Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study

NCT05935358 | Recruiting Phase 4 DMC FDA Drug

• 1 other identifier: GENA-22
• Study Type: interventional
• Target: 28
• Locations: 11 countries
• Sites: 18

Brief Summary

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

Conditions

Severe Hemophilia A

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (9)

• Intraoperative haemostatic efficacy

  During surgery: From first skin incision to last suture

• Postoperative haemostatic efficacy

  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

  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

  ≤30 minutes before and 15-30 minutes after Nuwiq injection

• Thrombin generation

  ≤30 minutes before and 15-30 minutes after Nuwiq injection

Study Arms (1)

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.

Drug: Nuwiq

Interventions

Nuwiq DRUG

Nuwiq is a purified B-domain-deleted FVIII glycoprotein synthesised by a genetically engineered human embryonic kidney cell line (HEK 293F).

Nuwiq

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Octapharma: lead

Study Sites (18)

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

University Hospital Centre Zagreb

Zagreb, Croatia

RECRUITING

Helsinki University Hospital

Helsinki, Finland

RECRUITING

CHU de Nantes Hôtel-Dieu

Nantes, France

RECRUITING

CHRU de Tours

Tours, France

RECRUITING

Vivantes Klinikum im Friedrichshein (KFH)

Berlin, Germany

RECRUITING

Gerinnungszentrum Rhein-Ruhr

Duisburg, Germany

RECRUITING

Universitätsklinikum Hamburg Eppendorf (UKE)

Hamburg, Germany

RECRUITING

Christian Medical College Vellore

Vellore, Tamil Nadu, India

RECRUITING

St. John's Medical College Hospital

Bengaluru, India

RECRUITING

Azienda Ospedaliera Pugliese Ciaccio

Catanzaro, Italy

RECRUITING

Azienda Ospedaliero Universitaria Careggi - Centro Emofilia

Florence, Italy

RECRUITING

Centro Trombosi e Malattie Emorragiche, ITCCS Humanitas Research Hospital

Milan, Italy

RECRUITING

Centre for Haemopilia, Institute for transfusion medicine of Republic of North Macedonia

Skopje, North Macedonia

RECRUITING

Clinical Center for Serbia Belgrade

Belgrade, Serbia

RECRUITING

Hospital Universitario La Paz

Madrid, Spain

RECRUITING

Hospital Universitario Virgen del Rocio

Seville, Spain

RECRUITING

St. James's University Hospital

Leeds, United Kingdom

NOT YET RECRUITING

Related Publications (1)

• 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.

  PMID: 41151952 DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Shveta Gupta, MD

  Arnold Palmer Hospital for Children

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sigurd Knaub, PhD

CONTACT

+41 554512141; Sigurd.Knaub@octapharma.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 8, 2023
• First Posted: July 7, 2023
• Study Start: November 28, 2023
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: October 24, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2); GB (1); IT (3); FI (1); NO (1); SE (1); CR (1); ES (2); IN (2); US (1); DE (3)