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Active, not recruitingNCT05181618Beyond ABRUpdated Oct 23, 2026

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

A Phase 4 interventional study of Emicizumab in Severe Hemophilia A and Moderate Hemophilia A, sponsored by Hoffmann-La Roche. Active, not recruiting at 26 sites in 12 countries. Open to participants aged 13 Years to 69 Years. Per ClinicalTrials.gov, last updated 2026-10-23.

Sponsored by Hoffmann-La Roche · Phase 4, Interventional, and Basic science

Updated Oct 23, 2026Primary completion movedStudy completion moved+1 moreGo to Updates ↓
Phase
Phase 4
Study type
Interventional
Enrollment
136
Allocation
Non-randomized
Ages
13 Years to 69 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Severe Hemophilia A
  • Moderate Hemophilia A

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03

In context

Hemophilia A

844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.

This study's enrollment of 136 is above the median of 30 across 499 interventional studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Hoffmann-La Roche is the lead sponsor of 2,101 studies on the registry; 87 are open to participants now.

Of its 330 completed or terminated interventional studies of FDA-regulated products, 248 (75%) have results posted.

Browse Hoffmann-La Roche studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
13 Years to 69 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 : agreement to remain abstinent (refrain from heterosexual intercourse) or use during the treatment period and for at least 24 weeks after the final dose of emicizumab

Exclusion criteria

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 2 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 2 years ago who are not experiencing pain in the joint(s), the participant may be enrolled but the specific joint(s) 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 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 in the investigator's judgement
  • 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 diseases) that may currently increase the risk of bleeding or thrombosis
  • History of clinically significant associated with monoclonal antibody therapies or components of the emicizumab injection
  • Planned surgery during the emicizumab loading dose phase
  • Known 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 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
Terms in these criteria, in plain language(7)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Autoimmune disease

A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.

Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 4
Primary purpose
Basic science
Allocation
Non-randomized
Intervention model
Single group
Masking
None (open label)
Enrollment
136 participants (actual)

Study arms

  • Experimental
    Cohort 1, Hemophilia A and Without Arthropathy: Emicizumab

    Cohort 1 comprises participants with severe or moderate hemophilia A and with no synovitis and no osteochondral damage (Haemophilia Early Arthropathy Detection with Ultrasound \[HEAD-US\] score of 0) in all index joints.

    Drug: Emicizumab

  • Experimental
    Cohort 2, Hemophilia A and with Synovitis Only: Emicizumab

    Cohort 2 comprises participants with severe or moderate hemophilia A and with synovitis (HEAD-US synovitis score of ≥1) in at least one index joint and no osteochondral damage (HEAD-US bone and cartilage score of 0).

    Drug: Emicizumab

  • Experimental
    Cohort 3, Hemophilia A and with Osteochondral Damage: Emicizumab

    Cohort 3 comprises participants with severe or moderate hemophilia A and with osteochondral damage (HEAD-US bone and cartilage score of ≥1) in at least one index joint and with any synovitis score.

    Drug: Emicizumab

Interventions

  • DrugEmicizumab

    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.

    Also known as: Hemlibra, RO5534262, RG6013, ACE910

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: 6 Months

  2. 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

    Time frame: 12 Months

  3. 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

    Time frame: 24 Months

  4. 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

    Time frame: 36 Months

  5. Clinical Joint Status at 6 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment

    Time frame: 6 Months

  6. Clinical Joint Status at 12 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment

    Time frame: 12 Months

  7. Clinical Joint Status at 24 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment

    Time frame: 24 Months

  8. Clinical Joint Status at 36 Months, Based on the Hemophilia Joint Health Score (HJHS v2.1) Excluding Gait Assessment

    Time frame: 36 Months

  9. Joint Status at 36 Months, Based on Centrally Reviewed International Prophylaxis Study Group (IPSG) Score (with MRI)

    Time frame: 36 Months

  10. 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.

    Time frame: 6 Months

  11. 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.

    Time frame: 12 Months

  12. 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.

    Time frame: 24 Months

  13. 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.

    Time frame: 36 Months

  14. Percentage of Joints That are Problem Joints at 6 Months

    Time frame: 6 Months

  15. Percentage of Joints That are Problem Joints at 12 Months

    Time frame: 12 Months

  16. Percentage of Joints That are Problem Joints at 24 Months

    Time frame: 24 Months

  17. Percentage of Joints That are Problem Joints at 36 Months

    Time frame: 36 Months

  18. 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

    Time frame: At Baseline (Day 1), Months 3, 6, 9, 12, 18, 24, 30, and 36

  19. Change from Baseline in the CATCH Domain Scores Over Time, as Assessed with the CATCH Questionnaire for Pediatric Participants

    Time frame: At Baseline (Day 1), Months 3, 6, 9, 12, 18, 24, 30, and 36

  20. 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)

    Time frame: At Baseline (Day 1), Months 3, 6, 9, 12, 18, 24, 30, and 36

  21. Daily Step Count Over Time, as Measured with a Wearable Activity Tracker

    Time frame: From Baseline until end of treatment period (up to 36 months)

  22. Daily Metabolic Equivalents of Tasks (METs) Over Time, as Measured with a Wearable Activity Tracker

    Time frame: From Baseline until end of treatment period (up to 36 months)

  23. Daily Time Spent in Moderate to Vigorous Physical Activity (MVPA) Over Time, as per the Activity Tracker Default Categorization

    Time frame: From Baseline until end of treatment period (up to 36 months)

  24. Daily Active Minutes of Physical Activity Over Time, as Measured with a Wearable Activity Tracker

    Time frame: From Baseline until end of treatment period (up to 36 months)

  25. Model-Based Annualized Bleed Rates for All Bleeds, Treated Bleeds, Spontaneous Bleeds, Joint Bleeds, Treated Joint Bleeds, and Target Joint Bleeds

    Time frame: From Baseline until end of treatment period (up to 36 months)

  26. Mean Calculated Annualized Bleed Rates for All Bleeds, Treated Bleeds, Spontaneous Bleeds, Joint Bleeds, Treated Joint Bleeds, and Target Joint Bleeds

    Time frame: From Baseline until end of treatment period (up to 36 months)

  27. Median Calculated Annualized Bleed Rates for All Bleeds, Treated Bleeds, Spontaneous Bleeds, Joint Bleeds, Treated Joint Bleeds, and Target Joint Bleeds

    Time frame: From Baseline until end of treatment period (up to 36 months)

  28. 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

    Time frame: At Month 6

Secondary outcomes

  1. Number of Participants with at Least One Adverse Event, with Severity Determined According to the World Health Organization (WHO) Toxicity Scale

    Time frame: From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)

  2. Number of Participants with at Least One Thromboembolic Event

    Time frame: From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)

  3. Number of Participants with at Least One Event of Thrombotic Microangiopathy (TMA)

    Time frame: From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)

  4. Number of Participants with at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event

    Time frame: From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)

  5. Number of Participants with at Least One Injection-Site Reaction

    Time frame: From Baseline until 24 weeks after the final dose of emicizumab (up to 3.5 years)

  6. Number of Participants with Anti-Drug Antibodies (ADAs) Against Emicizumab at Baseline and During the Study

    Time frame: At Baseline, Months 6, 12, 24, and 36

  7. Number of Participants who Develop Anti-FVIII Inhibitors During the Study

    Time frame: At Months 6, 12, 24, and 36

07

Study locations

26 sites
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedDate before it movedWhere the date was before it movedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Jun 2022 (actual). Primary completion is estimated for Nov 2026, 52 months after the start.

Of 39 phase 4 interventional studies under Hemophilia A, half reached primary completion within 31 months of starting (the median), counting those still running; a quarter within 13 and three quarters within 112. This study's planned 52 months is above that.

Since we started tracking on Sep 25, 2026, the dates moved once, on Oct 23, 2026: primary completion Nov 2027 → Nov 2026 and completion Nov 2027 → Dec 2026.

Earlier changes are on the record history on ClinicalTrials.gov ↗

How these figures are worked out, and which studies are left out of them.

Registered
Dec 6, 2021
First posted
Jan 6, 2022
StartActual
Jun 20, 2022
Primary completionEst.
Nov 19, 2026was Nov 2027
CompletionEst.
Dec 21, 2026was Nov 2027
Last update
Oct 23, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Sites
2 sites removed
Show 2 removed
  • Orthopaedic Institute for Children · Los Angeles, United States
  • AOU Federico II · Naples, Italy
Oct 23, 2026
Primary completion
Nov 30, 2027→Nov 19, 2026
Oct 23, 2026
Study completion
Nov 30, 2027→Dec 21, 2026
Oct 23, 2026
Show all 1 update
  1. Oct 23, 2026
    2 sites removed
    Show 2 removed
    • Orthopaedic Institute for Children · Los Angeles, United States
    • AOU Federico II · Naples, Italy
    Primary completion Nov 30, 2027→Nov 19, 2026
    Study completion Nov 30, 2027→Dec 21, 2026
    + 2 other changes: identifiers and verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT05181618
Lead sponsor
Hoffmann-La Roche
Responsible party
Sponsor
First posted
Jan 6, 2022
Start date
Jun 20, 2022
Primary completion
Nov 19, 2026 (estimated)
Completion
Dec 21, 2026 (estimated)
Last update
Oct 23, 2026

Study contacts

Clinical Trials
study director · Hoffmann-La Roche

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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