Survey of Inhibitors in Plasma-Product Exposed Toddlers

Completed · Phase 4

Conditions studied: Hemophilia A

In brief

The primary objective of the study is to assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in previously untreated patients (PUPs) or minimally blood component-treated (MBCTPs) in the first 50 EDs or in the first 3 years from enrollment, whichever occurs first. .

Key facts

Study ID

NCT01064284

Run by

Fondazione Angelo Bianchi Bonomi

People needed

303

Starts

2010-01-01

Expected to finish

2015-05-01

Last updated by the study team

2017-08-25

Who can join

Age: any, up to 6. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Male subjects
• Any ethnicity
• Age <6 years
• Severe haemophilia A (FVIII:C <1%), as confirmed at enrolment by the central laboratory.
• o Those patients diagnosed locally as severe but subsequently found to have FVIII levels >= 1% on testing at the central laboratory will be separately recorded in the screening list.
• Previously untreated (0 EDs to any FVIII concentrates or blood products) or minimally treated (<5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate.
• o Patients not meeting these criteria will be separately recorded in the screening list.
• Negative inhibitor measurement at both local and central laboratory at screening
• Ability to comply with study requirements
• Signed informed consent of legal tutors o Patients who will not accept to enter into the study or to be randomized will be separately recorded.

You may not qualify if…

• Previous history of FVIII inhibitor
• Other congenital or acquired bleeding defects
• Plasma FVIII level >= 1%, as assayed at the central laboratory
• o Those patients originally diagnosed locally as severe but subsequently found to have FVIII levels ranging from 1% to 2% on testing at the central laboratory will be separately recorded in the screening list.
• Concomitant congenital or acquired immunodeficiency
• Concomitant treatment with systemic immunosuppressive drugs
• Concomitant treatment with any investigational drug

Where it is running

• City of Hope National Medical Center — Duarte, California, United States
• Children's Hospital Los Angeles (CHLA) — Los Angeles, California, United States
• Hemophilia and Thrombosis CenterUniversity of Colorado Denver - Anschutz Aurora — Aurora, Colorado, United States
• Rush Hemophilia & Trombophilia Center - Rush University Medical Center — Chicago, Illinois, United States
• Louisiana Center for Bleeding and Clotting Disorders, Tulane University Medical Center — New Orleans, Louisiana, United States
• University of Mississippi Medical Center, Division of pediatric Hematology/Oncology — Jackson, Mississippi, United States
• Children's Mercy Hospital — Kansas City, Missouri, United States
• Hemophilia Treatment Center of Las Vegas — Las Vegas, Nevada, United States
• MeritCare Roger Maris Cancer Center, Pediatric Oncology — Fargo, North Dakota, United States
• Cook Children's Medical Center — Fort Worth, Texas, United States
• Hospital de Ninos Sor Maria Ludovica La Plata Servicio de Hematologia — La Plata, Buenos Aires, Argentina
• Fundacion de la Hemofilia — Buenos Aires, Argentina
• Landes- Frauen- und Kinderklinik Linz Abteilung für Kinder- und Jugendheilkunde — Linz, Austria
• Medizinische Universität Wien, Dept. Paediatrics — Vienna, Austria
• Centro de Pesquisa Clinica HEMORIO - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti — Rio de Janeiro, Brazil
• Centro de Hematologia e Hemoterapia do e.s - Hemoes — Vitória, Brazil
• Hospital de Niños Dr. Luis Calvo Mackenna Centro Hemofílico — Santiago, Chile
• Centro de Hemofilicos del Hospital de Niños Roberto del Rio Instituto de Investigaciones Hematologicas — Santiago, Chile
• Paediatric Haematology department, Cairo University Pediatric Hospital — Cairo, Egypt
• Faculty of Medicine Ain Shams University Department Pediatrics — Cairo, Egypt
• Centre for Blood Disorders — Chennai, India
• St. John's Medical College & Hospital — Karnataka, India
• Kasturba Medical College, Manipal University — Karnataka, India
• Karnataka Hemophilia Care and Hematology Research Center — Karnataka, India
• Kerala Institute of Medical Science (KIMS) — Kerala, India

Full record on ClinicalTrials.gov

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