Clinical Studies

The haemostatic efficacy of Nuwiq^® (simoctocog alfa) for prophylaxis, treatment of bleeds and surgery in people with haemophilia A has been demonstrated in numerous clinical trials.

As part of Octapharma's long-standing commitment to addressing the unmet clinical needs of patients with haemophilia A, Octapharma continues to carry out clinical trials and support investigator-initiated studies to better understand the clinical efficacy and safety of Nuwiq^® in different patient populations.

Completed clinical trials with Nuwiq^®

2008

2010

2012

2014

2016

2018

2020

2022

Phase II trial in 22 adult PTPs¹

Phase III trial on immunogenicity, efficacy and safety in 110 PUPs, and extension study^6,7

Phase III trial in 18 adult PTPs¹

Phase III trial on individualised prophylaxis in 55 adult PTPs¹⁰

Phase II trial on on-demand treatment in 22 PTPs above 12 years old²

Phase III trial on individualised prophylaxis in 66 adult PTPs^8,9

Phase III trial on prophylaxis in 32 adult PTPs³

Phase III trial on prophylaxis in 59 previously treated children, and extension study^4,5

Phase III trial on prophylaxis in 63 PTPs above 12 years old

Non-interventional Phase IV study in 78 adult PTPs¹¹

PTP: previously treated patient; PUP: previously untreated patient.

Ongoing studies with Nuwiq^®

2016

2018

2020

2022

2024

2026

2028

2030

Phase IV Protect-NOW trial on immunogenicity, safety and efficacy in 140 PUPs and MTPs¹⁵

Non-interventional Phase IV trial on safety and efficacy of prophylaxis in 50 patients¹⁴

MOTIVATE study on ITI in 120 patients¹³

NuPOWER¹⁶

NuDIMENSION¹⁷

Octapharma sponsored clinical study

Investigator-initiated study

ITI: immune tolerance induction; MTP: minimally treated patient; PTP: previously treated patient; PUP: previously untreated patient.

Ongoing and planned clinical studies with Nuwiq^®

Gaining insights on the optimal treatment in patients with haemophilia A

Real-world evidence on
the treatment of PUPs/MTPs with
severe haemophilia A ¹⁵

Perioperative management of women/girls with haemophilia A undergoing surgery¹⁷

Perioperative management of patients with haemophilia A on emicizumab prophylaxis¹⁶

MTP: minimally treated patient.

Investigator-initiated study

MOTIVATE study

The objectives of the MOTIVATE study are to:

Capture different approaches to the treatment and management of people with haemophilia A and inhibitors
Document current ITI approaches
Evaluate efficacy and safety of ITI, emicizumab prophylaxis, and ITI with emicizumab prophylaxis

Coordinating Investigators

Robert F. Sidonio Jr.
Emory University, Atlanta, GA, USA

Carmen Escuriola Ettingshausen
HZRM Hämöphilie-Zentrum Rhein Main, Frankfurt-Mörfelden, Germany

EU Clinical Trials register

motivate-study.com

ClinicalTrials.gov

ITI: immune tolerance induction; MTP: minimally treated patient; PTP: previously treated patient; PUP: previously untreated patient.

References

1
Lissitchkov T et al. Ther Adv Haematol 2019; 10:1-15;
2
Tiede A et al. Haemophilia 2016; 22:374-80;
3
Lissitchkov T et al. Haemophilia 2016; 22:225-31;
4
Klukowska A et al. Haemophilia 2016; 22:232-39;
5
Klukowska A et al. Haemophilia 2018; 24:595-603;
6
Liesner RJ et al. Thromb Haemost 2021; 121:1400-8;
7
Mathias M et al. Haemophilia 2023; 29:1005-12;
8
Lissitchkov T et al. Haemophilia 2017; 23:697-704;
9
Pasi K. Clin Med Insights Blood Disord 2021; 14:2634853521991517;
10
ClinicalTrials.gov, NCT02256917;
11
ClinicalTrials.gov, NCT02962765;
12
Escuriola Ettingshausen C and Sidonio RF. Ther Adv Hematol 2021; 12:20406207211032452;
13
ISRCTN registry, ISRCTN57549638;
14
Oldenberg J et al. TH Open 2023; 7:e110–16.
15
ClinicalTrials.gov, NCT05935358;
16
ClinicalTrials.gov, NCT05936580;