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Can a once-weekly shot protect joints in hemophilia a? new study aims to find out.

NCT ID NCT05181618

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 This study
Running, but no longer taking on new participants.
Completed
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 28, 2026 · Updated 4 times

Summary

This Phase IV study is testing emicizumab (Hemlibra) in 136 people aged 13 to 70 with moderate or severe hemophilia A who do not have inhibitors. The goal is to see if this drug, given as a shot under the skin, can improve joint health, increase physical activity, and enhance overall well-being compared to their previous treatment. Participants will be followed for up to 36 months, with regular checks on joint status, bleeding rates, and daily activity using a wearable tracker.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Emicizumab (Hemlibra)
What this could lead to
If successful, this study could show that emicizumab helps protect joints and improves daily life for people with hemophilia A, offering a more convenient treatment option.
What could go wrong
This is an open-label, single-arm study, so results may be biased. It is also a Phase IV trial, meaning the drug is already approved, so the focus is on refining use rather than a breakthrough. Risks include thromboembolic events and injection site reactions.

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 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

136 people

The number who actually took part.

Started

Jun 2022

Expected to finish

Nov 2027

An estimate. End dates often move.

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

13 to 69 years

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 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 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 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 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 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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Conditions

The condition(s) this trial relates to.

hemophilia A moderately severe hemophilia A severe hemophilia A

As listed by the trial registrant

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

Moderate hemophilia A Severe hemophilia A

Contacts and locations

Locations

  • AOU Careggi

    Florence, Tuscany, 50134, Italy

  • AOU Federico II

    Naples, Campania, 80131, Italy

  • Akdeniz Uni School of Medicine

    Antalya, 07059, Turkey (Türkiye)

  • Aziza Othmana Hospital

    Tunis, Tunisia

  • CHU Farhat Hached

    Sousse, 4000, Tunisia

  • Charité Universitätsklinikum Berlin

    Berlin, 13353, Germany

  • Complejo Hospitalario Universitario A Coruña (CHUAC)

    A Coruña, 15006, Spain

  • Ege Uni Medical School

    Izmir, 35100, Turkey (Türkiye)

  • Gazi Universitesi Tip Fakultesi

    Ankara, 06500, Turkey (Türkiye)

  • Hamilton Health Sciences Corporation

    Hamilton, Ontario, L9H 2B7, Canada

  • Hospital Regional Universitario Carlos Haya

    Málaga, 29010, Spain

  • Hospital Universitario Vall de Hebron

    Barcelona, 08035, Spain

  • Hospital Universitario la Paz

    Madrid, 28046, Spain

  • Hospital das Clinicas - UNICAMP

    Campinas, São Paulo, 13083-878, Brazil

  • Hospital das Clínicas Faculdades Médicas de Ribeirão Preto

    Ribeirão Preto, São Paulo, 14051-140, Brazil

  • Hospital de la Santa Creu i Sant Pau

    Barcelona, 08025, Spain

  • Hôpital d'enfants de Rabat - Service d'hémato-oncologie pédiatrique

    Rabat, 10090, Morocco

  • IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, Lombardy, 20122, Italy

  • Istanbul University Cerrahpasa Medical Faculty

    Istanbul, 34098, Turkey (Türkiye)

  • Manchester University NHS Foundation Trust (MFT)

    Manchester, M13 9WL, United Kingdom

  • Oklahoma Children's Hospital ? Jimmy Everest Center

    Oklahoma City, Oklahoma, 73104, United States

  • Orthopaedic Institute for Children

    Los Angeles, California, 90007, United States

  • Policlinico Univ. A. Gemelli

    Rome, Lazio, 00168, Italy

  • St Thomas Westminster

    London, SE1 7EH, United Kingdom

  • University Clinical Centre of Serbia

    Belgrade, 11000, Serbia

  • University of Miami Medical Center

    Miami, Florida, 33136, United States

  • Universitätsklinikum Bonn

    Bonn, 53127, Germany

  • Észak-Pesti Centrumkórház - Honvédkórház

    Budapest, 1134, Hungary

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