A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

Brief Title

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

Official Title

A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors

Brief Summary

      This is a multicenter, open-label, single-arm study designed to evaluate the safety,
      efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or
      moderate hemophilia A without inhibitors against factor VIII (FVIII).

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Model-Based Annualized Bleed Rate for Treated Bleeds

Secondary Outcome

 Model-Based Annualized Bleed Rate for All Bleeds

Condition

Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor

Intervention

Emicizumab

Study Arms / Comparison Groups

 Emicizumab

Description:  Participants with mild and moderate hemophilia A without factor VIII (FVIII) inhibitors will be enrolled to receive the emicizumab loading dose regimen followed by the participant's preference of one of 3 maintenance dose regimens.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

Start Date

February 10, 2020

Completion Date

April 18, 2023

Primary Completion Date

October 30, 2021

Eligibility Criteria

        Inclusion Criteria:

          -  Diagnosis of mild (FVIII level between >5% and <40%) or moderate (FVIII level between
             ≥1% and ≤5%) congenital Hemophilia A without FVIII inhibitors

          -  Weight ≥3 kilograms (kg)

          -  Need for prophylaxis based on investigator assessment

          -  A negative test for inhibitor (i.e., <0.6 Bethesda Units per milliliter [BU/mL])
             within 8 weeks prior to enrollment

          -  No documented inhibitor (i.e., <0.6 BU/mL), FVIII half-life <6 hours, or FVIII
             recovery <66% in the last 5 years

          -  Documentation of the details of prophylactic or episodic FVIII treatment and of number
             of bleeding episodes for at least the last 24 weeks prior to enrollment

          -  Adequate hematologic hepatic and renal function

          -  For women of childbearing potential: agreement to remain abstinent or use
             contraception (as defined in the protocol) during the treatment period and for at
             least 24 weeks after the final dose of study drug

        Exclusion Criteria:

          -  Inherited or acquired bleeding disorder other than mild or moderate congenital
             hemophilia A

          -  History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the
             investigator's judgment

          -  Previous (within the last 12 months) or current treatment for thromboembolic disease
             or signs of thromboembolic disease

          -  Other conditions that may currently increase the risk of bleeding or thrombosis

          -  History of clinically significant hypersensitivity associated with monoclonal antibody
             therapies or components of the emicizumab injection

          -  Planned surgery during the emicizumab loading dose phase (surgeries in participants on
             emicizumab from Week 5 onwards are allowed)

          -  Known HIV infection with CD4 counts <200 cells per microlitre (/μL)

          -  Concomitant disease, condition, significant abnormality on screening evaluation or
             laboratory tests, or treatment that could interfere with the conduct of the study, or
             that would in the opinion of the investigator, pose an additional unacceptable risk in
             administering study drug to the participant

          -  Receipt of any of the following: An investigational drug to treat or reduce the risk
             of hemophilic bleeds within 5 half-lives of last drug administration with the
             exception of prior emicizumab prophylaxis; A non-hemophilia-related investigational
             drug within last 30 days or 5 half-lives, whichever is shorter; or Any other
             investigational drug currently being administered or planned to be administered

          -  Inability to comply with the study protocol in the opinion of the investigator

          -  Pregnant or breastfeeding, or intending to become pregnant during the study (women of
             childbearing potential must have a negative serum pregnancy test result within 7 days
             prior to initiation of study drug)

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Clinical Trials, ,

Location Countries

Belgium

Location Countries

Belgium

Administrative Informations

NCT ID

NCT04158648

Organization ID

BO41423

Secondary IDs

2019-002179-32

Responsible Party

Sponsor

Study Sponsor

Hoffmann-La Roche

Study Sponsor

Clinical Trials, Study Director, Hoffmann-La Roche

Verification Date

November 2022