A Phase III Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic RO5534262 versus No Prophylaxis in Hemophilia A Patients with Inhibitors

Phase 1

• Conditions: Hemophilia A with Inhibitors; MedDRA version: 18.0 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 100000004850; MedDRA version: 18.0 Level: LLT Classification code 10053751 Term: Hemophilia A with anti factor VIII System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2015-002866-21-ES
• Lead Sponsor: Roche Farma, S.A., que representa en España a F. Hoffmann-La Roche Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-10-05
• Last Update Posted: 10 December 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 109

Inclusion Criteria

- Aged 12 years or older at the time of informed consent
- Body weight >= 40 kg at the time of screening
- Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (i.e., >= 5 Bethesda Units)
- Documentation of treatment with episodic or prophylactic bypassing agents for at least the last 24 weeks
- Adequate hematologic function
- Adequate hepatic function
- Adequate renal function
- For women who are not postmenopausal or surgically sterile: agreement to remain abstinent or use single or combined highly effective non-hormonal contraceptive methods
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 63
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

- Inherited or acquired bleeding disorder other than hemophilia A
- Ongoing (or plan to receive during the study) immune tolerance induction therapy or prophylaxis with FVIII
- History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator?s judgment
- Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or signs of thromboembolic disease
- Previous or concurrent autoimmune or connective tissue disease
- History of hypersensitivity associated with monoclonal antibody therapies or components of the RO5534262 injection
- Known HIV infection with CD4 count < 200 cells/microliter within 24 weeks prior to screening
- Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or planned use during the study, with the exception of antiretroviral therapy
- Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study or that would, in the opinion of the investigator or Sponsor, preclude the patient?s safe participation in and completion of the study or interpretation of the study results
- Planned surgery (excluding minor procedures such as tooth extraction or incision and drainage) during the study
- Receipt of
? RO5534262 in a prior investigational study
? An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration
? A non-hemophilia-related investigational drug within last 30 days or 5 half lives, whichever is shorter
? An investigational drug concurrently
- Unwillingness to use highly effective contraception methods for the specified duration in the protocol (females only, unless required otherwise by the local health authority)
- Clinically significant abnormality on screening evaluations or laboratory tests that, in the opinion of the investigator, may pose an additional risk in administering study drug to the patient
- Pregnancy or lactation, or intent to become pregnant during the study

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of prophylactic RO5534262 compared with no prophylaxis in patients with hemophilia A with inhibitors;Primary end point(s): Number of bleeds over time;Timepoint(s) of evaluation of this end point: Over 24 weeks or discontinuation of study participation, whichever occurs first;<br> Secondary Objective: ? To evaluate<br> - reduction in the number of bleeds<br> - reduction in the number of joint bleeds and target joint bleeds<br> - health-related quality of life<br> - health status<br> ? To evaluate the overall safety of prophylactic RO5534262<br> ? To characterize the exposure of prophylactic RO5534262<br>