A research study looking at long-term treatment with Mim8 in people with haemophilia A(NN7769-4532)

JRCT ID: jRCT2041230047

Registered date:24/06/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Participants with haemophilia A with or without FVIII inhibitors
• Date of first enrollment: 13/02/2023
• Target sample size: 425
• Countries of recruitment: Austria,Japan,Belgium,Japan,Bulgaria,Japan,Canada,Japan,China,Japan,Denmark,Japan,France,Japan,Germany,Japan,India,Japan,Ireland,Japan,Israel,Japan,Italy,Japan,Latvia,Japan,Lithuania,Japan,Malaysia,Japan,Mexico,Japan,Netherlands,Japan,Poland,,Japan,Portugal,Japan,Romania,Japan,Saudi Arabia,Japan,Serbia,Japan,Slovakia,Japan,South Africa,Japan,South Korea,Japan,Spain,Japan,Switzerland,Japan,Taiwan,Japan,Turkey,Japan,United Kingdom,Japan
• Study type: Interventional

Intervention(s)

The study consists of two arms (Arms 1 and 2), where participants from study NN7769-4513 will enter Arm 1 and participants from studies NN7769-4514, NN7769 4516, and NN7769 4728 will enter Arm 2.

Treatment period is consistent of part1 and part2.
During part1(first 26 weeks of the study), participants transferring from study NN7769-4513 will all receive Q2W dosing. Participants transferring from study NN7769-4514, NN7769-4516, and NN7769-4728 will use the same treatment frequency as in their previous study (QW, Q2W, or QM).
After part1, during part2 study participants in both arms will get the choice to switch to another treatment frequency (QW, Q2W, or QM)

Dose amount is based on weight band of participant.
Weight bands and corresponding doses for study
Once-Weekly:
1.6mg(below15kg), 4.0mg(15kg or above, below 45kg), 9.0mg(45kg or above)

Once-Monthly
9.0mg(below 15kg), 20.0mg(15kg or above, below 45kg), 46.0mg(45kg or above)

Outcome(s)

• Primary Outcome: Number of treatment-emergent adverse events (TEAEs)

Key inclusion & exclusion criteria

• Age minimum: >= 1age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Informed consent obtained before any study related activities. Study related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
2.Male or female with diagnosis of congenital haemophilia A based on medical records.
3.Ongoing participation in study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769 4728 at the time of transfer. Participant should qualify either of the following criteria:
a.Participant from study NN7769-4513, who has participated in the extension part of the study for at least 12 weeks prior to enrolment in study NN7769-4532, or,
b.Participant has completed the end of treatment visit for study NN7769-4514, NN7769 4516, or NN7769-4728.
4.Participant and/or participant's parent(s)/participant's legally acceptable representative (LAR) willingness and ability to comply with scheduled visits and study procedures, including the completion of diary.

Exclude criteria

1.Any disorder, except for conditions associated with haemophilia, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
2.Participant who has discontinued or been withdrawn from studies NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728.
3.Previous participation in this study. Participation is defined as signed informed consent.
4.Female who is pregnant, breast-feeding or intends to become pregnant.
5.Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures as defined in Appendix 4 or as required by local regulation or practice).
6.Participation (i.e., signed informed consent) in any interventional, clinical study (except from study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728) of an approved or non-approved investigational medicinal product.
7. Any planned majors surgery, during part1 of the study, For definition of major surgery see Table6-7.
8.Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.

Related Information

• Primary Sponsor: Esaki Risa
• Secondary ID(s): NCT05685238