Principal Investigator
Enrollment Status
Clinical Trial ID
Clinical Trial Summary
Study MO42623 is a Phase IV, multicenter, open-label, three cohort study designed to evaluate
the impact of emicizumab prophylaxis on overall health, physical activity, and joint outcomes
in participants aged ≥13 and <70 years with severe hemophilia A without factor VIII (FVIII)
inhibitors or moderate hemophilia A without FVIII inhibitors who are receiving FVIII
prophylaxis and who will start emicizumab treatment as part of this study.
Phase
Phase 4
Funding Agency/Sponsor
Industry
Disease
Other
Enrollment Eligibility
Inclusion Criteria:
- Diagnosis of severe congenital hemophilia A (intrinsic factor VIII [FVIII] level <1%)
or moderate congenital hemophilia A (intrinsic FVIII level ≤5%) if previously
prescribed prophylaxis
- A negative test for FVIII inhibitor (i.e., <0.6 Bethesda Units) during screening
period
- No history of FVIII inhibitory antibodies (<0.6 BU/mL using the Bethesda assay) in the
last 5 years. Participants who completed successful immune tolerance induction (ITI)
at least 5 years before screening are eligible, provided they have had no evidence of
inhibitor recurrence (permanent or temporary) as may be indicated by detection of an
inhibitor, FVIII half-life <6 hours, or FVIII recovery <66% since completing ITI
- Participants who were on standard FVIII prophylaxis, defined as the regular
administration of FVIII to prevent bleeding, for at least the last 24 weeks, can be
enrolled regardless of the number of bleeds during this period
- Adequate hematologic, hepatic and renal function
- For women of childbearing potential: agreement to remain abstinent (refrain from
heterosexual intercourse) or use contraception during the treatment period and for at
least 24 weeks after the final dose of emicizumab
Exclusion Criteria:
- Inherited or acquired bleeding disorder other than severe congenital hemophilia A
(intrinsic FVIII level <1%) or moderate congenital hemophilia A (intrinsic FVIII level
≤5%) without FVIII inhibitors who were previously prescribed prophylaxis for at least
24 weeks
- Participants who have previously received emicizumab prophylaxis
- Participants that plan to have joint replacement, joint procedure, synovectomy or
synoviorthesis at screening
- Participants who had joint replacement, joint procedure, synovectomy or
synoviorthesis: Less than 3 years ago; OR, More than 3 years ago and are still
experiencing pain in the joint. For participants who had joint replacement, joint
procedure, synovectomy or synoviorthesis more than 3 years ago who are not
experiencing pain in the joint, the participant may be enrolled but the specific joint
in which the procedure was conducted will be excluded from the study
- Participants who have conditions other than hemophilia A that can affect joint health
and structure (e.g., osteoarthritis) or with severely impaired mobility due to
conditions other than hemophilia A
- Participants with known reduced bone mineral density defined as clinically relevant
vitamin D deficiency
- Participants with pre-existing uncontrolled or unstable cardiovascular disease not
receiving targeted medication or in a stable condition
- Participants not eligible for MRI
- History of illicit drug or alcohol abuse within 48 weeks prior to screening
- Participants who are at high risk for thrombotic microangiopathy (TMA)
- Previous (within the last 12 months) or current treatment for thromboembolic disease
(with the exception of previous catheter-associated thrombosis for which
anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease
- Other conditions (e.g., certain autoimmune diseases) 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
- Known HIV infection not controlled by medication
- 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 at screening; A
non-hemophilia-related investigational drug within last 30 days or 5 half-lives at
screening, whichever is shorter; or, Any other investigational drug currently being
administered or planned to be administered
- Inability to comply with the study protocol
- Pregnant or breastfeeding, or intending to become pregnant during the study