Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery

NCT05936580 | Recruiting Phase 4 DMC FDA Drug

• 1 other identifier: GENA-23
• Study Type: interventional
• Target: 28
• Locations: 10 countries
• Sites: 15

Brief Summary

Recombinant factor VIII for the prevention of bleeding in women/girls with haemophilia A undergoing major surgery

Conditions

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 (8)

• Intraoperative haemostatic efficacy

  During surgery: From first skin incision to last suture

• Postoperative haemostatic efficacy

  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

• Allogenic blood products

  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

• FVIII plasma levels

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

• Perioperative haemostatic efficacy per WFH criteria

  ≤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: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Women/girls with haemophilia A (FVIII:C ≥1-\<40%) according to medical history. Additionally, women/girls with documented FVIII activity levels between ≥ 40% and 50% may be included if there is a documented history of clinically significant bleeding episodes consistent with haemophilia A; and/or either documented prior treatment with FVIII concentrates or a clinical indication that FVIII treatment would have been appropriate (e.g., use of FVIII from a family member, or treatment with alternative haemostatic agents due to access limitations)
• At least 12 years of age
• Scheduled to undergo major surgery\* requiring FVIII treatment, including elective and emergency procedures and caesarean section in pregnant women with haemophilia A
• 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)
• 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)
• Pregnancy, except in participants with a planned caesarean section
• 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 (15)

UT Health San Antonio

San Antonio, Texas, 78229, United States

RECRUITING

Helsinki University Hospital,Coagulation Disorder Unit

Helsinki, Finland

RECRUITING

Avenue de la République

Chambray-lès-Tours, France

RECRUITING

CHU de Nantes Hôtel-Dieu

Nantes, France

RECRUITING

Universitätsklinikum Bonn,Institut für Experimentelle Haematologie und Transfusionsmedizin

Bonn, Germany

RECRUITING

Gerinnungszentrum Rhein-Ruhr

Duisburg, Germany

RECRUITING

Universitätsklinikum Hamburg Eppendorf,II. Medizinische Klinik und Poliklinik

Hamburg, Germany

RECRUITING

Aziendo Ospedaliera "Puglieze Ciaccio"

Catanzaro, Italy

RECRUITING

Policlinico "P. Giaconne"

Palermo, Italy

RECRUITING

Clinical Center for Serbia

Belgrade, Serbia

RECRUITING

Hospital Universitario la Paz

Madrid, Spain

RECRUITING

Hospital Universitario Virgen del Rocio

Seville, Spain

RECRUITING

Insel Spital Bern

Bern, Switzerland

NOT YET RECRUITING

St. James's University Hospital

Leeds, United Kingdom

RECRUITING

Centro Hospitalario Pereira Rossell

Montevideo, Uruguay

NOT YET RECRUITING

Related Publications (1)

• Marquardt N, Langer F, Holstein K, Alvarez Roman MT, Nunez Vazquez R, Miljic P, Drillaud N, Ardillon L, Lehtinen AE, Santoro RC, Napolitano M, Siragusa S, Gidley G, Jansen M, Knaub S, Oldenburg J. Design of an international, phase IV, open-label study of simoctocog alfa in women/girls with hemophilia A undergoing surgery (NuDIMENSION). Ther Adv Hematol. 2024 Dec 1;15:20406207241300040. doi: 10.1177/20406207241300040. eCollection 2024.

  PMID: 39624518 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

• Johannes Oldenburg

  Experimental Haematology and Transfusion Medicine, University Clinic Bonn

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Cristina Solomon

CONTACT

+41795859042; cristina.solomon@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 10, 2023
• Study Start: April 1, 2026
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2027
• Last Updated: January 13, 2026

Record last verified: 2026-01

Locations

CH (1); US (1); ES (2); SE (1); FR (2); FI (1); DE (3); UR (1); GB (1); IT (2)