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

• Status: Recruiting
• Trial end date: 2022-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Grifols Therapeutics LLC; Instituto Grifols, S.A.
• Collaborator: Instituto Grifols, S.A.
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

1. Subjects diagnosed with severe hereditary VWD (VWF:RCo<15-20 IU/dL) of any type

2. Subjects less than 6 years of age

3. Subjects that do not adequately respond to treatment with desmopressin (DDAVP) or had a previous treatment failure with DDAVP. Note: Inadequate response to DDAVP treatment or a previous treatment failure with DDAVP is not required for subjects with VWD type 2b and 3. A confirmed diagnosis in medical history is sufficient for this criterion, since treatment with DDAVP is not indicated in or contraindicated for these VWD patients.

4. Signed informed consent form provided by an authorized representative on behalf of the subject in accordance with local law and institutional policy.

Exclusion Criteria:

1. Subject has been diagnosed of acquired VWD

2. Subjects bleeding at the time of the first infusion or the 10 days prior to the infusion

3. Subjects treated with DDAVP or another FVIII containing VWF concentrate during the 5 days prior to the infusion of the Fanhdi. This may be reduced to 3 days for subjects with type 3 VWD.

4. Subject is known or suspected to have present or past inhibitor activity (antibodies) directed against FVIII or VWF

5. Subject is known to have history of intolerance to any Fanhdi® containing substance

6. Subject is known to have history of anaphylactic reaction(s) to blood or blood components

7. Subjects presenting with severe platelet dysfunctions due to drugs (aspirin, other NSAIDs, etc.) or other pathologies (uremic thrombopathy, hematological diseases)

8. Subjects with known previous infection with Hepatitis A Virus (HAV), Hepatitis B Virus (HBV), Hepatitic C Virus (HCV), or HIV or clinical signs and symptoms consistent with current HAV, HBV, HCV or HIV infection.

9. Subjects presenting with anemia (hemoglobulin <11 g/dL)

10. Subjects diagnosed with metabolic diseases that are not clinically controlled, such as diabetes mellitus, that could potentially interfere with the interpretations of the study

11. Subjects participating in another clinical study within 30 days prior to screening or has received any investigational treatment within 3 months prior to screening

12. If it is anticipated that the subject will be treated with other products containing FVIII or VWF different from Fanhdi® during a period of one year

13. The subject is unlikely to adhere to the protocol requirements of the study