Survey of Inhibitors in Plasma-Product Exposed Toddlers

Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

IRCT

Registry ID

IRCT201104106161N1

Enrollment

300

Registered

2011-04-30

Start date

2009-04-01

Completion date

Unknown

Last updated

2018-02-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A. Hereditary factor VIII deficiency

Interventions

Intervention 1: Recombinant Factor VIII concentrate. Intervention 2: plasma-derived Factor VIII concentrate.

Treatment - Drugs

Recombinant Factor VIII concentrate

plasma-derived Factor VIII concentrate

Eligibility

Sex/Gender

Male

Age

No minimum to 6 Years

Inclusion criteria

Inclusion criteria: Inclusion criteria: 1-Male subjects; 2-Any ethnicity; 3-Age <6 years; 4-Severe haemophilia A (FVIII:C <1%), as confirmed by the central laboratory; 5-Previously untreated (0 EDs to any FVIII concentrate or blood products) or minimally treated (<5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate; 6-Negative inhibitor measurement at both local and central laboratory at screening; 7-Ability to comply with study requirements; 8-Signed informed consent of legal tutors. Exclusion criteria: 1-Plasma FVIII level =1%, as assayed at the central laboratory; 2-Previous history of FVIII inhibitor; 3-Other congenital or acquired bleeding defects; 4-Concomitant congenital or acquired immunodeficiency; 5-Concomitant treatment with systemic immunosuppressive drugs; 6-Concomitant treatment with any investigational drug.

Exclusion criteria

Exclusion criteria:

Design outcomes

Primary

Measure	Time frame
To assess the immunogenicity of plasma derived VWF/FVIII and rFVIII concentrates. Timepoint: in the first 50 EDs or in the first 3 years from enrolment. Method of measurement: by determining the frequency of inhibitor development.	—

Secondary

Measure	Time frame
To evaluate modality of occurrence of inhibitors (number of EDs, titre at onset, etc). Timepoint: in the first 50 EDs or in the first 3 years from enrolment, whichever comes first. Method of measurement: record and analysis of characteristics during the treatment course.	—

Countries

Iran (Islamic Republic of)

Contacts

Public ContactDr. Bahman Vasseghi

Clinart

bahman.vasseghi@clinart.net0097144370551

Outcome results

None listed

Source: IRCT (via WHO ICTRP) · Data processed: Feb 4, 2026