Health Condition 1: D66- Hereditary factor VIII deficiency
Conditions
Interventions
Intervention1: NNC0365-3769 B (Mim8): Pharmaceutical form: DV3407 pen-injector, Solution for injection
Trial product strength: 2.0 mg/mL, 5.0 mg/mL, 11.3 mg/mL, 25.0 mg/mL, 57.5 mg/mL
Dose and dose frequency: Only maintenance doses are administered. Dose frequency is either once-weekly, once every 2 weeks, or once monthly. Dose is set based on weight bands.
Control Intervention1: N
Sponsors
Novo Nordisk India Private Limited
Eligibility
Inclusion criteria
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 or female with diagnosis of congenital haemophilia A based on medical records. 3. Ongoing participation in study 4513, 4514, 4516, or 4728 at the time of transfer. Participant should qualify either of the following criteria: a. Participant from study 4513, who has participated in the extension part of the study for at least 12 weeks prior to enrolment in study 4532, or, b. Participant has completed the end of treatment visit for study 4514,4516, or 4728. 4. Participant and/or participants parent(s)/participants LAR willingness and ability to comply with scheduled visits and study procedures, including the completion of diary.
Exclusion criteria
Exclusion criteria: 1. Any disorder, except for conditions associated with haemophilia, which in the investigators opinion might jeopardise participants safety or compliance with the protocol. 2. Participant who has discontinued or been withdrawn from studies 4513, 4514, 4516, or 4728. 3. Previous participation in this study. Participation is defined as signed informed consent. 4. Female who is pregnant, breast-feeding or intends to become pregnant. 5. Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures as defined in Appendix 4 or as required by local regulation or practice). 6. Participation (i.e., signed informed consent) in any other interventional clinical study (except from study 4513, 4514, 4516, or 4728) of an approved or non-approved investigational medicinal product. 7. Any planned major surgery, during part 1 of the study. 8. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of treatment-emergent adverse eventsTimepoint: From Visit 1 (week 0) until end of study (up to 283 weeks) | — |
Secondary
| Measure | Time frame |
|---|---|
| Number of injection site reactionsTimepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Occurrence of anti-Mim8 antibodiesTimepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Number of treated bleeding episodesTimepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Number of treated spontaneous bleeding episodesTimepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Number of treated traumatic bleeding episodesTimepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Number of treated joint bleeding episodesTimepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Number of treated target joint bleeding episodes (Arm 2)Timepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Mim8 plasma concentrationTimepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Device handling experience using HDAT (applicable for participants in Arm 2 only)Timepoint: At Visit 8 (week 26) & Visit 10 (week 52);Change in participantsââ?¬â?¢ treatment burden using the Hemo-TEM (Applicable for participants in Arm 2 only excluding Chinese participants)Timepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Change in participantââ?¬â?¢s joint pain score using JPRS (Applicable for adult & adolescent participants in Arm 2 only excluding Chinese participants)Timepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks);Change in physical function domain of PedsQL Generic Core Scales (Applicable for participants in Arm 2 only excluding Chinese participants)Timepoint: From Visit 1 (week 0) until end of treatment (up to 262 weeks) | — |
Countries
Austria, Belgium, Bulgaria, Canada, China, Denmark, France, Germany, India, Ireland, Israel, Italy, Japan, Latvia, Lithuania, Malaysia, Mexico, Netherlands, Poland, Portugal, Republic of Korea, Romania, Saudi Arabia, Serbia, Slovakia, South Africa, Spain, Switzerland, Taiwan, Turkey, United Kingdom, United States of America
Contacts
Public ContactDr Maya Sharma
Novo Nordisk India Pvt. Ltd.
Outcome results
None listed