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

Phase 4RecruitingNCT05936580

Octapharma28 enrolled

Overview

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

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Hemophilia A

Interventions

NuwiqDRUG

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

Eligibility

Sex: FEMALEMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. 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) 2. At least 12 years of age 3. Scheduled to undergo major surgery\* requiring FVIII treatment, including elective and emergency procedures and caesarean section in pregnant women with haemophilia A 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, except in participants with a planned caesarean section 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

Locations (16)

UT Health San Antonio

San Antonio, Texas, United States

RECRUITING

Helsinki University Hospital,Coagulation Disorder Unit

Helsinki, Finland

Outcomes

Primary Outcomes

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 procedure

Secondary Outcomes

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 suture

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.

Central Contacts

Cristina Solomon

CONTACT

+41795859042 cristina.solomon@octapharma.com

Data from ClinicalTrials.gov

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