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Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis
Patients with severe haemophilia A receiving emicizumab will often need concomitant FVIII to provide haemostatic cover during major surgery. This prospective, open-label, uncontrolled, single-arm, multinational, multicentre study aims to evaluate the overall perioperative haemostatic efficacy of Nuwiq, a recombinant factor VIII, in combination with emicizumab prophylaxis in male patients over 12 with severe haemophilia A undergoing major surgery.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Nuwiq | Experimental | All patients receiving Nuwiq (recombinant FVIII). Nuwiq will be administered intravenously in accordance with the relevant prescribing information. Treatment will be repeated as necessary every 8-24 hours until adequate wound healing, then - if required - for at least another 7 days to maintain FVIII plasma levels of 30-60 IU/dL. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Nuwiq | Drug | Nuwiq is a purified B-domain-deleted FVIII glycoprotein synthesised by a genetically engineered human embryonic kidney cell line (HEK 293F). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overall haemostatic efficacy | Overall haemostatic efficacy of treatment measured as binary "success" or "failure". The overall perioperative haemostatic efficacy of Nuwiq will be adjudicated by an Independent Data Monitoring Committee (IDMC) and determined using a composite assessment algorithm that considers the surgeon's assessment of intraoperative haemostatic efficacy and the investigator's assessment of postoperative haemostatic efficacy to classify the overall haemostatic efficacy as success or failure. | During surgery and until completion of wound healing (e.g. removal of sutures, cessation of drainage, etc.) as defined by the investigator, during the study duration of 30 ± 3 days after the day of surgical procedure |
| Measure | Description | Time Frame |
|---|---|---|
| Intraoperative haemostatic efficacy | Assessment of intraoperative haemostatic efficacy of Nuwiq using a 4-point ordinal scale: Excellent: Intraoperative blood loss was lower than or equal to the average expected blood loss for the type of procedure performed in a patient with normal haemostasis and of the same sex, age, and stature Good: Intraoperative blood loss was higher than the average expected blood loss but lower or equal to the maximal expected blood loss for the type of procedure in a patient with normal haemostasis. Moderate: Intraoperative blood loss was higher than the maximum expected blood loss for the type of procedure performed in a patient with normal haemostasis, but haemostasis was controlled. None: Haemostasis was uncontrolled, necessitating a change in the clotting factor replacement regimen. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Sigurd Knaub, PhD | Contact | +41 554512141 | Sigurd.Knaub@octapharma.com |
| Name | Affiliation | Role |
|---|---|---|
| Shveta Gupta, MD | Arnold Palmer Hospital for Children | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UT Southwestern Medical Center | Recruiting | Dallas | Texas | 75390 | United States | |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41151952 | Derived | Srivastava A, Kanny A, Langer F, Kubicek-Hofmann C, Alvarez Roman MT, Nunez Vazquez R, Boban A, Dejanova-Ilijevska V, Miljic P, Garcia J, Halimeh S, Drillaud N, Valentin JB, Mancuso ME, Castaman G, Santoro RC, Lehtinen AE, Abraham A, Hashimoto M, Knaub S. NuPOWER (Nuwiq for Perioperative management Of patients With haemophilia A on Emicizumab Regular prophylaxis): protocol for an open-label, single-arm, multicentre study. BMJ Open. 2025 Oct 28;15(10):e102657. doi: 10.1136/bmjopen-2025-102657. |
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| During surgery: From first skin incision to last suture |
| Postoperative haemostatic efficacy | Assessment of postoperative haemostatic efficacy of Nuwiq using a 4-point ordinal scale: Excellent: No postoperative bleeding or oozing that was not due to complications of surgery. All postoperative bleeding (due to complications of surgery) was controlled with Nuwiq as anticipated for the type of procedure. Good: No postoperative bleeding or oozing that was not due to complications of surgery. Control of postoperative bleeding due to complications of surgery required increased dosing with Nuwiq or additional injections not originally anticipated for the type of procedure. Moderate: Some postoperative bleeding and oozing that was not due to complications of surgery. Control of postoperative bleeding required increased dosing with Nuwiq or additional injections not originally anticipated for the type of procedure. None: Extensive uncontrolled postoperative bleeding and oozing. | From end of surgery until completion of wound healing (e.g. removal of sutures, cessation of drainage, etc.) as defined by the investigator, during the study duration of 30 ± 3 days after the day of surgical procedure |
| Blood product transfusion levels | The number of allogeneic blood products (red blood cells, platelets, and other blood products) transfused | From day of surgery until completion of wound healing (e.g. removal of sutures, cessation of drainage, etc.) as defined by the investigator, during the study duration of 30 ± 3 days after the day of surgical procedure |
| FVIII plasma levels | Perioperative plasma levels | ≤30 minutes before and 15-30 minutes after Nuwiq injection |
| Thrombin generation | Perioperative thrombin generation | ≤30 minutes before and 15-30 minutes after Nuwiq injection |
| Perioperative haemostatic efficacy per World Federation of Hemophilia (WFH) criteria | Assessed on WFH recommended 4-point scale: Excellent: Intra and post-operative blood loss, with blood component transfusions, similar to non-haemophilic patients. No additional FVIII/bypassing agents Good: Intra/ post-operative blood loss slightly increased over expectation for non-haemophilic patient but clinically insignificant. Blood component transfusions similar to non-haemophilic patient. No additional FVIII/bypassing agents Fair: Intra/ post-operative blood loss increased over expectation for non-haemophilic patients, and additional treatment needed. Extra dose of FVIII/bypassing agents needed or increased blood component of anticipated transfusion requirement Poor: Significant intra- or post-operative blood loss substantially increased over non-haemophilic patient, requires haemophilia-related medical intervention. Unexpected hypotension, transfer to ICU due to bleeding or substantially increased blood component of anticipated transfusion requirement | Perioperative |
| Thrombotic events | Incidence of thrombotic events during the study | From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later |
| FVIII inhibitor formation | Incidence of FVIII inhibitor formation | From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later |
| Adverse events | Incidence of adverse events recorded during the full study period | From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later |
| University Hospital Centre Zagreb |
| Recruiting |
| Zagreb |
| Croatia |
| Helsinki University Hospital | Recruiting | Helsinki | Finland |
| CHU de Nantes Hôtel-Dieu | Recruiting | Nantes | France |
| CHRU de Tours | Recruiting | Tours | France |
| Vivantes Klinikum im Friedrichshein (KFH) | Recruiting | Berlin | Germany |
| Gerinnungszentrum Rhein-Ruhr | Recruiting | Duisburg | Germany |
| Universitätsklinikum Hamburg Eppendorf (UKE) | Recruiting | Hamburg | Germany |
| Christian Medical College Vellore | Recruiting | Vellore | Tamil Nadu | India |
| St. John's Medical College Hospital | Recruiting | Bengaluru | India |
| Azienda Ospedaliera Pugliese Ciaccio | Recruiting | Catanzaro | Italy |
| Azienda Ospedaliero Universitaria Careggi - Centro Emofilia | Recruiting | Florence | Italy |
| Centro Trombosi e Malattie Emorragiche, ITCCS Humanitas Research Hospital | Recruiting | Milan | Italy |
| Centre for Haemopilia, Institute for transfusion medicine of Republic of North Macedonia | Recruiting | Skopje | North Macedonia |
| Clinical Center for Serbia Belgrade | Recruiting | Belgrade | Serbia |
| Hospital Universitario La Paz | Recruiting | Madrid | Spain |
| Hospital Universitario Virgen del Rocio | Recruiting | Seville | Spain |
| St. James's University Hospital | Not yet recruiting | Leeds | United Kingdom |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D005169 | Factor VIII |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
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