Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
ConditionHemophilia A
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexFemale
Age12+
SponsorOctapharma
About this trial
Recombinant factor VIII for the prevention of bleeding in women/girls with haemophilia A undergoing major surgery
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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)
Trial design
Design model
Single group
Treatments tested in this trial
Nuwiq
DrugNuwiq is a purified B-domain-deleted FVIII glycoprotein synthesised by a genetically engineered human embryonic kidney cell line (HEK 293F).
Treatment groups
28 Participants
are divided into 1 treatment groupGroup A: NuwiqExperimental treatment 1 intervention
Trial outcomes
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.
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 procedureSecondary outcomes
1
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.
Time frame
During surgery: From first skin incision to last suture2
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.
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 procedure3
Allogenic blood products
Use of allogeneic blood products (red blood cells, platelets, and other 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 procedure4
FVIII plasma levels
Perioperative plasma levels
Time frame
≤30 minutes before and 15-30 minutes after Nuwiq injection Source ClinicalTrials.gov