This clinical trial is to study Immune Tolerance Induction, by Factor VIII concentrate containing von Willebrand factor, in severe or moderate haemophilia A patients with inhibitors.

Immune Tolerance Induction, by Factor VIII concentrate containing von Willebrand factor, in severe or moderate haemophilia A patients with inhibitors.

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2014/07/004749

Enrollment

Registered

2014-07-18

Start date

Unknown

Completion date

Unknown

Last updated

2021-11-24

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

Conditions

Health Condition 1: null- Severe or moderate haemophilia A patients with inhibitors.

Interventions

Intervention1: Lyophilized plasma-derived factor VIII: The product belongs to the factor VIII concentrates class, containing also VW factor in an average ratio VW/VIII of more than 1.4: 5. Dose: 50 I

Eligibility

Inclusion criteria

Inclusion criteria: 1. Subjects (his/her parent/legal representative), must have given a written informed consent 2. Male children: age 3. Severe or moderate hemophilia A (FVIII 4. High responders (clinical history of inhibitor peak > 5BU) Or low-responders with potential bleedings, assessed by responsible physicians as not to be treated with high FVIII doses. 5. Any level of inhibitor at study enrollment. 6. Willingness and ability to participate in the study. 7. No other experimental treatments

Exclusion criteria

Exclusion criteria: 1. Any clinically relevant abnormality, in hematological, biochemical and urinary routine examinations, or any condition or treatment which in the investigatorâ??s opinion, makes the patient not eligible for the study. 2. Intolerance to active substance or to any of the excipients of FVIII / VWF concentrate. 3. Concomitant systemic treatment with immunosuppressive drugs.

Design outcomes

Primary

Measure	Time frame
Inhibitor reduction (Details in Brief Summary). Timepoint: 6, 12 and 33 months of treatment.	—

Secondary

Measure	Time frame
Efficacy: Time to achieve IT: Complete or Partial response. IT persistence: Absence of relapse, assessed at 12 months from the IT achievement. FVIII genetic defect role in IT achievement: Role of FVIII mutations in influencing IT achievement. Role of an immediate IT to delayed IT in IT induction: Time elapsing between the onset of the inhibitor and the beginning of treatment. Timepoint: 12 months (for IT persistence).;Safety: Incidence of AE during the course of prophylactic treatment, with severity, correlation with the IP and final outcome. The tolerability of the IP will also be assessed at the different infusion rates of administration. Trend of the inhibitor title will be also assessed during the course of prophylactic treatment. Timepoint: NA.;Treatment compliance: Patientâ??s adherence to the optimal prolonged treatment.Timepoint: NA.	—

Measure

Time frame

Efficacy: Time to achieve IT: Complete or Partial response. IT persistence: Absence of relapse, assessed at 12 months from the IT achievement. FVIII genetic defect role in IT achievement: Role of FVIII mutations in influencing IT achievement. Role of an immediate IT to delayed IT in IT induction: Time elapsing between the onset of the inhibitor and the beginning of treatment. Timepoint: 12 months (for IT persistence).;Safety: Incidence of AE during the course of prophylactic treatment, with severity, correlation with the IP and final outcome. The tolerability of the IP will also be assessed at the different infusion rates of administration. Trend of the inhibitor title will be also assessed during the course of prophylactic treatment. Timepoint: NA.;Treatment compliance: Patientâ??s adherence to the optimal prolonged treatment.Timepoint: NA.

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Countries

Egypt, India

Contacts

Public ContactDr Shariq Anwar

JSS Medical Research India Private Limited

sonika.newar@jssresearch.com91-129-6613500

Outcome results

None listed

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