Safety, efficacy and exposure of subcutaneously administered NNC0365-3769 (Mim8) prophylaxis in children with haemophilia A with or without FVIII inhibitors
- Conditions
- blood clothing disorderHaemophilia A10064477
- Registration Number
- NL-OMON51874
- Lead Sponsor
- ovo Nordisk
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 4
-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.
- Male and female participants with the diagnosis of congenital haemophilia A
of any severity based on medical records.
- Aged 1*11 years (both inclusive) at the time of signing informed consent.
- 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. Participant 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%).
- For previously untreated participants :
a. Diagnosis of severe haemophilia A (endogenous FVIII activity < 1%) based on
medical
records.
- Child and parent(s)/caregiver(s) willingness and ability to comply with
scheduled visits and
study procedures, including the completion of diary and patient-reported
outcomes
questionnaires.
- Known or suspected hypersensitivity to study product or related products.
- Previous participation in this study. Participation is defined as signed
informed consent.
- Exposure to non-factor haemostatic products for bleeding prophylaxis within 6
months (or 5 half-lives of the medicinal product, whichever is shorter) prior
to planned first dose, for participants not included in the run-in.
- Known congenital or acquired coagulation disorders other than haemophilia A.
- Other conditions (e.g. autoimmune disease) or laboratory abnormality that may
increase risk of bleeding or thrombosis, as evaluated by the investigator.
- Any disorder, except for conditions associated with haemophilia A, that in
the investigator*s opinion might jeopardise the participant*s safety or
compliance with the protocol.
- Mental incapacity, unwillingness to cooperate or a language barrier
precluding adequate understanding and cooperation.
- Lack of adequate parental/caregiver support to enter accurately and timely
information regarding treatment and bleeding episodes into an (electronic)
diary.
- 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.
- Major surgery planned to take place after screening.
- Immune tolerance induction planned to take place after treatment initiation.
- 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.
- Serum creatinine above 1.5 x upper limit of normal (ULN), measured at
screening.
- Pregnancy (female participants).
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method