Back to resultsNuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
Primary Purpose
Hemophilia A
Status
Not yet recruiting
Phase
Phase 4
Locations
Spain
Study Type
Interventional
Intervention
Nuwiq
Sponsored by
About this trial
This is an interventional treatment trial for Hemophilia A
Eligibility Criteria
Inclusion Criteria: Women/girls with haemophilia A (FVIII:C ≥1-<40%) according to medical history At least 12 years of age Scheduled to undergo major elective surgery requiring FVIII treatment 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 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
Sites / Locations
- Hospital Universitario Virgen del Rocio
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Nuwiq
Arm Description
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.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
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.
Allogenic blood products
Use of allogeneic blood products (red blood cells, platelets, and other blood products)
FVIII plasma levels
Perioperative plasma levels
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
Adverse events
Incidence of adverse events recorded during the full study period
Thrombotic events
Incidence of thrombotic events during the study
FVIII inhibitor formation
Incidence of FVIII inhibitor formation
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05936580
Brief Title
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)
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
October 2023 (Anticipated)
Primary Completion Date
December 2025 (Anticipated)
Study Completion Date
December 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Octapharma
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
28 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Nuwiq
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Nuwiq
Intervention Description
Nuwiq is a purified B-domain-deleted FVIII glycoprotein synthesised by a genetically engineered human embryonic kidney cell line (HEK 293F).
Primary Outcome Measure Information:
Title
Overall haemostatic efficacy
Description
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 up to 24 hours after last injection of Nuwiq
Secondary Outcome Measure Information:
Title
Intraoperative haemostatic efficacy
Description
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
Title
Postoperative haemostatic efficacy
Description
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
From end of surgery until 24 hours after the last injection of Nuwiq
Title
Allogenic blood products
Description
Use of allogeneic blood products (red blood cells, platelets, and other blood products)
Time Frame
From day of surgery until 24 hours after the last injection of Nuwiq
Title
FVIII plasma levels
Description
Perioperative plasma levels
Time Frame
Time Frame: <30 minutes before and <30 minutes after Nuwiq injection
Title
Perioperative haemostatic efficacy per WFH criteria
Description
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
Time Frame
Perioperative
Title
Adverse events
Description
Incidence of adverse events recorded during the full study period
Time Frame
From start of first Nuwiq injection to 30 days following the surgical procedure
Title
Thrombotic events
Description
Incidence of thrombotic events during the study
Time Frame
From start of first Nuwiq injection to 30 days following the surgical procedure
Title
FVIII inhibitor formation
Description
Incidence of FVIII inhibitor formation
Time Frame
From start of first Nuwiq injection to 30 days following the surgical procedure
10. Eligibility
Sex
Female
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Women/girls with haemophilia A (FVIII:C ≥1-<40%) according to medical history
At least 12 years of age
Scheduled to undergo major elective surgery requiring FVIII treatment
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
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Sigurd Knaub
Phone
+41 554512141
Email
Sigurd.Knaub@octapharma.ch
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Johannes Oldenburg
Organizational Affiliation
Experimental Haematology and Transfusion Medicine, University Clinic Bonn
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hospital Universitario Virgen del Rocio
City
Sevilla
Country
Spain
12. IPD Sharing Statement
Learn more about this trial
Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
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