A clinical study exploring the use of rFVIIIFc in patients with severe Haemophilia A who are undergoing Immune Tolerance Induction (ITI) for the first time

Phase 1

• Conditions: Hemophilia A; MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850; MedDRA version: 20.0 Level: LLT Classification code 10053751 Term: Hemophilia A with anti factor VIII System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2017-000373-36-DE
• Lead Sponsor: Bioverativ Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-08-28
• Last Update Posted: 16 December 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 30

Inclusion Criteria

1.Ability of the subject or his legally authorized representative (e.g., parent or legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations.
2.Male subjects of any age diagnosed with severe hemophilia A (as confirmed from the medical record).
3. Currently diagnosed with high titer inhibitors (historical peak =5 BU/mL, according to medical records)
4.Previously treated with any plasma-derived or recombinant conventional or Extended Half-Life FVIII.
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1. Other coagulation disorder(s) in addition to hemophilia A.
2. Previous ITI
3. History of hypersensitivity or anaphylaxis associated with any rFVIIIFc administration.
4. Planned major surgery scheduled during the study unless deferred until after study completion (minor surgery such as tooth extraction or insertion/replacement of central venous access device is allowed)
5. Abnormal renal function (serum creatinine >1.5 mg/dL or 2x upper limit of normal (ULN) for subject age based on local laboratory range) as assessed by local laboratory.
6. Serum alanine aminotransferase or aspartate aminotransferase >5 × upper limit of normal (ULN) as assessed by local laboratory.
7. Serum total bilirubin >3 × ULN as assessed by local laboratory
8. Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy, other than rFVIIIFc, for investigational use is administered within 30 days (or 5 half-lives of the agent, whichever is longer) prior to the Baseline Visit.
9. Inability to comply with study requirements.
10. Presence of any medical or psychological condition or laboratory result that in the opinion of the Investigator can interfere with the subject’s ability to comply with the protocol requirements or make the subject not appropriate for inclusion to the study and treatment with Elocta/Eloctate (rFVIIIFc)
11. Other unspecified reasons that, in the opinion of the Investigator or Sponsor, make the subject unsuitable for enrollment.
12. Unwillingness or inability to comply with the requirements of the protocol, including the presence of any condition (physical, mental, or social) that prevents the subject from participating in visits as scheduled or entering required information into the EPD in a timely manner
13. Concurrent systemic treatment with immunosuppressive drugs within 12 weeks (3 months) prior to Screening. Exceptions to this include: ribavirin for treatment of hepatitis C virus (HCV), and/or systemic steroids (a total of 2 courses of pulse treatments lasting no more than 7 days within 12 weeks [3 months] prior to Day 1) and/or inhaled steroids
14. High risk of cardiovascular, cerebrovascular, or other thromboembolic events, as judged by the Investigator
The following criteria refer to tests performed within 26 weeks (6.5 months) prior to Screening. If not available, a new test will be drawn at the Screening visit.
15. Cluster of differentiation 4 (CD4) lymphocytes <200 mm3 at Screening, if known as human immunodeficiency virus (HIV) antibody positive based on medical history or HIV testing
16. Viral load of >400 copies/mL at Screening, if known as HIV antibody positive, based on medical history.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified