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

Hemophilia A Clinical Trial

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)

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05936580
• Other study ID #: GENA-23
• Status: Not yet recruiting
• Phase: Phase 4
• Start date: February 2024
• Est. completion date: December 2025

Study information

• Verified date: January 2024
• Source: Octapharma
• Contact: Sigurd Knaub
• Phone: +41 554512141
• Email: Sigurd.Knaub[@]octapharma.ch
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 28
• Est. completion date: December 2025
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

1. Women/girls with haemophilia A (FVIII:C =1-<40%) according to medical history

2. At least 12 years of age

3. Scheduled to undergo major elective surgery requiring FVIII treatment

4. Freely given written informed consent of the patient, or parent/legal representative where applicable, obtained in accordance with local regulations

Exclusion Criteria:

1. Coagulation disorder other than haemophilia A

2. Present or past FVIII inhibitor (=0.6 Bethesda units [BU]/mL)

3. 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)

4. Known hypersensitivity to Nuwiq's active substance or its excipients (sucrose, sodium chloride, calcium chloride dihydrate, arginine hydrochloride, sodium citrate dihydrate, poloxamer 188)

5. Pregnancy

6. Already had surgery in this study

7. Current participation in another interventional clinical trial

8. Treatment with any investigational medicinal product (IMP) within 30 days prior to screening visit

Related Conditions & MeSH terms

• Hemophilia A

Intervention

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

Locations

Country	Name	City	State
Finland	Helsinki University Hospital,Coagulation Disorder Unit	Helsinki
France	Avenue de la République	Chambray-lès-Tours
France	CHU de Nantes Hôtel-Dieu	Nantes
Germany	Universitätsklinikum Bonn,Institut für Experimentelle Haematologie und Transfusionsmedizin	Bonn
Germany	Universitätsklinikum Hamburg Eppendorf,II. Medizinische Klinik und Poliklinik	Hamburg
Italy	Aziendo Ospedaliera "Puglieze Ciaccio"	Catanzaro
Italy	Policlinico "P. Giaconne"	Palermo
Serbia	Clinical Center for Serbia	Belgrade
Spain	Hospital Universitario la Paz	Madrid
Spain	Hospital Universitario Virgen del Rocio	Sevilla

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Countries where clinical trial is conducted

Finland,  France,  Germany,  Italy,  Serbia,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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 up to 24 hours after last injection of Nuwiq
Secondary	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.	During surgery: From first skin incision to last suture
Secondary	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.	From end of surgery until 24 hours after the last injection of Nuwiq
Secondary	Allogenic blood products	Use of allogeneic blood products (red blood cells, platelets, and other blood products)	From day of surgery until 24 hours after the last injection of Nuwiq
Secondary	FVIII plasma levels	Perioperative plasma levels	Time Frame: <30 minutes before and <30 minutes after Nuwiq injection
Secondary	Perioperative haemostatic efficacy per WFH criteria	Assessed using 4-point scale recommended by WFH: Excellent: Intra- and post-operative blood loss similar to non-haemophilic patient. No extra doses of FVIII/bypassing agents needed Good: Intra- and/or post-operative blood loss slightly increased over expectation for non-haemophilic patient, but judged to be clinically insignificant. No extra doses of FVIII/bypassing agents needed Fair: Intra- and/or post-operative blood loss increased over expectation for the non-haemophilic patient, and additional treatment needed. Extra dose of FVIII/bypassing agents needed, or increased blood component of anticipated transfusion requirement Poor: Significant intra- and/or post-operative blood loss substantially increased over expectation for non-haemophilic patient, requires intervention not explained by medical issue other than haemophilia. Unexpected hypotension, unexpected transfer to ICU due to bleeding, substantially increased blood component of the anticipated transfusion requirement	Perioperative
Secondary	Adverse events	Incidence of adverse events recorded during the full study period	From start of first Nuwiq injection to 30 days following the surgical procedure
Secondary	Thrombotic events	Incidence of thrombotic events during the study	From start of first Nuwiq injection to 30 days following the surgical procedure
Secondary	FVIII inhibitor formation	Incidence of FVIII inhibitor formation	From start of first Nuwiq injection to 30 days following the surgical procedure