Protocol Summary

Protocol No.: OCR15074

Sponsor Protocol No.: BH30071

Protocol Title.: A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors

Principal Investigator: Rajasekhar, Anita

Objective: This is a randomized, global, multicenter, open-label, Phase 3 clinical study in participants with severe hemophilia A without inhibitors against Factor VIII (FVIII) who are 12 years or older. The study evaluates two prophylactic emicizumab regimens versus no prophylaxis in this population with emphasis on efficacy, safety, and pharmacokinetics.

Phase: Phase III

Age Group: Both

Age: 12 Years - N/A

Gender: All

Scope: National

Treatment:

Experimental: A: Emicizumab 1.5 mg/kg/week
Participants who received episodic treatment with FVIII prior to study entry will receive emicizumab prophylaxis at a dose of 3 milligrams per kilogram per week (mg/kg/week) subcutaneously for 4 weeks, followed by 1.5 mg/kg/week emicizumab subcutaneously until the end of study (maximum up to 2 years).

Experimental: B: Emicizumab 3 mg/kg/2 weeks
Participants who received episodic treatment with FVIII prior to study entry will receive emicizumab prophylaxis at a dose of 3 mg/kg/week subcutaneously for 4 weeks, followed by 3 mg/kg/2 weeks emicizumab subcutaneously until the end of study (maximum up to 2 years).

Active Comparator: C: No Prophylaxis
Participants who received episodic treatment with FVIII prior to study entry will be randomized to continue episodic FVIII treatment when they start the trial; they will have the opportunity to switch to emicizumab prophylaxis after 24 weeks on-study.

Experimental: D: Emicizumab 1.5 mg/kg/week (Pre-study FVIII Prophylaxis)
Participants who received FVIII prophylaxis prior to study entry will receive emicizumab prophylaxis at a dose of 3 mg/kg/week subcutaneously for 4 weeks, followed by 1.5 mg/kg/week emicizumab subcutaneously until the end of study (maximum up to 2 years).

Detailed Eligibility:

Inclusion Criteria:
- Body weight >/= 40 kilogram (kg) at the time of screening
- Diagnosis of severe congenital hemophilia A
- Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks
- Adequate hematologic function
- Adequate hepatic function
- Adequate renal function
- For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of less than (<) 1 percent (%) per year during the treatment period and for at least 5 elimination half-lives (24 weeks) after the last dose of study drug
Exclusion Criteria:
- Inherited or acquired bleeding disorder other than hemophilia A
- Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
- Conditions that may increase risk of bleeding or thrombosis
- History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
- Known human immunodeficiency virus (HIV) infection with cluster of differentiation (CD) 4 count ) 200 and meet all other criteria are eligible
- Use of systemic immunomodulators at enrollment or planned use during the study, with the exception of anti-retroviral therapy
- Participants who are at high risk for thrombotic microangiopathy (TMA) (for example, have a previous medical or family history of TMA), in the investigator's judgment
- Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study
- Planned surgery (excluding minor procedures) during the study
- Receipt of emicizumab 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 concurrently, within last 30 days or 5 half-lives, whichever is shorter
- Pregnant or lactating, or intending to become pregnant during the study

Applicable Conditions:

  • Pediatric Hemophilia A
  • Participation Institution:

  • UF Gainesville
  • More Information: View study listing on ClinicialTrials.gov http://www.clinicaltrials.gov/ct2/show/NCT02847637