Safety, efficacy and exposure of subcutaneously administered NNC0365-3769 (Mim8) prophylaxis in children with haemophilia A with or without FVIII inhibitors (NN7769-4516)
- Conditions
- children with haemophilia A with or without FVIII inhibitors
- Registration Number
- JPRN-jRCT2031220670
- Lead Sponsor
- Esaki Risa
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 70
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
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).
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method umber of treatment emergent adverse events from treatment initiation to followup visit (week 0 to week 72)
- Secondary Outcome Measures
Name Time Method