A research study looking at Mim8 in childrenwith haemophilia A with or without inhibitors

Phase 3

Not yet recruiting

• Conditions: Health Condition 1: D66- Hereditary factor VIII deficiency

• Registration Number: CTRI/2023/01/048708
• Lead Sponsor: ovo Nordisk AS

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 2024-04-29

Recruitment & Eligibility

• Status: Not Yet Recruiting
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

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 Factor Eight concentrate or bypassing agent in the last 26 weeks prior to screening.

b. Participants with endogenous factor eight activity greater than or equal to 1 percentage, based on medical records, must have at least 1 treated bleed during the previous 26 weeks before screening for which factor eight concentrate or bypassing agent has been prescribed (No requirements for participants with factor right activity below 1 percentage).

For previously untreated participants -

a. Diagnosis of severe haemophilia A (endogenous Factor eight activity below 1 percentage) based on medical records.

Child and parent or caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires. (For China mainland, assessed at the investigator discretion unless otherwise stated.)

Exclusion Criteria

Known or suspected hypersensitivity to trial product or related products. (For China mainland, assessed at the investigator discretion unless otherwise stated.)

Previous participation in this study. Participation is defined as signed informed consent.

Participation (that is, 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.

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.

Known congenital or acquired coagulation disorders other than haemophilia A.

Other conditions (eg, autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis, as evaluated by the investigator. (For China mainland, assessed at the investigator discretion unless otherwise stated.)

Any disorder, except for conditions associated with haemophilia A, that in the investigator opinion might jeopardise the participant safety or compliance with the protocol. (For China mainland, assessed at the investigator discretion unless otherwise stated.)

Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate understanding and cooperation. (For China mainland; assessed at the investigator discretion unless otherwise stated.)

Lack of adequate parental or caregiver support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary. (For China mainland, assessed at the investigator discretion unless otherwise stated.)

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. (For China mainland, assessed at the investigator discretion unless otherwise stated.)

Immune tolerance induction planned to take place after treatment initiation. (For China mainland, assessed at the investigator discretion unless otherwise stated.)

Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal measured at screening.

Hepatic dysfunction defined as aspartate aminotransferase (AST) and or alanine aminotransferase (ALT) greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal measured at screening.

Serum creatinine above 1.5 multiplied by the upper limit of normal (ULN), measured at screening.

Pregnancy (female participants). (Will be assessed at investigator discretion, according to suspicion of pregnancy.)

Study & Design

• Study Type: Interventional
• Study Design: Not specified