A research study investigating Mim8 in children with haemophilia A with or without inhibitors

Phase 1

• Conditions: Haemophilia A Haemophilia A with inhibitors; MedDRA version: 20.0Level: LLTClassification code 10018938Term: Haemophilia A (Factor VIII)System Organ Class: 100000004850; MedDRA version: 20.0Level: LLTClassification code 10053751Term: Hemophilia A with anti factor VIIISystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2020-003467-26-ES
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-01-20
• Last Update Posted: 19 April 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 70

Inclusion 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 above or equal to 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 below 1%).
5. For previously untreated participants:
a. Diagnosis of severe haemophilia A (endogenous FVIII activity below 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 70
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Known or suspected hypersensitivity to study product or related products.
2. Previous participation in this study. Participation is defined as signed informed consent.
3. 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.
4. Known congenital or acquired coagulation disorders other than haemophilia A.
5. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of
bleeding or thrombosis, as evaluated by the investigator.
6. 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.
7. Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate
understanding and cooperation.
8. Lack of adequate parental/caregiver support to enter accurately and timely information
regarding treatment and bleeding episodes into an (electronic) diary.
9. 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.
10. Major surgery planned to take place after screening.
11. Immune tolerance induction planned to take place after treatment initiation.
12. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine
aminotransferase (ALT) above 3 times the upper limit of normal combined with total bilirubin above 1.5 times the upper limit of normal measured at screening.
13. Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening.
14. Pregnancy (female participants).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified