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Weekly shot may shield joints in hemophilia a patients

NCT ID NCT06752850

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This study tests whether a once-weekly medication (efanesoctocog alfa) can improve or prevent joint damage in 37 people with moderate or severe hemophilia A. Participants receive the drug for 12 months, and doctors use ultrasound and MRI to check joint health. The goal is to see if this treatment reduces swelling and lowers the risk of joint bleeds.

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

37 people

The number who actually took part.

Started

Dec 2024

Expected to finish

Dec 2026

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

12 years and older

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: 1. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. Parents' or legally designated representatives' consent is required for patients who are \<18 years of age or unable to give consent, or as applicable per local laws. Patients who are \<18 years of age should provide assent in addition to the parents'/legally designated representatives' consent, if appropriate. 2. Male or female patients who are ≥12 years of age and diagnosed with moderate or severe haemophilia A (defined as ≤5% of normal FVIII clotting activity) at the time of signing the ICF. 3. A female patient is eligible to participate if she is not pregnant at enrolment and does not plan to become pregnant during the study. A woman of child-bearing potential (WOCBP) must have a negative highly sensitive serum pregnancy test at the Screening Visit. 4. Must have received prophylactic treatment per local label with any marketed FVIII product or emicizumab for ≥12 months prior to the Baseline Visit. 5. Have at least one eligible index joint (ankle, elbow, knee). 6. Have 12 months of documented pre-study treatment data on haemophilia prescriptions and on treated bleeding episodes prior to the Baseline Visit. 7. Willingness and the ability of the patient or their legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study. Exclusion Criteria: 1. Blood clotting disorders other than haemophilia A 2. Already on efanesoctocog alfa treatment 3. Positive inhibitor result (assessed by local laboratory) from the Screening Visit, defined as ≥0.6 Bethesda units (BU)/mL. 4. History of inhibitors without successful immune tolerance induction (ITI) * Successful ITI is defined as: * Negative inhibitor titer (\<0.6 BU/mL) * FVIII recovery \> 66% of expected * FVIII half-life ≥ 6 hours 5. ITI performed within the last 2 years prior to the Baseline Visit. 6. Currently receiving treatment with any of the prohibited concomitant medications, as specified by the protocol. 7. Planned major orthopaedic procedure in any eligible index joint during the course of the study. 8. Patients are not eligible for participation in the study if they cannot undergo MRI assessments at the Baseline Visit. 9. Patients with known hypersensitivity to the active substance or to any of the excipients. 10. Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures. 11. Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), within 3 months prior to inclusion in the study.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Sobi Investigational Site

    Milan, Italy

  • Sobi Investigational Site

    Naples, Italy

  • Sobi Investigational Site

    Rozzano, Italy

  • Sobi Investigational Site

    Oslo, Norway

  • Sobi Investigational Site

    Barcelona, Spain

  • Sobi Investigational Site

    Madrid, Spain

  • Sobi Investigational Site

    Seville, Spain

  • Sobi Investigational Site

    Valencia, Spain

  • Sobi Investigational Site

    Gothenburg, Sweden

  • Sobi Investigational Site

    Malmö, Sweden

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