Trial intelligence / Profile preview

Mim8 in haemophilia A with or without inhibitors

NCT05053139Phase 3

Key facts

Status: Completed
Start: Dec 2021
Primary completion: Mar 2024
Countries: 34
Sponsor: Novo Nordisk
Modality: Bispecific Antibodies, Monoclonal Antibodies

Drugs: denecimig
Conditions: hemophilia A

Overview

Phase 3, randomised, open-label trial of denecimig in hemophilia A (5 arms), sponsored by Novo Nordisk. Primary endpoint: Annualized Bleed Rate (treated bleeds) at 26 weeks. Started Dec 2021; status: completed.

Design

5 arms

Study design: RandomizedOpen-label

no PPX - no PPX - Mim8 PPXQW/QM
No prophylaxis (on-demand) in main period, then Mim8 prophylaxis in extension
no PPX - Mim8 PPXQW - Mim8 PPXQW
Mim8 once-weekly prophylaxis throughout
no PPX - Mim8 PPXQM - Mim8 PPXQM
Mim8 once-monthly prophylaxis throughout
PPX - Mim8 PPXQW
Run-in with standard prophylaxis, then Mim8 once-weekly
PPX - Mim8 PPXQM
Run-in with standard prophylaxis, then Mim8 once-monthly

Who it enrols

Adults and adolescents (≥12 years, ≥30 kg) with congenital haemophilia A of any severity, with or without inhibitors, who have been prescribed FVIII concentrates or bypassing agents in the last 26 weeks. Patients on no prophylaxis must have had ≥5 bleeds in last 26 weeks; patients on prophylaxis must have had ≥1 bleed in last 26 weeks. Excludes patients with gene therapy, ongoing ITI, other coagulation disorders, significant hepatic/renal impairment, or thromboembolic disease history.

Endpoints

Primary

Annualized Bleed Rate (treated bleeds)26 weeks

Secondary

Injection Site Reactions26 weeks
Occurrence of anti-Mim8 antibodies52 weeks
Annualized Bleed Rate (treated spontaneous bleeds)26 weeks
Annualized Bleed Rate (treated joint bleeds)26 weeks
Annualized Bleed Rate (treated traumatic bleeds)26 weeks
Annualized Bleed Rate (target joint bleeds)26 weeks
Consumption of factor product per bleed treatment26 weeks
Change in physical function domain of Pediatric Quality of Life Inventory26 weeks

Results

Primary endpoints met

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