Trial for bleeding disorder drug in toddlers pulled before it started
NCT ID NCT02472665
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study was designed to test how FANHDI, a clotting factor concentrate, works in children under 6 with severe von Willebrand disease (types 2 or 3). The goal was to measure how the drug moves through the body and how well it controls bleeding. However, the trial was withdrawn before enrolling any participants, so no data were collected.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- FANHDI (a plasma-derived factor VIII/von Willebrand factor concentrate)
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 4
Runs after approval, following long-term safety and how well the treatment works in everyday use.
- Start date
-
Dec 2013
- Finished
-
Dec 2024
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
2 months to 6 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Von Willebrand disease are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Hospital Sant Joan de Déu Barcelona
Esplugues de Llobregat, Barcelona, 08950, Spain
-
Hospital Universitario La Paz
Madrid, Spain
-
Hospital Universitario Miguel Servet
Zaragoza, Spain
-
Hospital Universitario Virgen del Rocío
Seville, Sevilla, 41013, Spain
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Newborn screening study aims to catch rare diseases at birth
- Tiny camera may spot hidden bleeding in rare blood disorder
- New hope for kids with rare bleeding disease: study tests rVWF therapy
- Could a simple shot replace IV treatments for severe bleeding disorder?
- New study tracks joint replacement success in bleeding disorder patients
- New hope for toddlers with rare bleeding disorder: wilate shows promise