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

Official title

Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery - an International, Open-label, Non-controlled Study (NuDIMENSION)

Overview

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

Detailed description

Female patients with mild to moderate haemophilia A will often need FVIII concentrates to provide haemostatic cover during major surgery. This prospective, open-label, non-controlled, single-arm, multinational, multicentre study aims to evaluate the overall perioperative haemostatic efficacy of Nuwiq in women/girls over 12 with haemophilia A undergoing major surgery requiring FVIII treatment

Interventions

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

Primary outcome measures

• 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]

Secondary outcome measures (8)

• Intraoperative haemostatic efficacy [Time frame: During surgery: From first skin incision to last suture]
• Postoperative 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]
• Allogenic blood products [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]
• FVIII plasma levels [Time frame: ≤30 minutes before and 15-30 minutes after Nuwiq injection]
• Perioperative haemostatic efficacy per WFH criteria [Time frame: ≤30 minutes before and 15-30 minutes after Nuwiq injection]
• Adverse events [Time frame: From start of first Nuwiq injection to 30 ±3 days following the surgical procedure]
• Thrombotic events [Time frame: From start of first Nuwiq injection to 30 ±3 days following the surgical procedure]
• FVIII inhibitor formation [Time frame: From start of first Nuwiq injection to 30 ±3 days following the surgical procedure]

Eligibility criteria

Inclusion criteria

• 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

Exclusion criteria

• 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

France · 3 centers

• Avenue de la République — Chambray-lès-Tours
• CHU de Nantes Hôtel-Dieu — Nantes
• Chu de Strasbourg — Stasbourg

Germany · 3 centers

• Universitätsklinikum Bonn,Institut für Experimentelle Haematologie und Transfusionsmedizin — Bonn
• Gerinnungszentrum Rhein-Ruhr — Duisburg
• Universitätsklinikum Hamburg Eppendorf,II. Medizinische Klinik und Poliklinik — Hamburg

Italy · 2 centers

• Aziendo Ospedaliera "Puglieze Ciaccio" — Catanzaro
• Policlinico "P. Giaconne" — Palermo

Spain · 2 centers

• Hospital Universitario la Paz — Madrid
• Hospital Universitario Virgen del Rocio — Seville

United States · 1 center

• UT Health San Antonio — San Antonio

Finland · 1 center

• Helsinki University Hospital,Coagulation Disorder Unit — Helsinki

Serbia · 1 center

• Clinical Center for Serbia — Belgrade

Switzerland · 1 center

• Insel Spital Bern — Bern

United Kingdom · 1 center

• St. James's University Hospital — Leeds

Uruguay · 1 center

• Centro Hospitalario Pereira Rossell — Montevideo

Publications

• 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: 10.117 PMID 39624518

Identifiers

NCT: NCT05936580 · GENA-23

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗