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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05181618
Other study ID # MO42623
Secondary ID 2020-005092-1320
Status Active, not recruiting
Phase Phase 4
First received
Last updated
Start date June 20, 2022
Est. completion date December 28, 2026

Study information

Verified date May 2024
Source Hoffmann-La Roche
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

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.


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Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Emicizumab
The emicizumab dosing regimen will be 3 milligrams per kilogram of body weight (mg/kg) subcutaneously (SC) once a week (QW) for 4 weeks followed by participant 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) in agreement with the investigator.

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Sponsors (1)

Lead Sponsor Collaborator
Hoffmann-La Roche

Countries where clinical trial is conducted

United States,  Brazil,  Canada,  Germany,  Hungary,  Italy,  Morocco,  Serbia,  Spain,  Tunisia,  Turkey,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Joint Status at 6 Months, Based on Centrally Reviewed Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) Scores with a Specific Focus on the Synovitis Score in Participants with Synovitis 6 Months
Primary Joint Status at 12 Months, Based on Centrally Reviewed Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) Scores with a Specific Focus on the Synovitis Score in Participants with Synovitis 12 Months
Primary Joint Status at 24 Months, Based on Centrally Reviewed Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) Scores with a Specific Focus on the Synovitis Score in Participants with Synovitis 24 Months
Primary Joint Status at 36 Months, Based on Centrally Reviewed Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) Scores with a Specific Focus on the Synovitis Score in Participants with Synovitis 36 Months
Primary Clinical Joint Status at 6 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment 6 Months
Primary Clinical Joint Status at 12 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment 12 Months
Primary Clinical Joint Status at 24 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment 24 Months
Primary Clinical Joint Status at 36 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment 36 Months
Primary Joint Status at 36 Months, Based on Centrally Reviewed International Prophylaxis Study Group (IPSG) Score (with MRI) 36 Months
Primary Number of Problem Joints at 6 Months Problem joints are defined as joints having chronic joint pain and/or limited range of movement due to compromised joint integrity (i.e., chronic synovitis and/or hemophilic arthropathy) with or without persistent bleeding. 6 Months
Primary Number of Problem Joints at 12 Months Problem joints are defined as joints having chronic joint pain and/or limited range of movement due to compromised joint integrity (i.e., chronic synovitis and/or hemophilic arthropathy) with or without persistent bleeding. 12 Months
Primary Number of Problem Joints at 24 Months Problem joints are defined as joints having chronic joint pain and/or limited range of movement due to compromised joint integrity (i.e., chronic synovitis and/or hemophilic arthropathy) with or without persistent bleeding. 24 Months
Primary Number of Problem Joints at 36 Months Problem joints are defined as joints having chronic joint pain and/or limited range of movement due to compromised joint integrity (i.e., chronic synovitis and/or hemophilic arthropathy) with or without persistent bleeding. 36 Months
Primary Percentage of Joints That are Problem Joints at 6 Months 6 Months
Primary Percentage of Joints That are Problem Joints at 12 Months 12 Months
Primary Percentage of Joints That are Problem Joints at 24 Months 24 Months
Primary Percentage of Joints That are Problem Joints at 36 Months 36 Months
Primary Change from Baseline in the CATCH Domain Scores Over Time, as Assessed with the Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire for Adult Participants At Baseline (Day 1), Months 3, 6, 9, 12, 18, 24, 30, and 36
Primary Change from Baseline in the CATCH Domain Scores Over Time, as Assessed with the CATCH Questionnaire for Pediatric Participants At Baseline (Day 1), Months 3, 6, 9, 12, 18, 24, 30, and 36
Primary Change from Baseline in the Average Daily Time Spent Doing Physical Activities by Intensity Level Over Time, as Assessed by Participant Responses to the International Physical Activity Questionnaire Short Format (IPAQ-SF) At Baseline (Day 1), Months 3, 6, 9, 12, 18, 24, 30, and 36
Primary Daily Step Count Over Time, as Measured with a Wearable Activity Tracker From Baseline until end of treatment period (up to 36 months)
Primary Daily Metabolic Equivalents of Tasks (METs) Over Time, as Measured with a Wearable Activity Tracker From Baseline until end of treatment period (up to 36 months)
Primary Daily Time Spent in Moderate to Vigorous Physical Activity (MVPA) Over Time, as per the Activity Tracker Default Categorization From Baseline until end of treatment period (up to 36 months)
Primary Daily Active Minutes of Physical Activity Over Time, as Measured with a Wearable Activity Tracker From Baseline until end of treatment period (up to 36 months)
Primary Model-Based Annualized Bleed Rates for All Bleeds, Treated Bleeds, Spontaneous Bleeds, Joint Bleeds, Treated Joint Bleeds, and Target Joint Bleeds From Baseline until end of treatment period (up to 36 months)
Primary Mean Calculated Annualized Bleed Rates for All Bleeds, Treated Bleeds, Spontaneous Bleeds, Joint Bleeds, Treated Joint Bleeds, and Target Joint Bleeds From Baseline until end of treatment period (up to 36 months)
Primary Median Calculated Annualized Bleed Rates for All Bleeds, Treated Bleeds, Spontaneous Bleeds, Joint Bleeds, Treated Joint Bleeds, and Target Joint Bleeds From Baseline until end of treatment period (up to 36 months)
Primary Number of Participants who Prefer Emicizumab SC Treatment, Their Previous Hemophilia IV Treatment, or Have No Preference, as Assessed Through Use of the Emicizumab Preference Survey at Month 6 At Month 6
Secondary Number of Participants with at Least One Adverse Event, with Severity Determined According to the World Health Organization (WHO) Toxicity Scale From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)
Secondary Number of Participants with at Least One Thromboembolic Event From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)
Secondary Number of Participants with at Least One Event of Thrombotic Microangiopathy (TMA) From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)
Secondary Number of Participants with at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)
Secondary Number of Participants with at Least One Injection-Site Reaction From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)
Secondary Number of Participants with Anti-Drug Antibodies (ADAs) Against Emicizumab at Baseline and During the Study At Baseline, Months 6, 12, 24, and 36
Secondary Number of Participants who Develop Anti-FVIII Inhibitors During the Study At Months 6, 12, 24, and 36
See also
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