A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis

  • Hemophilia A
Trial Status:

Recruiting

This trial runs in
Cities
  • Barcelona
  • Bonn
  • Hamilton
  • Los Angeles
  • Milano
  • Málaga
  • Napoli
  • New Hyde Park
  • Roma
Trial Identifier:

NCT05181618 2020-005092-13 MO42623

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      The source of the below information is the publicly available website ClinicalTrials.gov. It has been summarised and edited into simpler language.

      The below information is taken directly from the publicly available website ClinicalTrials.gov within a week of any updates, and has not been edited.

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      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.

      Hoffmann-La Roche Sponsor
      Phase 4 Phase
      NCT05181618 , MO42623 , 2020-005092-13 Trial Identifier
      Emicizumab Treatments
      Severe Hemophilia A, Moderate Hemophilia A Condition
      Official Title

      A Multicenter, Open-Label Phase IV Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes, in Participants Aged ≥13 and <70 Years With Severe or Moderate Hemophilia A Without FVIII Inhibitors on Emicizumab Prophylaxis

      Eligibility Criteria

      All Gender
      ≥13 Years & ≤ 69 Years Age
      No Healthy Volunteers
      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

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