Overview

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

Status:
Recruiting
Trial end date:
2023-04-18
Target enrollment:
0
Participant gender:
All
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).
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Hoffmann-La Roche
Treatments:
Antibodies, Bispecific
Factor VIII
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)