Evaluating Fanhdi® for treating severe Von Willebrand Disease in young children

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

This study is testing if a new treatment called Fanhdi can help young children with severe Von Willebrand Disease manage their bleeding better.

Quick facts

• Phase: PHASE4
• Study type: Interventional
• Enrollment: 8 (estimated)
• Ages: 2 Months to 6 Years
• Sex: All
• Sponsor: Grifols Therapeutics LLC (industry)
• Locations: 4 sites (Esplugues de Llobregat, Barcelona and 3 other locations)
• Trial ID: NCT02472665 on ClinicalTrials.gov

What this trial studies

This multicenter, open-label study aims to assess the efficacy and safety of Fanhdi®, a high-purity von Willebrand factor and FVIII concentrate, in pediatric patients under 6 years old with severe hereditary Von Willebrand Disease (type 2 or 3). Participants will receive a single dose of Fanhdi for pharmacokinetic evaluation and will be monitored for 12 months to evaluate the treatment's effectiveness in preventing and managing bleeding episodes. Type 3 VWD subjects will receive a second dose after 6 months for further evaluation. The study will help determine the optimal use of Fanhdi in this vulnerable population.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients under 6 years of age diagnosed with severe hereditary Von Willebrand Disease without active bleeding.

Not a fit: Patients with acquired Von Willebrand Disease or those experiencing active bleeding at the time of treatment will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve bleeding management in young children with severe Von Willebrand Disease.

How similar studies have performed: While there have been studies on FVIII concentrates, this specific approach with Fanhdi in a pediatric population is novel.

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.

Where this trial is running

Esplugues de Llobregat, Barcelona and 3 other locations

• Hospital Sant Joan de Déu Barcelona — Esplugues de Llobregat, Barcelona, Spain (RECRUITING)
• Hospital Universitario La Paz — Madrid, Spain (RECRUITING)
• Hospital Universitario Virgen del Rocío — Seville, Spain (RECRUITING)
• Hospital Universitario Miguel Servet — Zaragoza, Spain (RECRUITING)

Study contacts

• Study coordinator: Núria Ribó

Conditions: Von Willebrand Disease, pediatric, plasma-derived FVIII concentrate