WithdrawnPhase 4

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

Stopped: Due to recruitment difficulties for pediatric patients. The reason for study cancellation is not related to safety concerns or any adverse events.

Spain0Started 2013-12

Plain-language summary

Multicenter, prospective, non-controlled study in a pediatric cohort (\<6 years-old) with severe (type 2 or 3) hereditary Von Willebrand Disease (VWD).

Who can participate

Age range

2 Months – 6 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Subjects diagnosed with severe (type 2 or 3) hereditary VWD (VWF:RCo\<15-20 IU/dL), or VWF:Act\<15-20 IU/dL.
. Subjects under 6 years of age.
. 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

. Subjects diagnosed with acquired VWD.
. Subjects with active bleeding at the time of the first infusion or within 10 days prior to the infusion.
. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

AUC^0-inf of coagulation factor VIII activity (FVIII:C)

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-inf of von Willebrand factor: Ristocetin cofactor activity (VWF:RCo)

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-inf of von Willebrand factor antigen (VWF:Ag)

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-inf of von Willebrand factor: Collagen binding activity (VWF:CB)

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-T of FVIII:C

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-T of VWF:RCo

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-T of VWF:Ag

Timeframe: Prior to the first infusion up to 72 hours postinfusion

Trial details

NCT IDNCT02472665

SponsorGrifols Therapeutics LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-12-10

View on ClinicalTrials.gov More Von Willebrand Disease trials

Who can participate

Age range

2 Months – 6 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Subjects diagnosed with severe (type 2 or 3) hereditary VWD (VWF:RCo\<15-20 IU/dL), or VWF:Act\<15-20 IU/dL.
. Subjects under 6 years of age.
. 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

. Subjects diagnosed with acquired VWD.
. Subjects with active bleeding at the time of the first infusion or within 10 days prior to the infusion.
. 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.

What they're measuring

AUC^0-inf of coagulation factor VIII activity (FVIII:C)

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-inf of von Willebrand factor: Ristocetin cofactor activity (VWF:RCo)

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-inf of von Willebrand factor antigen (VWF:Ag)

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-inf of von Willebrand factor: Collagen binding activity (VWF:CB)

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-T of FVIII:C

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-T of VWF:RCo

Timeframe: Prior to the first infusion up to 72 hours postinfusion

AUC^0-T of VWF:Ag

Timeframe: Prior to the first infusion up to 72 hours postinfusion