The paradigm clinical trial programme for nonacog beta pegol enrolled children and adults with severe or moderately severe haemophilia B. A total of 115 previously treated patients with a total of more than 10,625 exposure days for up to 3.3 years of treatment with nonacog beta pegol.
The paradigm 1 PK trial (16 patients treated) - a single-dose escalation trial evaluating safety and PK of nonacog beta pegol compared with marketed recombinant and plasma-derived factor IX products. Nonacog beta pegol showed up to twofold increase in recovery, higher activity levels and a fivefold prolongation of half-life compared to existing treatment.
The paradigm 2 pivotal trial (74 patients treated, including a subset of patients from the paradigm 1 trial) - a 52-week single-blinded randomised trial evaluating safety, efficacy and PK for adults and adolescents in routine prophylaxis and treatment of bleeds. When provided prophylactic at 40 IU/kg weekly, nonacog beta pegol appeared to have a safe and well-tolerated profile and showed a median annualised spontaneous bleeding rate of 0.0. Furthermore, 97% of breakthrough bleeds were treated successfully and 90% of target joints no longer classified as such.
The paradigm 3 surgery trial (13 patients treated, including a subset of people from the paradigm 2 trial) - a dedicated trial confirming safety and efficacy during and after major surgical procedures. In all patients, a single preoperative dose provided effective haemostatic coverage, and no patient required additional doses on the day of surgery. Additionally, three doses proved sufficient in maintaining haemostasis during the first two weeks following the procedure.
The paradigm 4 extension trial (71 patients previously treated in the paradigm 2 or paradigm 3 trials) - a safety extension trial with longer-term exposure demonstrated a well-tolerated profile with no inhibitors or other safety signals identified.
The paradigm 5 paediatric trial (25 patients treated) - a 52-week single-arm trial evaluating once-weekly prophylaxis and treatment of bleeding episodes in previously treated children 1-12 years of age. Nonacog beta pegol appeared to have a safe profile, and all patients maintained mean factor activity levels above 15% one week after dosing of 40 IU/kg and a median ABR of 0.0 and 2.0 for children aged 0-6 and 7-12 years old respectively.
For further information
|Katrine Sperling||+45 3079 firstname.lastname@example.org|
|Ken Inchausti (US)||+1 609 514 email@example.com|
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Company announcement No 39 / 2016