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

Launched by GRIFOLS THERAPEUTICS LLC · Jun 15, 2015

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called Fanhdi® for young children under 6 years old who have severe types of Von Willebrand Disease (VWD). VWD is a bleeding disorder that makes it difficult for blood to clot properly. The trial aims to see how well Fanhdi works and how safe it is for these young patients. To be eligible for this study, children must have been diagnosed with severe hereditary VWD and meet specific health criteria. Importantly, they should not have active bleeding or a history of certain allergies or infections.

If your child participates in this trial, they will receive Fanhdi and be monitored closely by healthcare professionals. The study is currently recruiting participants, and parents or guardians will need to provide consent for their child's involvement. This trial could help improve the understanding and treatment of VWD in pediatric patients, potentially leading to better care for children with this condition.

Gender

ALL

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.