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New shot cuts bleeding in hemophilia a patients

NCT ID NCT04158648

Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 28, 2026 · Updated 2 times

Summary

This study tested a medicine called emicizumab in 73 people with mild or moderate hemophilia A who do not have inhibitors. The goal was to see if it safely reduces the number of bleeding episodes. Participants received regular injections, and the results showed fewer bleeds compared to before treatment. This is a disease-control approach, not a cure, as ongoing treatment is needed.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

73 people

The number who actually took part.

Started

Feb 2020

Finished

Dec 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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)

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Conditions

The condition(s) this trial relates to.

hemophilia A mild hemophilia A moderately severe hemophilia A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemophilia A Mild hereditary factor viii deficiency disease without inhibitor Moderate hereditary factor viii deficiency disease without inhibitor

Contacts and locations

Locations

  • Amsterdam UMC Location AMC

    Amsterdam, 1105 AZ, Netherlands

  • CH de Bicetre

    Le Kremlin-Bicêtre, 94275, France

  • Cardiff and Vale NHS Trust

    Cardiff, CF14 4XW, United Kingdom

  • Charlotte Maxeke Johannesburg Hospital

    Johannesburg, 2193, South Africa

  • Childrens Hospital LA

    Los Angeles, California, 90027, United States

  • Cliniques Universitaires St-Luc

    Brussels, 1200, Belgium

  • Eastern Health - General Hospital

    St. John's, Newfoundland and Labrador, A1B 3V6, Canada

  • Great Ormond street Hospital for Children NHS Foundation Trust

    London, WC1N 3HR, United Kingdom

  • Groupe Hospitalier Necker Enfants Malades

    Paris, 75015, France

  • Hemophilia of Georgia Center for Bleeding & Clotting Disorders

    Atlanta, Georgia, 30308, United States

  • Hopital Cardio-vasculaire Louis Pradel

    Bron, 69677, France

  • Hospital Universitario Virgen del Rocio

    Seville, 41013, Spain

  • Hospital Universitario la Paz

    Madrid, 28046, Spain

  • Indiana Hemophilia & Thrombosis center

    Indianapolis, Indiana, 46260, United States

  • Instytut Hematologii i Transfuzjologii

    Warsaw, 02-776, Poland

  • Kaye Edmonton Clinic

    Edmonton, Alberta, T6G 1Z1, Canada

  • Klinikum der Universität München, Campus Innenstadt

    München, 80336, Germany

  • Royal Free Hospital

    London, NW3 2QG, United Kingdom

  • UZ Leuven Gasthuisberg

    Leuven, 3000, Belgium

  • University of Michigan, C.S. Mott Children's Hospital

    Ann Arbor, Michigan, 48109, United States

  • Universitätsklinikum Bonn

    Bonn, 53127, Germany

  • Washington Institute for Coagulation

    Seattle, Washington, 98101, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.