 92 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 20 mg (body weight 30 - <45 kg) or 46 mg (body weight >=45 kg) respectively, based on agreement with the investigator.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once weekly or once monthly in Arm 1 during extension phase as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing.

Arm 2a: Mim8 PPX QW

Arm description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once weekly or once monthly in Arm 2a as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing.

Arm 2b: Mim8 PPX QM

Arm description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once-monthly dose in Arms 2b as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing

Arm 3: PPX - Mim8 PPX QW

Arm description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered the main part of the study, to receive loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once weekly dose in Arm 3 as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing

Arm 4: PPX- Mim8 PPX QM

Arm description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered main part of the study, to receive loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Arm type

Experimental

Investigational medicinal product name

NNC0365-3769 B (Mim8)

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

A loading dose was administered once, followed by once monthly dose in Arm 4 as maintenance doses. Dose amount is based on weight band of subject, whether it is a loading or maintenance dose, and the frequency of dosing

Number of subjects in period 2 ^[1]	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW	Arm 4: PPX- Mim8 PPX QM
Started	18	21	21	104	108
Arm 1: Mim8 QW	8 ^[2]	0 ^[3]	0 ^[4]	0 ^[5]	0 ^[6]
Arm1: Mim8 QM	10 ^[7]	0 ^[8]	0 ^[9]	0 ^[10]	0 ^[11]
Completed	17	21	21	104	106
Not completed	1	0	0	0	2
Consent withdrawn by subject	1	-	-	-	1
Physician decision	-	-	-	-	1

Notes
[1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
Justification: As per the data this is correct, this is a bug thrown by PharmaCM
[2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)
[3] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)
[4] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)
[5] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)
[6] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)
[7] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)
[8] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)
[9] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)
[10] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)
[11] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left.
Justification: Arm 1: Mim8 QW: The milestone is only applicable for Arm 1 (QW)

Baseline characteristics

Top of page

Reporting group title

Arm1: No PPX - Mim8 PPX QW/QM

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and continued no prophylaxis (on-demand treatment with their standard of care products) treatment in the main part of the study (26 weeks). After the main part, subjects continued in the extension part of the study (26 weeks) to receive either once-weekly (QW) loading dose of 24 milligram (mg) (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 4 mg (body weight 30-<45 kg) or 9 mg (body weight >=45 kg) respectively, or once-monthly (QM) loading dose of 40 mg (body weight 30-<45 kg) or 92 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 20 mg (body weight 30 - <45 kg) or 46 mg (body weight >=45 kg) respectively, based on agreement with the investigator.

Reporting group title

Arm 2a: Mim8 PPX QW

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 2b: Mim8 PPX QM

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 3: PPX - Mim8 PPX QW

Reporting group description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered the main part of the study, to receive loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 4: PPX- Mim8 PPX QM

Reporting group description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered main part of the study, to receive loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW	Arm 4: PPX- Mim8 PPX QM	Total
Number of subjects	18	22	21	111	109	281
Age Categorical
Units: Subjects
In utero	0	0	0	0	0	0
Preterm newborn infants (gestational age < 37 wks)	0	0	0	0	0	0
Newborns (0-27 days)	0	0	0	0	0	0
Infants and toddlers (28 days-23 months)	0	0	0	0	0	0
Children (2-11 years)	0	0	0	0	0	0
Adolescents (12-17 years)	6	6	5	37	33	87
Adults (18-64 years)	12	16	16	73	73	190
From 65-84 years	0	0	0	1	3	4
85 years and over	0	0	0	0	0	0
Age Continuous
Units: years
arithmetic mean (standard deviation)	30 ( 13 )	31 ( 16 )	33 ( 16 )	31 ( 16 )	31 ( 16 )	-
Gender Categorical
Units: Subjects
Female	1	0	2	0	1	4
Male	17	22	19	111	108	277
Race (NIH/OMB)
Units: Subjects
American Indian or Alaska Native	0	0	0	0	0	0
Asian	12	12	12	25	28	89
Native Hawaiian or Other Pacific Islander	0	0	0	1	0	1
Black or African American	1	1	2	1	2	7
White	4	9	7	72	68	160
More than one race	1	0	0	7	7	15
Unknown or Not Reported	0	0	0	5	4	9
Ethnicity (NIH/OMB)
Units: Subjects
Hispanic or Latino	0	1	2	2	2	7
Not Hispanic or Latino	17	21	19	104	103	264
Unknown or Not Reported	1	0	0	5	4	10

End points

Top of page

Reporting group title

Arm1: No PPX - Mim8 PPX QW/QM

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and continued no prophylaxis (on-demand treatment with their standard of care products) treatment in the main part of the study (26 weeks). After the main part, subjects continued in the extension part of the study (26 weeks) to receive either once-weekly (QW) loading dose of 24 milligram (mg) (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 4 mg (body weight 30-<45 kg) or 9 mg (body weight >=45 kg) respectively, or once-monthly (QM) loading dose of 40 mg (body weight 30-<45 kg) or 92 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 20 mg (body weight 30 - <45 kg) or 46 mg (body weight >=45 kg) respectively, based on agreement with the investigator.

Reporting group title

Arm 2a: Mim8 PPX QW

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 2b: Mim8 PPX QM

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 3: PPX - Mim8 PPX QW

Reporting group description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered the main part of the study, to receive loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 4: PPX- Mim8 PPX QM

Reporting group description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered main part of the study, to receive loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm1: No PPX - Mim8 PPX QW/QM

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and continued no prophylaxis (on-demand treatment with their standard of care products) treatment in the main part of the study (26 weeks). After the main part, subjects continued in the extension part of the study (26 weeks) to receive either once-weekly (QW) loading dose of 24 milligram (mg) (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 4 mg (body weight 30-<45 kg) or 9 mg (body weight >=45 kg) respectively, or once-monthly (QM) loading dose of 40 mg (body weight 30-<45 kg) or 92 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 20 mg (body weight 30 - <45 kg) or 46 mg (body weight >=45 kg) respectively, based on agreement with the investigator.

Reporting group title

Arm 2a: Mim8 PPX QW

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 2b: Mim8 PPX QM

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 3: PPX - Mim8 PPX QW

Reporting group description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered the main part of the study, to receive loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 4: PPX- Mim8 PPX QM

Reporting group description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered main part of the study, to receive loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Subject analysis set title

Arm1: No PPX - Mim8 PPX QW/QM

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on no prophylaxis treatment did not enter the run-in period and continued no prophylaxis (on-demand treatment with their standard of care products) treatment in the main part of the study (26 weeks). After the main part, subjects continued in the extension part of the study (26 weeks) to receive either once-weekly (QW) loading dose of 24 milligram (mg) (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 4 mg (body weight 30-<45 kg) or 9 mg (body weight >=45 kg) respectively, or once-monthly (QM) loading dose of 40 mg (body weight 30-<45 kg) or 92 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 20 mg (body weight 30 - <45 kg) or 46 mg (body weight >=45 kg) respectively, based on agreement with the investigator.

Subject analysis set title

Arm 2a: Mim8 PPX QW

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Subject analysis set title

Arm 2b: Mim8 PPX QM

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Subject analysis set title

Arm 3: PPX - Mim8 PPX QW (Run-in Part)

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered the main part of the study, to receive loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Subject analysis set title

Arm 3: PPX - Mim8 PPX QW (Main Part)

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks. Subjects entered the main part of the study, to receive loading dose of 24 mg (body weight 30-<45 kg) to 55 mg (body weight >=45 kg) Mim8 QW followed by maintenance dose of 4 mg (body weight 30 -<45 kg)-9 mg >=45 kg) subcutaneously. After the main part, subjects continued to receive the same dosing schedule of Mim8 QW subcutaneously in the extension part of the study (26 weeks).

Subject analysis set title

Arm 4: PPX- Mim8 PPX QM (Run-in Part)

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered main part of the study, to receive loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Subject analysis set title

Arm 4: PPX- Mim8 PPX QM (Main Part)

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered main part of the study, to receive loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Subject analysis set title

Arm 2a: Mim8 PPX QW

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of 24 mg (body weight 30-<45 kg) to 55 mg (body weight >=45 kg) Mim8 QW followed by maintenance dose of 4 mg (body weight 30 -<45 kg)-9 mg (body weight >=45 kg) subcutaneously. After the main part, subjects continued to receive the same dosing schedule of Mim8 QW subcutaneously in the extension part of the study (26 weeks).

Subject analysis set title

Arm 2b: Mim8 PPX QM

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of 40 mg (body weight 30-<45 kg)-92 mg (body weight ≥45 kg) Mim8 QM followed by maintenance dose 20 mg (body weight 30-<45 kg)-46 mg (body weight >=45 kg) subcutaneously. After the main part, subjects continued to receive the same dosing schedule of Mim8 QM subcutaneously in the extension part of the study (26 weeks).

Subject analysis set title

Arm 3: PPX - Mim8 PPX QW

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks. Subjects entered the main part of the study, to receive loading dose of 24 mg (body weight 30-<45 kg) to 55 mg (body weight >=45 kg) Mim8 QW followed by maintenance dose of 4 mg (body weight 30 -<45 kg)-9 mg >=45 kg) subcutaneously. After the main part, subjects continued to receive the same dosing schedule of Mim8 QW subcutaneously in the extension part of the study (26 weeks).

Subject analysis set title

Arm 4: PPX- Mim8 PPX QM

Subject analysis set type

Full analysis

Subject analysis set description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks. Subjects entered main part of the study, to receive loading dose of 40 mg (body weight 30-<45 kg)-92 mg (body weight ≥45 kg) Mim8 QM followed by maintenance dose 20 mg (body weight 30-<45 kg)-46 mg (body weight >=45 kg) subcutaneously. After the main part, subjects continued to receive the same dosing schedule of Mim8 QM subcutaneously in the extension part of the study (26 weeks).

Primary: No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated bleeds (Annualised Bleeding Rate)

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End point title

No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated bleeds (Annualised Bleeding Rate)

End point description

Number of treated bleeds per year (Annualised Bleeding Rate) data is reported. Annualised bleeding rate (ABR) is the number of bleeding episodes per year. Full analysis set (FAS) population included all subjects randomized.

End point type

Primary

End point timeframe

From randomisation (week 0) to end of main part (Week 26)

End point values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM
Number of subjects analysed	17	21	20
Units: Bleeding episodes per year
arithmetic mean (confidence interval 95%)	15.75 (10.70 to 23.20)	0.45 (0.18 to 1.14)	0.20 (0.06 to 0.72)

No PPX Vs Mim8 PPX QM

Comparison groups

Arm1: No PPX - Mim8 PPX QW/QM v Arm 2b: Mim8 PPX QM

Number of subjects included in analysis

37

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Negative Binomial Regression

Parameter type

Annualised bleeding rate ratio

Point estimate

0.013

Confidence interval

level

95%

sides

2-sided

lower limit

0.003

upper limit

0.048

No PPX Vs Mim8 PPX QW

Comparison groups

Arm1: No PPX - Mim8 PPX QW/QM v Arm 2a: Mim8 PPX QW

Number of subjects included in analysis

38

Analysis specification

Pre-specified

Analysis type

superiority

P-value

< 0.0001

Method

Negative binomial regression

Parameter type

Annualised bleeding rate ratio

Point estimate

0.029

Confidence interval

level

95%

sides

2-sided

lower limit

0.011

upper limit

0.078

Primary: Prophylaxis treatment (Arms 3 and 4): Number of treated bleeds (Annualised Bleeding Rate)

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End point title

Prophylaxis treatment (Arms 3 and 4): Number of treated bleeds (Annualised Bleeding Rate)

End point description

Number of treated bleeds per year (Annualised Bleeding Rate) data is reported. Annualised bleeding rate(ABR) is the number of bleeding episodes per year. Full analysis set (FAS) population included all participants randomised

End point type

Primary

End point timeframe

From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (Week 26)

End point values	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 3: PPX - Mim8 PPX QW (Main Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Main Part)
Number of subjects analysed	98	98	98	98
Units: Bleeding episodes per year
arithmetic mean (confidence interval 95%)	4.83 (3.59 to 6.51)	2.51 (1.42 to 4.42)	3.10 (2.23 to 4.29)	1.78 (1.17 to 2.71)

PPX (run-in) vs Mim8 PPX QM (main)

Statistical analysis description

The actual analyzed population is 98 subjects

Comparison groups

Arm 4: PPX- Mim8 PPX QM (Run-in Part) v Arm 4: PPX- Mim8 PPX QM (Main Part)

Number of subjects included in analysis

196

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0066

Method

Negative binomial regression

Parameter type

Annualised bleeding rate ratio

Point estimate

0.574

Confidence interval

level

95%

sides

2-sided

lower limit

0.385

upper limit

0.857

PPX (Run-in) vs Mim8 PPX QW (Main)

Statistical analysis description

The actual analyzed population is 98 subjects

Comparison groups

Arm 3: PPX - Mim8 PPX QW (Run-in Part) v Arm 3: PPX - Mim8 PPX QW (Main Part)

Number of subjects included in analysis

196

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.0406

Method

Negative binomial regression

Parameter type

Annualised bleeding rate ratio

Point estimate

0.52

Confidence interval

level

95%

sides

2-sided

lower limit

0.278

upper limit

0.973

Secondary: Arms 2a, 2b, 3 and 4: Number of injection site reactions

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End point title

Arms 2a, 2b, 3 and 4: Number of injection site reactions

End point description

Number of injection site reactions data is reported from randomisation (week 0) to end of main (week 26). Full analysis set (FAS) population included all subjects randomized.

End point type

Secondary

End point timeframe

From randomisation (week 0) to end of main (week 26)

End point values	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)
Number of subjects analysed	21	20	98	98
Units: Number of events
number (not applicable)	2	2	86	13

No statistical analyses for this end point

Secondary: Prophylaxis treatment (Arms 3 and 4): Number of treated spontaneous bleeds

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End point title

Prophylaxis treatment (Arms 3 and 4): Number of treated spontaneous bleeds

End point description

From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (Week 26). Full analysis set (FAS) population included all subjects randomised

End point type

Secondary

End point timeframe

From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (Week 26)

End point values	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 3: PPX - Mim8 PPX QW (Main Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Main Part)
Number of subjects analysed	98	98	98	98
Units: Spontaneous bleeding episodes per year
arithmetic mean (confidence interval 95%)	2.73 (1.94 to 3.86)	1.49 (0.65 to 3.42)	1.83 (1.10 to 3.04)	0.74 (0.42 to 1.32)

No statistical analyses for this end point

Secondary: Arms 2a, 2b, 3 and 4: Number of participants with anti-Mim8 antibodies

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End point title

Arms 2a, 2b, 3 and 4: Number of participants with anti-Mim8 antibodies

End point description

Number of participants with anti-Mim8 antibodies from randomisation (week 0) to end of extension (week 52) is reported. Safety analysis set (SAS) included all subjects randomly assigned to study treatment and who take at least 1 dose of study product.

End point type

Secondary

End point timeframe

From randomisation (week 0) to end of extension (week 52)

End point values	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW	Arm 4: PPX- Mim8 PPX QM
Number of subjects analysed	22	21	111	109
Units: Subjects	2	2	12	5

No statistical analyses for this end point

Secondary: No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated spontaneous bleeds (Annualised Bleeding Rate)

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End point title

No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated spontaneous bleeds (Annualised Bleeding Rate)

End point description

Number of treated spontaneous bleeds per year (ABR) data is reported from randomisation (week 0) to end of main (week 26). ABR is the number of bleeding episodes per year. Full analysis set (FAS) population included all subjects randomised

End point type

Secondary

End point timeframe

From randomisation (week 0) to end of main (Week 26)

End point values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM
Number of subjects analysed	17	21	20
Units: Spontaneous bleeding episodes per year
arithmetic mean (confidence interval 95%)	11.78 (7.48 to 18.54)	0.09 (0.01 to 0.69)	0.24 (0.07 to 0.85)

No statistical analyses for this end point

Secondary: Prophylaxis treatment (Arms 3 and 4): Number of treated joint bleeds

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End point title

Prophylaxis treatment (Arms 3 and 4): Number of treated joint bleeds

End point description

Number of treated joint bleeds data is reported from initiation of run-in (26-52 weeks prior to week 0) to week 0. Full analysis set (FAS) included all subjects randomized.

End point type

Secondary

End point timeframe

From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomization (week 0) to end of main (Week 26)

End point values	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 3: PPX - Mim8 PPX QW (Main Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Main Part)
Number of subjects analysed	98	98	98	98
Units: Spontaneous bleeding episodes per year
arithmetic mean (confidence interval 95%)	3.60 (2.62 to 4.95)	1.80 (0.91 to 3.55)	2.06 (1.43 to 2.97)	1.09 (0.60 to 1.99)

No statistical analyses for this end point

Secondary: No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated joint bleeds

Top of page

End point title

No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated joint bleeds

End point description

Number of treated joint bleeds data is reported from randomisation (week 0) to end of main (Week 26)

End point type

Secondary

End point timeframe

From randomisation (week 0) to end of main (Week 26)

End point values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM
Number of subjects analysed	17	21	20 ^[1]
Units: Joint bleeding episodes per year
arithmetic mean (confidence interval 95%)	10.60 (6.28 to 17.89)	0.48 (0.18 to 1.29)	99999 (99999 to 99999)

Notes
[1] - Since number of subjects with bleeding is zero, mean and 95% CI data is not available.

No statistical analyses for this end point

Secondary: No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated traumatic bleeds

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End point title

No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated traumatic bleeds

End point description

Number of treated traumatic bleeds data is reported from randomisation (week 0) to end of main (Week 26)

End point type

Secondary

End point timeframe

From randomisation (week 0) to end of main (Week 26)

End point values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM
Number of subjects analysed	17	20	21
Units: Traumatic bleeding episodes per year
arithmetic mean (confidence interval 95%)	1.86 (0.86 to 4.05)	0.19 (0.05 to 0.67)	99999 (99999 to 99999)

No statistical analyses for this end point

Secondary: Prophylaxis treatment (Arms 3 and 4): Number of treated traumatic bleeds

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End point title

Prophylaxis treatment (Arms 3 and 4): Number of treated traumatic bleeds

End point description

Number of treated traumatic bleeds data is reported From initiation of run-in (26-52 weeks prior to week 0) to week 0

End point type

Secondary

End point timeframe

From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomization (week 0) to end of main (Week 26)

End point values	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 3: PPX - Mim8 PPX QW (Main Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Main Part)
Number of subjects analysed	98	98	98	98
Units: Traumatic bleeding episodes per year
arithmetic mean (confidence interval 95%)	1.95 (1.39 to 2.74)	0.89 (0.58 to 1.37)	1.28 (0.94 to 1.75)	0.88 (0.57 to 1.37)

No statistical analyses for this end point

Secondary: Prophylaxis treatment (Arms 3 and 4): Number of treated target joint bleeds

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End point title

Prophylaxis treatment (Arms 3 and 4): Number of treated target joint bleeds

End point description

Number of treated target joint bleeds data is reported From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (Week 26)

End point type

Secondary

End point timeframe

From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (Week 26)

End point values	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 3: PPX - Mim8 PPX QW (Main Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Main Part)
Number of subjects analysed	98	98	98	98
Units: Spontaneous bleeding episodes per year
arithmetic mean (confidence interval 95%)	1.12 (0.62 to 2.02)	0.38 (0.14 to 1.08)	0.48 (0.21 to 1.09)	0.35 (0.11 to 1.08)

No statistical analyses for this end point

Secondary: No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated target joint bleeds

Top of page

End point title

No prophylaxis treatment (Arms 1, 2a, and 2b): Number of treated target joint bleeds

End point description

Number of treated target joint bleeds data is reported from randomisation (week 0) to end of main (Week 26)

End point type

Secondary

End point timeframe

From randomisation (week 0) to end of main (Week 26)

End point values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM
Number of subjects analysed	17	21	20
Units: Spontaneous bleeding episodes per year
arithmetic mean (confidence interval 95%)	3.91 (1.96 to 7.77)	0.26 (0.07 to 0.99)	99999 (99999 to 99999)

No statistical analyses for this end point

Secondary: (Arms 2a, 2b, 3 and 4): Mim8 plasma concentration

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End point title

(Arms 2a, 2b, 3 and 4): Mim8 plasma concentration

End point description

Mim8 plasma concentration data is presented from randomisation (week 0) to end of extension (week 52) in this endpoint. Data is reported in which subjects were a part of at any time from week 0 to week 52, not at specific time points assessed from week 0 to week 52. FAS included all subjects who were randomized

End point type

Secondary

End point timeframe

From randomisation (week 0) to end of extension (week 52)

End point values	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW	Arm 4: PPX- Mim8 PPX QM
Number of subjects analysed	22	21	109	108
Units: microgram per mililiter (μg/mL)
arithmetic mean (standard deviation)	4.93 ( 1.96 )	3.50 ( 1.42 )	4.81 ( 1.65 )	4.18 ( 1.76 )

No statistical analyses for this end point

Secondary: Prophylaxis treatment (Arms 3 and 4): Consumption of factor product per bleed treatment (number of injections)

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End point title

Prophylaxis treatment (Arms 3 and 4): Consumption of factor product per bleed treatment (number of injections)

End point description

Number of injections consumed per bleed treatment data is reported from initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (Week 26). FAS included all subjects who were randomised.

End point type

Secondary

End point timeframe

From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (Week 26).

End point values	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 3: PPX - Mim8 PPX QW (Main Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Main Part)
Number of subjects analysed	98	98	98	98
Units: Number of injections
arithmetic mean (standard deviation)	1.8 ( 2.6 )	1.8 ( 1.6 )	1.4 ( 1.0 )	1.5 ( 1.2 )

No statistical analyses for this end point

Secondary: No prophylaxis treatment (Arms 1, 2a, and 2b): Consumption of factor product per bleed treatment (number of injections)

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End point title

No prophylaxis treatment (Arms 1, 2a, and 2b): Consumption of factor product per bleed treatment (number of injections)

End point description

Consumption of factor product per bleed treatment (number of injections) is reported from randomisation (week 0) to end of main (Week 26). Full analysis set (FAS) included all subjects who were randomised.

End point type

Secondary

End point timeframe

From randomisation (week 0) to end of main (Week 26)

End point values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM
Number of subjects analysed	17	21	20
Units: Number of injections
arithmetic mean (standard deviation)	1.7 ( 1.9 )	1.2 ( 0.4 )	1.0 ( 0.0 )

No statistical analyses for this end point

Secondary: All participants (Arms 1, 2a, 2b, 3 and 4): Change in physical function domain of PedsQLTM

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End point title

All participants (Arms 1, 2a, 2b, 3 and 4): Change in physical function domain of PedsQLTM

End point description

PedsQL measures quality of life and the physical functioning domain measures physical functioning. Higher scores indicate a better quality of life and better physical functioning. Positive change indicates improvement and negative change indicates worsening. The score ranges from 0-100. FAS included all subjects who were randomised. Here, overall number of subjects analysed (N) = subjects with available data for this outcome measure.

End point type

Secondary

End point timeframe

From randomisation (week 0) to the end of the main part (week 26)

End point values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)
Number of subjects analysed	12	13	15	79	81
Units: Score points
arithmetic mean (standard deviation)	-5.5 ( 17.8 )	14.2 ( 22.0 )	20.8 ( 20.3 )	1.9 ( 13.1 )	2.1 ( 10.1 )

No statistical analyses for this end point

Secondary: Arms 1, 2a, 2b, 3 and 4: Change in participant’s treatment burden using the Hemo-TEM

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End point title

Arms 1, 2a, 2b, 3 and 4: Change in participant’s treatment burden using the Hemo-TEM

End point description

Hemophilia Treatment Experience Measure (Hemo -TEM) measures treatment burden. Higher scores indicate a greater treatment burden and negative change indicates improvement. The score ranges from 0-100. FAS included all subjects who were randomised. Here, Overall number of subjects analysed (N) = subjects with available data for this outcome measure.

End point type

Secondary

End point timeframe

From randomisation (week 0) to the end of the main part (week 26)

End point values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)
Number of subjects analysed	12	13	15	82	85
Units: Score points
arithmetic mean (standard deviation)	-2.2 ( 14.1 )	-9.9 ( 13.4 )	-11.2 ( 11.6 )	-10.8 ( 15.5 )	-10.0 ( 10.5 )

No statistical analyses for this end point

Secondary: Arms 1, 2a, 2b, 3 and 4: Change in participant’s joint pain score using Joint Pain Rating Scale (JPRS)

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End point title

Arms 1, 2a, 2b, 3 and 4: Change in participant’s joint pain score using Joint Pain Rating Scale (JPRS)

End point description

JPRS measures joint pain. Higher scores indicate a higher joint pain intensity. The questionnaire asks "In the past 7 days, how would you rate your worst pain in your joints?". Positive change indicates improvement and negative change indicates worsening. The score ranges from 0-10. FAS included all subjects who were randomised. Here, Overall number of subjects analysed (N) = subjects with available data for this outcome measure.

End point type

Secondary

End point timeframe

From randomisation (week 0) to the end of the main part (week 26)

End point values	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 2b: Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW (Run-in Part)	Arm 4: PPX- Mim8 PPX QM (Run-in Part)
Number of subjects analysed	12	13	15	82	85
Units: Score points
arithmetic mean (standard deviation)	-0.8 ( 2.4 )	-0.5 ( 3.4 )	-1.8 ( 2.8 )	-0.1 ( 2.4 )	-0.3 ( 1.9 )

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

From randomisation (week 0) to end of the trial (Week 52)

Adverse event reporting additional description

The data presented for Arm1 includes both the main period (No PPX) and the extension period (Mim8 PPX QW/QM)

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

27

Reporting group title

Arm1: No PPX - Mim8 PPX QW/QM

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and continued no prophylaxis (on-demand treatment with their standard of care products) treatment in the main part of the study (26 weeks). After the main part, subjects continued in the extension part of the study (26 weeks) to receive either once-weekly (QW) loading dose of 24 milligram (mg) (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 4 mg (body weight 30-<45 kg) or 9 mg (body weight >=45 kg) respectively, or once-monthly (QM) loading dose of 40 mg (body weight 30-<45 kg) or 92 mg (body weight >=45 kg) Mim8 subcutaneously followed by maintenance dose of 20 mg (body weight 30 - <45 kg) or 46 mg (body weight >=45 kg) respectively, based on agreement with the investigator.

Reporting group title

Arm 2a: Mim8 PPX QW

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 4: PPX- Mim8 PPX QM

Reporting group description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered main part of the study, to receive loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 3: PPX - Mim8 PPX QW

Reporting group description

Subjects on coagulation factor prophylaxis prior to enrolment continued with the same product type and dosing frequency in the run-in period for at least 26 weeks and up to 52 weeks. Subjects entered the main part of the study, to receive loading dose of either 24 mg (body weight 30-<45 kg) or 55 mg (body weight >=45 kg) Mim8 QW subcutaneously followed by maintenance dose of 4 mg (body weight 30 -<45 kg) or 9 mg >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QW subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Reporting group title

Arm 2b: Mim8 PPX QM

Reporting group description

Subjects on no prophylaxis treatment did not enter the run-in period and received loading dose of wither 40 mg (body weight 30-<45 kg) or 92 mg (body weight ≥45 kg) Mim8 QM subcutaneously followed by maintenance dose of 20 mg (body weight 30-<45 kg) or 46 mg (body weight >=45 kg) respectively. After the main part, subjects continued to receive the same dosing regimen of Mim8 QM subcutaneously in the extension part of the study (26 weeks). The dose could only be changed in relation to weight band change.

Serious adverse events	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 4: PPX- Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW	Arm 2b: Mim8 PPX QM
Total subjects affected by serious adverse events
subjects affected / exposed	1 / 18 (5.56%)	1 / 22 (4.55%)	9 / 109 (8.26%)	7 / 111 (6.31%)	0 / 21 (0.00%)
number of deaths (all causes)	0	0	0	0	0
number of deaths resulting from adverse events	0	0	0	0	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Testicular teratoma benign
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	0 / 109 (0.00%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Injury, poisoning and procedural complications
Head injury
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Ligament sprain
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	0 / 109 (0.00%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Limb injury
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Skin laceration
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	0 / 109 (0.00%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Nervous system disorders
Epilepsy
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
VIth nerve paralysis
subjects affected / exposed	0 / 18 (0.00%)	1 / 22 (4.55%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	1 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Eye disorders
Optic ischaemic neuropathy
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	0 / 109 (0.00%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Gastrointestinal disorders
Haemorrhoids
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Upper gastrointestinal haemorrhage
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	0 / 109 (0.00%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Hepatobiliary disorders
Cholecystitis chronic
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	0 / 109 (0.00%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
Arthropathy
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Haematoma muscle
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Synovitis
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Infections and infestations
Pneumonia
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Abdominal abscess
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Appendicitis
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Bronchitis
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 2	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Cellulitis
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Psoas abscess
subjects affected / exposed	0 / 18 (0.00%)	1 / 22 (4.55%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Soft tissue infection
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	0 / 109 (0.00%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Product issues
Device loosening
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	0 / 109 (0.00%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Arm1: No PPX - Mim8 PPX QW/QM	Arm 2a: Mim8 PPX QW	Arm 4: PPX- Mim8 PPX QM	Arm 3: PPX - Mim8 PPX QW	Arm 2b: Mim8 PPX QM
Total subjects affected by non serious adverse events
subjects affected / exposed	10 / 18 (55.56%)	9 / 22 (40.91%)	62 / 109 (56.88%)	60 / 111 (54.05%)	6 / 21 (28.57%)
Investigations
Prothrombin fragment 1.2 increased
subjects affected / exposed	2 / 18 (11.11%)	2 / 22 (9.09%)	9 / 109 (8.26%)	14 / 111 (12.61%)	1 / 21 (4.76%)
occurrences all number	2	2	9	15	1
Surgical and medical procedures
Intra-uterine contraceptive device removal
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	0
Cardiac disorders
Sinus bradycardia
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	0
Nervous system disorders
Headache
subjects affected / exposed	1 / 18 (5.56%)	1 / 22 (4.55%)	9 / 109 (8.26%)	12 / 111 (10.81%)	1 / 21 (4.76%)
occurrences all number	1	1	16	15	1
Dizziness
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	1 / 109 (0.92%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences all number	1	0	1	3	0
General disorders and administration site conditions
Injection site reaction
subjects affected / exposed	0 / 18 (0.00%)	2 / 22 (9.09%)	3 / 109 (2.75%)	7 / 111 (6.31%)	1 / 21 (4.76%)
occurrences all number	0	2	3	103	2
Pyrexia
subjects affected / exposed	2 / 18 (11.11%)	1 / 22 (4.55%)	5 / 109 (4.59%)	3 / 111 (2.70%)	2 / 21 (9.52%)
occurrences all number	2	1	6	3	2
Injection site erythema
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	2 / 109 (1.83%)	3 / 111 (2.70%)	0 / 21 (0.00%)
occurrences all number	1	0	2	5	0
Gastrointestinal disorders
Abdominal pain upper
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	1	0	0
Diarrhoea
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	4 / 109 (3.67%)	5 / 111 (4.50%)	1 / 21 (4.76%)
occurrences all number	1	0	4	8	1
Gastrooesophageal reflux disease
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	1	0	0
Dyspepsia
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	0 / 109 (0.00%)	3 / 111 (2.70%)	2 / 21 (9.52%)
occurrences all number	0	0	0	4	2
Gastritis
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	0 / 109 (0.00%)	2 / 111 (1.80%)	1 / 21 (4.76%)
occurrences all number	1	0	0	2	1
Nausea
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	1	0	0
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	3 / 109 (2.75%)	6 / 111 (5.41%)	1 / 21 (4.76%)
occurrences all number	0	0	3	6	1
Skin and subcutaneous tissue disorders
Umbilical erythema
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	0
Seborrhoeic dermatitis
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	0
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	0 / 18 (0.00%)	1 / 22 (4.55%)	10 / 109 (9.17%)	6 / 111 (5.41%)	0 / 21 (0.00%)
occurrences all number	0	1	15	6	0
Back pain
subjects affected / exposed	1 / 18 (5.56%)	1 / 22 (4.55%)	7 / 109 (6.42%)	6 / 111 (5.41%)	0 / 21 (0.00%)
occurrences all number	1	1	7	6	0
Sacroiliitis
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	0
Infections and infestations
Influenza
subjects affected / exposed	0 / 18 (0.00%)	1 / 22 (4.55%)	5 / 109 (4.59%)	6 / 111 (5.41%)	0 / 21 (0.00%)
occurrences all number	0	1	6	8	0
Respiratory tract infection
subjects affected / exposed	0 / 18 (0.00%)	2 / 22 (9.09%)	0 / 109 (0.00%)	2 / 111 (1.80%)	0 / 21 (0.00%)
occurrences all number	0	2	0	2	0
Upper respiratory tract infection
subjects affected / exposed	1 / 18 (5.56%)	2 / 22 (9.09%)	17 / 109 (15.60%)	11 / 111 (9.91%)	3 / 21 (14.29%)
occurrences all number	2	6	20	18	4
Body tinea
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	0	0	0
COVID-19
subjects affected / exposed	0 / 18 (0.00%)	1 / 22 (4.55%)	9 / 109 (8.26%)	7 / 111 (6.31%)	0 / 21 (0.00%)
occurrences all number	0	1	9	7	0
Nasopharyngitis
subjects affected / exposed	0 / 18 (0.00%)	0 / 22 (0.00%)	16 / 109 (14.68%)	11 / 111 (9.91%)	0 / 21 (0.00%)
occurrences all number	0	0	23	16	0
Tooth infection
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	0 / 109 (0.00%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences all number	1	0	0	1	0
Urinary tract infection
subjects affected / exposed	2 / 18 (11.11%)	0 / 22 (0.00%)	0 / 109 (0.00%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	2	0	0	0	0
Metabolism and nutrition disorders
Hyperuricaemia
subjects affected / exposed	2 / 18 (11.11%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	2	0	1	0	0
Hypokalaemia
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	1 / 109 (0.92%)	0 / 111 (0.00%)	0 / 21 (0.00%)
occurrences all number	1	0	1	0	0
Hyperlipidaemia
subjects affected / exposed	1 / 18 (5.56%)	0 / 22 (0.00%)	1 / 109 (0.92%)	1 / 111 (0.90%)	0 / 21 (0.00%)
occurrences all number	1	0	1	1	0

More information

Top of page

Were there any global substantial amendments to the protocol? Yes

08 Oct 2021

Protocol version 5.0: Change of treatment prophylaxis to bleeding prophylaxis in the primary objective and remove subjects in the sentence; Bleeds into target joints must be treated or prescribed treatment to be a target joint bleed; The run-in period is planned to be of up to 52 weeks duration; Participants on no prophylaxis are not allowed to participate in the run-in; In total approximately 244 participants are planned to be enrolled into this study, with at least 30 adolescent (12-17 years) participants; Patients on coagulation factor prophylaxis will preferably continue the same product type; Changed that on-demand treatment will receive Standard of Care product instead of usual product.

22 Nov 2022

Protocol version 9.0: A new study design for participants who are on no prophylaxis/on-demand treatment prior to enrolment and a change of primary endpoint analyses for Mim8 QW and QM in treatment Arms 3 and 4 were also included based on authority feedback; Primary endpoint analyses for Mim8 QW and QM treatment arms will be performed separately in a hierarchical manner. Superiority test will be performed for Arms 3 and 4 without a non-inferiority test; The total number of participants has been updated to 267; Exclusion criterion updated regarding requirement of participation in any interventional clinical study prior to this study, regarding exposure to nonfactor haemostatic products and regarding timing of planned major surgery; In the estimand section the handling of intercurrent events has been elaborated and ‘major surgery’ has been removed as an intercurrent event.

29 Sep 2023

Protocol version 11.0: Reintroduced non-inferiority tests for Arms 3 and 4 as the first step in the statistical testing hierarchy followed by the superiority tests. The power calculation based on non inferiority and superiority are provided; Added an additional secondary endpoint related to Mim8 plasma concentration addressing health authority feedback; Extended the visit window for Visit 13 for countries and sites where participants’ transfer to study NN7769-4532 is not possible either due to study not being approved or sites not being open for transfer at the time of visit; Included country-specific requirements for EU countries, United States and Russia.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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