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WithdrawnNCT02472665Updated May 22, 2026

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

A Phase 4 interventional study of plasma-derived FVIII/VWF concentrate in Von Willebrand Disease, sponsored by Grifols Therapeutics LLC. Withdrawn at 4 sites in Spain. Open to participants aged 2 Months to 6 Years. Per ClinicalTrials.gov, last updated 2026-05-22.

Sponsored by Grifols Therapeutics LLC (part of Grifols) · Phase 4, Interventional, and Treatment

Why this study was withdrawn
Due to recruitment difficulties for pediatric patients. The reason for study cancellation is not related to safety concerns or any adverse events.
Phase
Phase 4
Study type
Interventional
Enrollment
0
Allocation
Not applicable
Ages
2 Months to 6 Years
Sex
All
01

Study summary

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

Read the detailed description

This is a multicenter, prospective, open-label, and single-arm study. The study population is planned to include 8 pediatric subjects (\<6 years of age) with severe (type 2 or 3) hereditary VWD without inhibitors and with no active bleeding at the time of inclusion. Eligible subjects will receive a single dose of Fanhdi for a PK evaluation and will be followed for 12 months for which the efficacy and safety of Fanhdi will be assessed. In addition, the type 3 VWD subjects, after 6 months of follow-up of the first infusion, will receive the second dose as in the 1st PK evaluation and undergo a 2nd PK evaluation.

The study will consist of 2 phases:

  • PK profile evaluation in which all eligible subjects will receive a single dose of 80 IU/kg von Willebrand factor: Ristocetin cofactor activity (VWF:RCo) of Fanhdi. In addition, after 6 months of follow-up of the first infusion, type 3 VWD subjects will receive the second dose of Fanhdi and undergo a 2nd PK evaluation with a reduced sampling schedule.
  • A 12-month Follow-up period during which the safety and efficacy of Fanhdi will be assessed in the prevention and management of bleeding episodes and/or management of perioperative hemostasis during surgery and/or invasive procedures.
02

Conditions studied

  • Von Willebrand Disease

Keywords

  • pediatric
  • plasma-derived FVIII concentrate
03

In context

von Willebrand Diseases

96 studies on the registry are indexed under von Willebrand Diseases; 25 are open to participants now.

Browse von Willebrand Diseases studies →

Lead sponsor

Grifols Therapeutics LLC is the lead sponsor of 46 studies on the registry; 4 are open to participants now.

Of its 13 completed or terminated interventional studies of FDA-regulated products, 13 (100%) have results posted.

First submitted after Grifols Therapeutics LLC became part of Grifols.

Browse Grifols Therapeutics LLC studies →All Grifols studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Months to 6 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 form (ICF) provided by an authorized representative on behalf of the subject in accordance with local law and institutional policy.

Exclusion criteria

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 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), virus (HBV), virus (HCV), or (HIV), or have clinical signs and symptoms consistent with current HAV, HBV, HCV or HIV infection.
  9. Subjects presenting anemia ( \<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 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.
Terms in these criteria, in plain language(7)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
0 participants (actual)

Study arms

  • Experimental
    plasma-derived FVIII/VWF concentrate

    Pharmacokinetic single dose study with Fanhdi (high-purity Von Willebrand containing FVIII concentrate)

    Drug: plasma-derived FVIII/VWF concentrate

Interventions

  • Drugplasma-derived FVIII/VWF concentrate

    1 single dose of 80 IU/kg VWF:RCo of Fanhdi will be administered

    Also known as: Fanhdi

06

What researchers measure

Primary outcomes

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

    Cumulative area under the concentration time curve extrapolated to infinity of FVIII:C

    Time frame: Prior to the first infusion up to 72 hours postinfusion

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

    Cumulative area under the concentration time curve extrapolated to infinity of VWF:RCo

    Time frame: Prior to the first infusion up to 72 hours postinfusion

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

    Cumulative area under the concentration time curve extrapolated to infinity of VWF:Ag

    Time frame: Prior to the first infusion up to 72 hours postinfusion

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

    Cumulative area under the concentration time curve extrapolated to infinity of VWF:CB

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  5. AUC^0-T of FVIII:C

    Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of FVIII:C

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  6. AUC^0-T of VWF:RCo

    Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of VWF:RCo

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  7. AUC^0-T of VWF:Ag

    Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of VWF:Ag

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  8. AUC^0-T of VWF:CB

    Cumulative area under the concentration time curve calculated from 0 to time of last observed quantifiable concentration of VWF:CB

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  9. in vivo recovery of FVIII:C

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  10. in vivo recovery of VWF:RCo

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  11. in vivo recovery of VWF:Ag

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  12. in vivo recovery of VWF:CB

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  13. Half-life of FVIII:C

    Terminal elimination half-life

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  14. Half-life of VWF:RCo

    Terminal elimination half-life

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  15. Half-life of VWF:Ag

    Terminal elimination half-life

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  16. C^max of FVIII:C

    Maximum observed plasma and/or serum concentration of FVIII:C

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  17. C^max of VWF:RCo

    Maximum observed plasma and/or serum concentration of VWF:RCo

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  18. C^max of VWF:Ag

    Maximum observed plasma and/or serum concentration of VWF:Ag

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  19. C^max of VWF:CB

    Maximum observed plasma and/or serum concentration of VWF:CB

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  20. T^max of FVIII:C

    Time of maximum observed plasma and/or serum concentration of FVIII:C

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  21. T^max of VWF:RCo

    Time of maximum observed plasma and/or serum concentration of VWF:RCo

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  22. T^max of VWF:Ag

    Time of maximum observed plasma and/or serum concentration of VWF:Ag

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  23. T^max of VWF:CB

    Time of maximum observed plasma and/or serum concentration of VWF:CB

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  24. Mean residence time of FVIII:C

    Average amount of time that a single molecule of drug stays in the body.

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  25. Mean residence time of VWF:RCo

    Average amount of time that a single molecule of drug stays in the body of VWF:RCo

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  26. Mean residence time of VWF:Ag

    Average amount of time that a single molecule of drug stays in the body of VWF:Ag

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  27. Mean residence time of VWF:CB

    Average amount of time that a single molecule of drug stays in the body of VWF:CB

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  28. Clearance of FVIII:C

    Total plasma and/or serum clearance

    Time frame: Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion

  29. Clearance of VWF:RCo

    Total plasma and/or serum clearance

    Time frame: Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion

  30. Clearance of VWF:Ag

    Total plasma and/or serum clearance

    Time frame: Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion

  31. Clearance of VWF:CB

    Total plasma and/or serum clearance

    Time frame: Prior to the first infusion, 30 minutes postinfusion, 10 hours postinfusion, and at 24, 48, and 72 hours postinfusion

  32. Elimination rate constant of FVIII:C

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  33. Elimination rate constant of VWF:RCo

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  34. Elimination rate constant of VWF:Ag

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  35. Elimination rate constant of VWF:CB

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  36. Volume of distribution of FVIII:C

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  37. Volume of distribution of VWF:RCo

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  38. Volume of distribution of VWF:Ag

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  39. Volume of distribution of VWF:CB

    Time frame: Prior to the first infusion up to 72 hours postinfusion

  40. VWF multimeric pattern

    For type 3 VWD subjects

    Time frame: Prior to the first infusion up to 12 hours postinfusion

07

Study locations

4 sites
  • Hospital Sant Joan de Déu Barcelona
    Esplugues de Llobregat, Barcelona 08950, Spain
  • Hospital Universitario Virgen del Rocío
    Seville, Sevilla 41013, Spain
  • Hospital Universitario La Paz
    Madrid, Spain
  • Hospital Universitario Miguel Servet
    Zaragoza, Spain
08

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
Not marked actual or estimatedDates not marked actual or estimated

The record gives a start of Dec 2013, a primary completion of Dec 2024 and a completion of Dec 2024. It does not say whether these dates are actual or estimated.

How these figures are worked out, and which studies are left out of them.

Registered
Jun 10, 2015
First posted
Jun 16, 2015
Start
Dec 2013
Primary completionActual
Dec 10, 2024
CompletionActual
Dec 10, 2024
Last update
May 22, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on May 22, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT02472665
Lead sponsor
Grifols Therapeutics LLC
Collaborators
Instituto Grifols, S.A.
Responsible party
Sponsor
First posted
Jun 16, 2015
Start date
Dec 2013
Primary completion
Dec 10, 2024
Completion
Dec 10, 2024
Last update
May 22, 2026

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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