Efficacy and Safety of Fanhdi®, a High-purity Von Willebrand Containing FVIII Concentrate, in Pediatric Patients With Von Willebrand Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 25 March 2024
• Main ID: NCT02472665
• Date of registration: 10/06/2015
• Prospective Registration: No
• Primary sponsor: Grifols Therapeutics LLC
• Public title: Efficacy and Safety of Fanhdi®, a High-purity Von Willebrand Containing FVIII Concentrate, in Pediatric Patients With Von Willebrand Disease
• Scientific title: 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
• Date of first enrolment: December 2013
• Target sample size: 8
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02472665
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

Spain

Contacts

• Name: Núria Ribó

Key inclusion & exclusion 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.



Age minimum: 2 Months
Age maximum: 6 Years
Gender: All

Health Condition(s) or Problem(s) studied

Von Willebrand Disease

Intervention(s)

Drug: plasma-derived FVIII/VWF concentrate

Primary Outcome(s)

AUC^0-inf of von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Clearance of FVIII:C [Time Frame: Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion]

AUC^0-T of FVIII:C [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Half-life of FVIII:C [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Mean residence time of VWF:RCo [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Volume of distribution of VWF:Ag [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

AUC^0-inf of von Willebrand factor antigen (VWF:Ag) [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

C^max of VWF:Ag [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

C^max of VWF:CB [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

C^max of VWF:RCo [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Clearance of VWF:CB [Time Frame: Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion]

Half-life of VWF:Ag [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

T^max of FVIII:C [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

AUC^0-inf of von Willebrand factor: Collagen binding activity (VWF:CB) [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

AUC^0-T of VWF:Ag [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Half-life of VWF:RCo [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

in vivo recovery of FVIII:C [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

in vivo recovery of VWF:Ag [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

in vivo recovery of VWF:CB [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

T^max of VWF:CB [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Volume of distribution of VWF:RCo [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

VWF multimeric pattern [Time Frame: Prior to the first infusion up to 12 hours postinfusion]

AUC^0-T of VWF:RCo [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

C^max of FVIII:C [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Clearance of VWF:Ag [Time Frame: Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion]

Elimination rate constant of VWF:CB [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Mean residence time of VWF:Ag [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Mean residence time of VWF:CB [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

AUC^0-T of VWF:CB [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Clearance of VWF:RCo [Time Frame: Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion]

Elimination rate constant of VWF:RCo [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

in vivo recovery of VWF:RCo [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Mean residence time of FVIII:C [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

T^max of VWF:Ag [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Volume of distribution of VWF:CB [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

AUC^0-inf of coagulation factor VIII activity (FVIII:C) [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Elimination rate constant of FVIII:C [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Elimination rate constant of VWF:Ag [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

T^max of VWF:RCo [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Volume of distribution of FVIII:C [Time Frame: Prior to the first infusion up to 72 hours postinfusion]

Secondary ID(s)

IG1005

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Instituto Grifols, S.A.