Clinical trial NCT05935358: Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study
Official title: Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study (NuPOWER)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Severe Hemophilia A
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2023-07-07
Enrollment: 28
Sex: MALE
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Severe haemophilia A (FVIII activity \[FVIII:C\] \<1%) according to medical history
* Male patients at least 12 years of age
* Previous treatment with any FVIII product(s) for at least 150 exposure days
* On regular prophylaxis with emicizumab for at least 1 month prior to a scheduled major elective surgery requiring FVIII treatment
* Freely given written informed consent of the patient, or parent/legal representative where applicable, obtained in accordance with local regulations

Exclusion Criteria:

* Coagulation disorder other than haemophilia A
* Present or past FVIII inhibitor (≥0.6 Bethesda units \[BU\]/mL) according to medical history
* Severe liver or kidney disease (alanine aminotransferase \[ALT\] and/or aspartate aminotransferase \[AST\] levels \>5 times the upper limit of normal; or creatinine \>120 μmol/L)
* Known hypersensitivity to Nuwiq's active substance or its excipients (sucrose, sodium chloride, calcium chloride dihydrate, arginine hydrochloride, sodium citrate dihydrate, poloxamer 188)
* Already had surgery in this study
* Current participation in another interventional clinical trial
* Treatment with any investigational medicinal product (IMP) within 30 days prior to screening visit

Primary outcomes:
measure: Overall haemostatic efficacy
timeFrame: 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
Secondary outcomes:
measure: Intraoperative haemostatic efficacy
timeFrame: During surgery: From first skin incision to last suture
measure: Postoperative haemostatic efficacy
timeFrame: 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
measure: Blood product transfusion levels
timeFrame: 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
measure: FVIII plasma levels
timeFrame: ≤30 minutes before and 15-30 minutes after Nuwiq injection
measure: Thrombin generation
timeFrame: ≤30 minutes before and 15-30 minutes after Nuwiq injection
measure: Perioperative haemostatic efficacy per World Federation of Hemophilia (WFH) criteria
timeFrame: Perioperative
measure: Thrombotic events
timeFrame: From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later
measure: FVIII inhibitor formation
timeFrame: From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later
measure: Adverse events
timeFrame: From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later
Other outcomes:
Locations:
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Zagreb
country: Croatia
facility: University Hospital Centre Zagreb
city: Helsinki
country: Finland
facility: Helsinki University Hospital
city: Nantes
country: France
facility: CHU de Nantes Hôtel-Dieu
city: Tours
country: France
facility: CHRU de Tours
city: Berlin
country: Germany
facility: Vivantes Klinikum im Friedrichshein (KFH)
city: Duisburg
country: Germany
facility: Gerinnungszentrum Rhein-Ruhr
city: Hamburg
country: Germany
facility: Universitätsklinikum Hamburg Eppendorf (UKE)
city: Vellore
country: India
facility: Christian Medical College Vellore
state: Tamil Nadu
city: Bengaluru
country: India
facility: St. John's Medical College Hospital
city: Catanzaro
country: Italy
facility: Azienda Ospedaliera Pugliese Ciaccio
city: Florence
country: Italy
facility: Azienda Ospedaliero Universitaria Careggi - Centro Emofilia
city: Milan
country: Italy
facility: Centro Trombosi e Malattie Emorragiche, ITCCS Humanitas Research Hospital
city: Skopje
country: North Macedonia
facility: Centre for Haemopilia, Institute for transfusion medicine of Republic of North Macedonia
city: Belgrade
country: Serbia
facility: Clinical Center for Serbia Belgrade
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Leeds
country: United Kingdom
facility: St. James's University Hospital
Study officials:
affiliation: Arnold Palmer Hospital for Children
name: Shveta Gupta, MD
role: PRINCIPAL_INVESTIGATOR
References: