Safety, efficacy and exposure of subcutaneously administered NNC0365-3769 (Mim8) prophylaxis in children with haemophilia A with or without FVIII inhibitors (NN7769-4516)

JRCT ID: jRCT2031220670

Registered date:02/03/2023

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: children with haemophilia A with or without FVIII inhibitors
• Date of first enrollment: 04/04/2022
• Target sample size: 70
• Countries of recruitment: Canada,Japan,China,Japan,Germany,Japan,India,Japan,Israel,Japan,Italy,Japan,Korea,Japan,Lithuania,Japan,Netherlands,Japan,Poland,Japan,Portugal,Japan,Russian,Japan,South Africa,Japan,Spain,Japan,Switzerland,Japan,Taiwan,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Treatment period is consistent of part1 and part2.
During part1, study product is administered weekly. The treatment period is 26 weeks. A loading dose will be administered once followed by once-weekly maintenance doses in part1.
After part1, during part2 study product is administered weekly or monthly. The treatment period is 26 weeks. In part2 maintenance doses will be administered either once-monthly or once-weekly.

Dose amount is based on weight band of participant, whether it is a loading or maintenance dose, and the frequency of dosing.
Weight bands and corresponding doses for study
Once-Weekly:
Loading dose:9.0mg(below 15kg), 24.0mg(15kg or above, below 45kg), 55.0mg(45kg or above)
Maintenance dose:1.6mg(below15kg), 4.0mg(15kg or above, below 45kg), 9.0mg(45kg or above)

Once-Monthly
Maintenance dose: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 from treatment initiation to followup visit (week 0 to week 72)

Key inclusion & exclusion criteria

• Age minimum: >= 1age old
• Age maximum: <= 11age old
• 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 and female participants with the diagnosis of congenital haemophilia A of any severity
based on medical records.
3. Aged 1-11 years (both inclusive) at the time of signing informed consent.
4. For previously treated participants:
a. Participant has been prescribed treatment with FVIII concentrate or bypassing agent in the
last 26 weeks prior to screening.
b. Participants with endogenous FVIII activity >=1%, based on medical records, must have at
least 1 treated bleed during the previous 26 weeks before screening for which factor VIII
concentrate or bypassing agent has been prescribed (No requirements for participants with
FVIII activity <1%).
5. For previously untreated participants:
a. Diagnosis of severe haemophilia A (endogenous FVIII activity < 1%) based on medical
records.
6. Child and parent/caregiver willingness and ability to comply with scheduled visits and study
procedures, including the completion of diary and patient-reported outcomes questionnaires

Exclude criteria

1. Known or suspected hypersensitivity to trial product or related products.a
2. Previous participation in this study. Participation is defined as signed informed consent.
3. Participation (i.e., signed informed consent) in any interventional clinical study with receipt of
last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is
shorter) before planned randomisation.
4. Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5
half-lives of the medicinal product, whichever is shorter) before planned randomisation, for
participants not included in the run-in.
5. Known congenital or acquired coagulation disorders other than haemophilia A.
6. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis, as evaluated by the investigator.a
7. Any disorder, except for conditions associated with haemophilia A, that in the opinion of investigator might jeopardise the safety of participants or compliance with the protocol.a
8. Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate
understanding and cooperation.a
9. Lack of adequate parental/caregiver support to enter accurately and timely information
regarding treatment and bleeding episodes into an (electronic) diary.a
10. Previous or current treatment for thromboembolic disease (with the exception of previous
catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease.
11. Major surgery planned to take place after screening.a For definition of major surgery, see Table 6-7.
12. Immune tolerance induction planned to take place after treatment initiation.a
13. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine
aminotransferase (ALT) >3 times the upper limit of normal combined with total bilirubin >1.5
times the upper limit of normal measured at screening.
14. Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening.
15. Pregnancy (female participants).

Related Information

• Primary Sponsor: Esaki Risa
• Secondary ID(s): NCT05306418