Severe Hemophilia APhase 4NuPOWERNCT05935358
Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study
A Phase 4 study testing Nuwiq in people with Severe Hemophilia A, run by Octapharma at 18 sites in 11 countries.
Trial summary
Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Severe Hemophilia A
- What is being tested?
- Nuwiq
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 18 sitesin 11 countries
- Study dates
- 28 Nov 2023to Sep 2026
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
18 sites in 11 countries. Contact the study team or a site near you to ask about taking part.
18 sites in 11 countries
Germany3 sites, 3 recruiting
- Recruiting
Vivantes Klinikum im Friedrichshein (KFH)
Berlin
- Recruiting
Gerinnungszentrum Rhein-Ruhr
Duisburg
- Recruiting
Universitätsklinikum Hamburg Eppendorf (UKE)
Hamburg
Italy3 sites, 3 recruiting
- Recruiting
Azienda Ospedaliera Pugliese Ciaccio
Catanzaro
- Recruiting
Azienda Ospedaliero Universitaria Careggi - Centro Emofilia
Florence
- Recruiting
Centro Trombosi e Malattie Emorragiche, ITCCS Humanitas Research Hospital
Milan
France2 sites, 2 recruiting
- Recruiting
CHU de Nantes Hôtel-Dieu
Nantes
- Recruiting
CHRU de Tours
Tours
India2 sites, 2 recruiting
- Recruiting
St. John's Medical College Hospital
Bengaluru
- Recruiting
Christian Medical College Vellore
Vellore, Tamil Nadu
Spain2 sites, 2 recruiting
- Recruiting
Hospital Universitario La Paz
Madrid
- Recruiting
Hospital Universitario Virgen del Rocio
Seville
United States1 site, 1 recruiting
- Recruiting
UT Southwestern Medical Center
Dallas, Texas, 75390
Croatia1 site, 1 recruiting
- Recruiting
University Hospital Centre Zagreb
Zagreb
North Macedonia1 site, 1 recruiting
- Recruiting
Centre for Haemopilia, Institute for transfusion medicine of Republic of North Macedonia
Skopje
Serbia1 site, 1 recruiting
- Recruiting
Clinical Center for Serbia Belgrade
Belgrade
United Kingdom1 site, 1 recruiting
- Not yet recruiting
St. James's University Hospital
Leeds
About this study
Official title: Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study (NuPOWER)
Recombinant factor VIII for the prevention of bleeding in patients with severe haemophilia A undergoing major surgery while receiving emicizumab prophylaxis
Read the 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.
- Conditions
- Severe Hemophilia A
- Being tested
- Nuwiq
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NuwiqDrug
Nuwiq is a purified B-domain-deleted FVIII glycoprotein synthesised by a genetically engineered human embryonic kidney cell line (HEK 293F).
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Nuwiq |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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 (9)
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.
Measured: 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.
Measured: 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
Measured: 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
Measured: ≤30 minutes before and 15-30 minutes after Nuwiq injection
Thrombin generation
Perioperative thrombin generation
Measured: ≤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
Measured: Perioperative
Thrombotic events
Incidence of thrombotic events during the study
Measured: 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
Measured: 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
Measured: From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2023
- Study start28 Nov 2023
- Primary completionAug 2026 (estimated)
- Study completionSep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Severe Hemophilia AStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Shveta Gupta, MD, Principal investigator, Arnold Palmer Hospital for Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05935358
- Sponsor's protocol ID
- GENA-22
- Acronym
- NuPOWER
- Status last verified
- May 2026
- First submitted
- 8 Jun 2023
- Last updated
- 12 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 41151952
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study (NuPOWER)
- ClinicalTrials.gov ID
- NCT05935358
- Acronym / sponsor ID
- NuPOWER / GENA-22
- Conditions
- Severe Hemophilia A
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 28 Nov 2023 (actual)
- Primary completion
- Aug 2026 (estimated)
- Study completion
- Sep 2026 (estimated)
- First posted
- 7 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nuwiq | Experimental | Drug: NuwiqAll 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. |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NuwiqNuwiq is a purified B-domain-deleted FVIII glycoprotein synthesised by a genetically engineered human embryonic kidney cell line (HEK 293F). | Drug | Nuwiq |
Primary outcome
Overall haemostatic efficacy
Time frame: 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
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.
9 secondary outcomes
Intraoperative haemostatic efficacy
Time frame: During surgery: From first skin incision to last suture
Postoperative haemostatic efficacy
Time frame: 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
Time frame: 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
Time frame: ≤30 minutes before and 15-30 minutes after Nuwiq injection
Thrombin generation
Time frame: ≤30 minutes before and 15-30 minutes after Nuwiq injection
Perioperative haemostatic efficacy per World Federation of Hemophilia (WFH) criteria
Time frame: Perioperative
Thrombotic events
Time frame: 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
Time frame: From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later
Adverse events
Time frame: From start of first Nuwiq injection to 30 ± 3 days following the surgical procedure or until day of discharge, whichever is later
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2026.
- Derived from this study: PubMed 41151952Srivastava 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Shveta Gupta, MD principal investigator
Arnold Palmer Hospital for Children
18 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Nuwiq in people with Severe Hemophilia A, run by Octapharma at 18 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 12 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Germany, Italy, France, India, Spain and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sigurd Knaub, PhD (+41 554512141, Sigurd.Knaub@octapharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall haemostatic efficacy (time frame: 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05935358, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 May 2026.