Clinical trial
Evaluation of the Pharmacokinetic Profile, Clinical Efficacy and Safety of the Von Willebrand Factor Contained in FANHDI® (Double-inactivated Human Anti-hemophilic Factor) in Pediatric Subjects With Severe Von Willebrand Disease
Name
IG1005
Description
Multicenter, prospective, non-controlled study in a pediatric cohort (\<6 years-old) with severe (type 2 or 3) hereditary Von Willebrand Disease (VWD).
Trial arms
Trial start
2013-12-01
Estimated PCD
2024-04-01
Trial end
2024-12-01
Status
Recruiting
Phase
Early phase I
Treatment
plasma-derived FVIII/VWF concentrate
1 single dose of 80 IU/kg VWF:RCo of Fanhdi will be administered
Arms:
plasma-derived FVIII/VWF concentrate
Other names:
Fanhdi
Size
8
Primary endpoint
AUC^0-inf of coagulation factor VIII activity (FVIII:C)
Prior to the first infusion up to 72 hours postinfusion
AUC^0-inf of von Willebrand factor: Ristocetin cofactor activity (VWF:RCo)
Prior to the first infusion up to 72 hours postinfusion
AUC^0-inf of von Willebrand factor antigen (VWF:Ag)
Prior to the first infusion up to 72 hours postinfusion
AUC^0-inf of von Willebrand factor: Collagen binding activity (VWF:CB)
Prior to the first infusion up to 72 hours postinfusion
AUC^0-T of FVIII:C
Prior to the first infusion up to 72 hours postinfusion
AUC^0-T of VWF:RCo
Prior to the first infusion up to 72 hours postinfusion
AUC^0-T of VWF:Ag
Prior to the first infusion up to 72 hours postinfusion
AUC^0-T of VWF:CB
Prior to the first infusion up to 72 hours postinfusion
in vivo recovery of FVIII:C
Prior to the first infusion up to 72 hours postinfusion
in vivo recovery of VWF:RCo
Prior to the first infusion up to 72 hours postinfusion
in vivo recovery of VWF:Ag
Prior to the first infusion up to 72 hours postinfusion
in vivo recovery of VWF:CB
Prior to the first infusion up to 72 hours postinfusion
Half-life of FVIII:C
Prior to the first infusion up to 72 hours postinfusion
Half-life of VWF:RCo
Prior to the first infusion up to 72 hours postinfusion
Half-life of VWF:Ag
Prior to the first infusion up to 72 hours postinfusion
C^max of FVIII:C
Prior to the first infusion up to 72 hours postinfusion
C^max of VWF:RCo
Prior to the first infusion up to 72 hours postinfusion
C^max of VWF:Ag
Prior to the first infusion up to 72 hours postinfusion
C^max of VWF:CB
Prior to the first infusion up to 72 hours postinfusion
T^max of FVIII:C
Prior to the first infusion up to 72 hours postinfusion
T^max of VWF:RCo
Prior to the first infusion up to 72 hours postinfusion
T^max of VWF:Ag
Prior to the first infusion up to 72 hours postinfusion
T^max of VWF:CB
Prior to the first infusion up to 72 hours postinfusion
Mean residence time of FVIII:C
Prior to the first infusion up to 72 hours postinfusion
Mean residence time of VWF:RCo
Prior to the first infusion up to 72 hours postinfusion
Mean residence time of VWF:Ag
Prior to the first infusion up to 72 hours postinfusion
Mean residence time of VWF:CB
Prior to the first infusion up to 72 hours postinfusion
Clearance of FVIII:C
Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion
Clearance of VWF:RCo
Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion
Clearance of VWF:Ag
Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion
Clearance of VWF:CB
Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion
Elimination rate constant of FVIII:C
Prior to the first infusion up to 72 hours postinfusion
Elimination rate constant of VWF:RCo
Prior to the first infusion up to 72 hours postinfusion
Elimination rate constant of VWF:Ag
Prior to the first infusion up to 72 hours postinfusion
Elimination rate constant of VWF:CB
Prior to the first infusion up to 72 hours postinfusion
Volume of distribution of FVIII:C
Prior to the first infusion up to 72 hours postinfusion
Volume of distribution of VWF:RCo
Prior to the first infusion up to 72 hours postinfusion
Volume of distribution of VWF:Ag
Prior to the first infusion up to 72 hours postinfusion
Volume of distribution of VWF:CB
Prior to the first infusion up to 72 hours postinfusion
VWF multimeric pattern
Prior to the first infusion up to 12 hours postinfusion
Eligibility criteria
Inclusion Criteria:
1. Subjects diagnosed with severe (type 2 or 3) hereditary VWD (VWF:RCo\<15-20 IU/dL), or VWF:Act\<15-20 IU/dL.
2. Subjects under 6 years of age.
3. Signed informed consent form (ICF) provided by an authorized representative on behalf of the subject in accordance with local law and institutional policy.
Exclusion Criteria:
1. Subjects diagnosed with acquired VWD.
2. Subjects with active bleeding at the time of the first infusion or within 10 days prior to the infusion.
3. Subjects who have been treated with DDAVP or another FVIII containing VWF concentrate during the 5 days prior to the infusion of the Fanhdi. This treatment-free period may be reduced to 3 days for subjects with type 3 VWD.
4. Subject who are positive for anti-VWF or anti-FVIII antibodies (≥0.5 Bethesda Units) or has been positive in the history of their disease.
5. Subjects with a known allergies/intolerance to any substance contained in Fanhdi.
6. Subjects with a known history of anaphylactic reaction(s) to blood or blood components.
7. Subjects presenting severe platelet activity dysfunction due to the use of drugs (aspirin, other nonsteroidal anti-inflammatory drugs \[NSAIDs\], etc.) or a congenital or acquired platelet function disorder or other concomitant processes that may interfere with coagulation.
8. Subjects have a known previous infection with hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV), or have clinical signs and symptoms consistent with current HAV, HBV, HCV or HIV infection.
9. Subjects presenting anemia (hemoglobin \<11 g/dL).
10. Subjects diagnosed with metabolic diseases that are not clinically controlled, such as diabetes mellitus, which could potentially interfere with the interpretations of the study.
11. Participated in another clinical trial within 30 days prior to the screening visit or has received any investigational product (IP) within 3 months prior to the screening visit.
12. If it is anticipated that the subject will be treated with other products containing FVIII or VWF different from Fanhdi throughout the subject's participation.
13. Subjects who, in the opinion of the investigator, may have compliance problems with the protocol.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE4'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 8, 'type': 'ESTIMATED'}}
Updated at
2024-03-13
1 organization
2 products
1 indication
Organization
Grifols TherapeuticsIndication
von Willebrand disease