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A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors (HAVEN 6)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04158648
Recruitment Status : Recruiting
First Posted : November 12, 2019
Last Update Posted : July 1, 2020
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

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).

Condition or disease Intervention/treatment Phase
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor Hemophilia A Drug: Emicizumab Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : February 10, 2020
Estimated Primary Completion Date : July 19, 2022
Estimated Study Completion Date : July 19, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia
Drug Information available for: Emicizumab

Arm Intervention/treatment
Experimental: Emicizumab
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.
Drug: Emicizumab
Four loading doses of emicizumab 3 mg/kg will be administered subcutaneously once a week (QW) for 4 weeks followed by participant's preference of one of the following maintenance regimens: 1.5 mg/kg QW, 3 mg/kg once every 2 weeks (Q2W), or 6 mg/kg once every 4 weeks (Q4W).
Other Names:
  • Hemlibra
  • RO5534262
  • RG6013
  • ACE910




Primary Outcome Measures :
  1. Number of Participants Who Experienced at Least One Adverse Event by Severity, According to the World Health Organization (WHO) Toxicity Grading Scale [ Time Frame: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after emicizumab discontinuation; up to approximately 30 months) ]
  2. Number of Participants Who Experienced at Least One Thromboembolic Event [ Time Frame: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after emicizumab discontinuation; up to approximately 30 months) ]
  3. Number of Participants Who Experienced at Least One Event of Thrombotic Microangiopathy [ Time Frame: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after emicizumab discontinuation; up to approximately 30 months) ]
  4. Number of Participants with at Least One Laboratory Abnormality [ Time Frame: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after emicizumab discontinuation; up to approximately 30 months) ]
    Laboratory parameters for hematology and blood chemistry will be measured and compared with a standard reference range. Values outside the standard reference range are considered abnormalities. Not every laboratory abnormality qualifies as an adverse event. A laboratory test result will be reported as an adverse event if it meets any of the following criteria: is accompanied by clinical symptoms; results in a change in study treatment or a medical intervention; or is clinically significant in the investigator's judgment.

  5. Number of Participants Who Experienced at Least One Injection-Site Reaction by Severity, According to the WHO Toxicity Grading Scale [ Time Frame: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after emicizumab discontinuation; up to approximately 30 months) ]
  6. Number of Participants with Adverse Events Leading to Study Drug Discontinuation [ Time Frame: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  7. Number of Participants Who Experienced at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event [ Time Frame: From Screening (Day -28 to -1) to Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after emicizumab discontinuation; up to approximately 30 months) ]
  8. Change from Baseline in Respiratory Rate Over Time [ Time Frame: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 30 months) ]
  9. Change from Baseline in Pulse Rate Over Time [ Time Frame: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 30 months) ]
  10. Change from Baseline in Body Temperature Over Time [ Time Frame: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 30 months) ]
  11. Change from Baseline in Systolic Blood Pressure Over Time [ Time Frame: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 30 months) ]
  12. Change from Baseline in Diastolic Blood Pressure Over Time [ Time Frame: Baseline, Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 30 months) ]
  13. Change from Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcB, QTcF, RR, PR, and QRS Intervals [ Time Frame: Baseline, Weeks 5, 25, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 30 months) ]
  14. Change from Baseline in Heart Rate Over Time, as Measured by Electrocardiogram (ECG) [ Time Frame: Baseline, Weeks 5, 25, and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (up to approximately 30 months) ]
  15. Model-Based Annualized Bleeding Rate for Treated Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    The model-based annualized bleeding rate (ABR) will be estimated using a negative binomial regression model.

  16. Median Calculated Annualized Bleeding Rate for Treated Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  17. Mean Calculated Annualized Bleeding Rate for Treated Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]

Secondary Outcome Measures :
  1. Model-Based Annualized Bleeding Rate for All Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    The model-based annualized bleeding rate (ABR) will be estimated using a negative binomial regression model.

  2. Median Calculated Annualized Bleeding Rate for All Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  3. Mean Calculated Annualized Bleeding Rate for All Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  4. Model-Based Annualized Bleeding Rate for Joint Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    The model-based annualized bleeding rate (ABR) will be estimated using a negative binomial regression model.

  5. Median Calculated Annualized Bleeding Rate for Joint Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  6. Mean Calculated Annualized Bleeding Rate for Joint Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  7. Model-Based Annualized Bleeding Rate for Target Joint Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    The model-based annualized bleeding rate (ABR) will be estimated using a negative binomial regression model.

  8. Median Calculated Annualized Bleeding Rate for Target Joint Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  9. Mean Calculated Annualized Bleeding Rate for Target Joint Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  10. Model-Based Annualized Bleeding Rate for Spontaneous Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    The model-based annualized bleeding rate (ABR) will be estimated using a negative binomial regression model.

  11. Median Calculated Annualized Bleeding Rate for Spontaneous Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  12. Mean Calculated Annualized Bleeding Rate for Spontaneous Bleeds [ Time Frame: Bleed data collected every week and on days of bleed from Baseline until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  13. Hemophilia Joint Health Scores Over Time [ Time Frame: Days -7 to -1, Weeks 25, 49, and every 24 weeks thereafter until Study Completion/Discontinuation Visit (after at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
  14. CATCH Questionnaire for Adult Participants: Change from Baseline in the Daily Activity Risk Perception and Impact Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  15. CATCH Questionnaire for Adult Participants: Change from Baseline in the Social Activity Risk Perception and Impact Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  16. CATCH Questionnaire for Adult Participants: Change from Baseline in the Recreational Activity Risk Perception and Impact Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  17. CATCH Questionnaire for Adult Participants: Change from Baseline in the Work Impact Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  18. CATCH Questionnaire for Adult Participants: Change from Baseline in the Preoccupation Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  19. CATCH Questionnaire for Adult Participants: Change from Baseline in the Treatment Burden Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  20. CATCH Questionnaire for Adult Participants: Change from Baseline in the Pain Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  21. CATCH Questionnaire for Pediatric Participants: Change from Baseline in the Daily Activity Risk Perception and Impact Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  22. CATCH Questionnaire for Pediatric Participants: Change from Baseline in the Social Activity Risk Perception and Impact Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  23. CATCH Questionnaire for Pediatric Participants: Change from Baseline in the Recreational Activity Risk Perception and Impact Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  24. CATCH Questionnaire for Pediatric Participants: Change from Baseline in the School Impact Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  25. CATCH Questionnaire for Pediatric Participants: Change from Baseline in the Preoccupation Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  26. CATCH Questionnaire for Pediatric Participants: Change from Baseline in the Treatment Burden Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  27. CATCH Questionnaire for Pediatric Participants: Change from Baseline in the Pain Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  28. CATCH Questionnaire for Caregivers: Change from Baseline in the Preoccupation Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  29. CATCH Questionnaire for Caregivers: Change from Baseline in the Treatment Burden Domain Score Over Time [ Time Frame: Baseline (Week 1), Weeks 13, 25, 37 and 49, and every 12 weeks thereafter until Study Completion/Discontinuation Visit (at least 52 weeks of emicizumab treatment in the study or 24 weeks after final dose of emicizumab; up to approximately 30 months) ]
    CATCH = Comprehensive Assessment Tool of Challenges in Hemophilia

  30. Number of Participants and Caregivers who Prefer Emicizumab SC Treatment, Their Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Week 17 [ Time Frame: Week 17 ]
    SC = subcutaneous; IV = intravenous

  31. Change from Baseline in Duration of Moderate to Vigorous Physical Activity Over Time [ Time Frame: Baseline (Weeks 1-2) and Weeks 12-13, 24-25, 36-37, and 48-49 ]
  32. Change from Baseline in Mean Daily Step Count Over Time [ Time Frame: Baseline (Weeks 1-2) and Weeks 12-13, 24-25, 36-37, and 48-49 ]
  33. Plasma Trough Concentration (Ctrough) of Emicizumab Over Time [ Time Frame: Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, and 49, and every 12 weeks thereafter until study completion/discontinuation (up to approximately 30 months) ]
  34. Number of Participants with Anti-Drug Antibodies Against Emicizumab at Baseline and Post-Baseline [ Time Frame: Pre-dose at Baseline (Week 1) and Weeks 5, 13, 25, 33, 41, and 49, and every 12 weeks thereafter until study completion/discontinuation (up to approximately 30 months) ]
  35. Number of Participants Who Develop Anti-FVIII Inhibitors Over Time [ Time Frame: Screening (Day -28 to -1) and Weeks 1, 13, 25, 37, and 49, and every 12 weeks thereafter until study completion/discontinuation (up to approximately 30 months) ]
  36. Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change from Baseline in the MBQ Total Score Over Time [ Time Frame: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 30 months) ]
  37. Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change from Baseline in the Heaviness Subscale Score Over Time [ Time Frame: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 30 months) ]
  38. Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change from Baseline in the Quality of Life Subscale Score Over Time [ Time Frame: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 30 months) ]
  39. Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change from Baseline in the Irregularity Subscale Score Over Time [ Time Frame: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 30 months) ]
  40. Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change from Baseline in the Pain Subscale Score Over Time [ Time Frame: Baseline (Week 1), Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, and every 4 weeks thereafter until Study Completion (up to approximately 30 months) ]
  41. Menstruation Diary with the Pictorial Blood Assessment Chart (PBAC) for Female Participants of Childbearing Potential: PBAC Scores Over Time [ Time Frame: Baseline (Day 1) and monthly (on days of menstruation) until Study Completion (up to approximately 30 months) ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04158648


Contacts
Layout table for location contacts
Contact: Reference Study ID Number: BO41423 www.roche.com/about_roche/roche_worldwide.htm 888-662-6728 (U.S. Only) global-roche-genentech-trials@gene.com

Locations
Show Show 27 study locations
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT04158648    
Other Study ID Numbers: BO41423
2019-002179-32 ( EudraCT Number )
First Posted: November 12, 2019    Key Record Dates
Last Update Posted: July 1, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

Qualified researchers may request access to individual patient level data through the request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/).

For further details on Roche's Global Policy on Sharing of Clinical Study Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).


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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Hoffmann-La Roche:
Mild Hemophilia A Without Factor VIII Inhibitors
Moderate Hemophilia A Without Factor VIII Inhibitors
Additional relevant MeSH terms:
Layout table for MeSH terms
Hemophilia A
Deficiency Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Malnutrition
Nutrition Disorders