Study on Joint Health in Hemophilia A and B Patients Using Efmoroctocog Alfa or Eftrenonacog Alfa

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What is this study about?

This clinical trial is focused on studying the effects of two treatments for patients with Hemophilia A and Hemophilia B. These are genetic disorders where blood does not clot properly, leading to excessive bleeding. The treatments being studied are called efmoroctocog alfa and eftrenonacog alfa. Efmoroctocog alfa is a recombinant fusion protein that acts like a human coagulation factor VIII, while eftrenonacog alfa is a similar protein that acts like factor IX. These treatments are given as injections and are used to prevent bleeding episodes in patients with hemophilia.

The purpose of this study is to assess the health of joints in patients with hemophilia who are receiving these treatments over an 18-month period. The study will use ultrasound, a type of imaging that uses sound waves to create pictures of the inside of the body, to evaluate joint health. Participants will receive either efmoroctocog alfa or eftrenonacog alfa as part of their regular treatment plan. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatments.

Throughout the study, participants will have regular check-ups to monitor their joint health and overall well-being. The study aims to understand how these treatments affect joint health and to gather information that could help improve care for people with hemophilia. The study will last for 18 months, during which participants will continue their usual care while being monitored for any changes in their condition.

1 enrollment and consent

Upon joining the study, you will provide a signed and dated informed consent. If you are under the legal age, your legally authorized representative will provide consent, and your assent will be obtained as per local regulations.

2 baseline assessment

You will undergo a baseline assessment, which includes documenting your treatment history and any bleeding episodes from the past six months. This information is crucial for establishing your starting point in the study.

3 start of prophylactic treatment

You will begin or continue prophylactic treatment with either efmoroctocog alfa or eftrenonacog alfa. These medications are administered as a solution for injection. The specific dosage and frequency will be determined by your healthcare provider.

4 regular follow-up visits

Throughout the 18-month study period, you will attend regular follow-up visits. During these visits, your joint health will be assessed using ultrasound to monitor any changes in your joint status.

5 data collection

Data will be collected on various aspects of your health, including joint health scores, bleeding rates, and physical function. This information will help evaluate the effectiveness of the treatment.

6 end of study assessment

At the end of the 18-month period, a final assessment will be conducted to evaluate changes in your joint health and overall outcomes. This will include a comparison of your baseline and end-of-study data.

Who Can Join the Study?

  • Age must be 6 years or older.
  • Must have a diagnosis of hemophilia A or B (a condition where blood does not clot properly).
  • Must have at least 6 months of documented treatment history, including treatment prescriptions and bleeding episodes, before the start of the study.
  • Must have been previously treated for hemophilia A or B with any approved recombinant (lab-made) or plasma-derived FVIII or FIX concentrate for at least 6 months.
  • Must have started preventive treatment with rFVIIIFc or rFIXFc (specific medications for hemophilia) before joining the study or at the latest by the first study visit, following local rules.
  • Must provide signed and dated informed consent. If under the legal age, consent must be provided by a legally authorized representative, and agreement (assent) should be obtained from children, following local rules.
  • To join the florio HAEMO sub-study, must have used the florio HAEMO app (a medical device used in regular clinical practice) for at least 3 months and agree to have data collected from the app by providing separate consent or assent.

Who Cannot Join the Study?

  • Patients who do not have Hemophilia A or Hemophilia B cannot participate.
  • Patients who are not within the age range of 2 to 4 years old cannot participate.
  • Patients who are not part of the specified clinical trial group cannot participate.
  • Patients who are not male or female cannot participate.
  • Patients who are not considered part of a vulnerable population cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Centre Hospitalier Universitaire De Lille Lille France
IRCCS Humanitas Research Hospital Rozzano Italy
Hospital Universitario De Salamanca Salamanca Spain
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Hospital Universitario Y Politecnico La Fe Valencia Spain
University Hospital Of Clermont-Ferrand Clermont Ferrand France

Other Sites

Site Name City Country Status
Spitalul Clinic Municipal De Urgenta Timisoara Timisoara Romania
Uniteversity Muliprofile Hospital For Active Treatment Tsaritsa Yoanna-Isul EAD Sofia Bulgaria
Centre Hospitalier Metropole Savoie Chambery France
University Medical Center Ljubljana Ljubljana Slovenia
KBC Zagreb Zagreb Croatia
Centre Hospitalier Universitaire Rouen Rouen France
Spitalul Clinic Judetean De Urgenta Sf. Spiridon Iasi Iasi Romania
Centre Hospitalier Universitaire De Toulouse Toulouse France
Fakultni Nemocnice Brno Brno Czechia
Assistance Publique Hopitaux De Paris Paris France
Hospices Civils De Lyon Lyon France
University Hospital Olomouc Olomouc Czechia
Centre Hospitalier Universitaire De Nimes Nimes France
Centre Hospitalier Universitaire De Poitiers Poitiers France
Universita’ Di Pisa Pisa Italy
Institute Of Hematology And Blood Transfusion Prague Czechia
Hospital Universitario Rio Hortega Valladolid Spain
Spitalul Judetean De Urgenta Dr. Constantin Opris Baia Mare Baia Mare Romania
Centre Hospitalier Universitaire De Caen Normandie Caen France
Hôpital Pontchaillou-CHU Rennes Rennes France
Krajska nemocnice Liberec a.s. Liberec Czechia
Children’s Health Ireland Dublin Ireland
Medical Centre Hungarian Defence Forces Budapest Hungary
Hospital Maison Blanche Reims France
Specialized Hospital For Active Treatment Of Oncological Diseases Sofia Region EOOD Sofia Bulgaria
Bicetre Hospital Le Kremlin-Bicetre France
Fundeni Clinical Institute Bucharest Romania
Hospital Hotel Dieu Nantes France
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Cnwk Da Nmfqq Vandoeuvre Les Nancy France
Swzttbtd Czicsg Mgdzsxlit Fshlanvniua Cbqybid Craiova Romania
Criq dh Tjwwie Hieevtm Ttuppnbof Chambray Les Tours France
Fwtgsksp nuhkzglzz Mtmfc a Hxsoaqf Prague Czechia
Aqkvlnkssj Pfctewdd Hfggqogb Dt Mssfmkudh Marseille France
Hhkekkba Dg Ld Sylfl Celf I Skpd Pkh Barcelona Spain
Ftyokfetb Pzhj Lv Ifjabsfoulsyd Beadwhbfz Dhr Hmdbxsmh Uezshqwkgdarh Lq Pab Madrid Spain
Haauwsve Ucqizlgtqnzaf dm A Cktptk A Coruna Galicia Spain
Hspbofzc Uekzkonqtjxgjx Svbefhiive &skrule Hkqkaxx dr Hsbutyowumb STRASBOURG, Alsace France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Bulgaria Bulgaria
Not recruiting
15.03.2023
Croatia Croatia
Not recruiting
15.03.2023
Czechia Czechia
Not recruiting
15.03.2023
France France
Not recruiting
15.03.2023
Hungary Hungary
Not recruiting
15.03.2023
Ireland Ireland
Not recruiting
15.03.2023
Italy Italy
Not yet recruiting
15.03.2023
Romania Romania
Not recruiting
15.03.2023
Slovenia Slovenia
Not recruiting
15.03.2023
Spain Spain
Not recruiting
15.03.2023

Trial locations

Efmoroctocog alfa is a medication used in the treatment of hemophilia A. Hemophilia A is a condition where the blood does not clot properly due to a lack of a specific protein. This medication helps to replace the missing protein, allowing the blood to clot more effectively. In this clinical trial, efmoroctocog alfa is used as a preventive treatment to help reduce bleeding episodes and improve joint health over time.

Eftrenonacog alfa is a medication used for treating hemophilia B, which is similar to hemophilia A but involves a different missing protein. This medication works by providing the body with the protein it lacks, helping the blood to clot properly. In the trial, eftrenonacog alfa is used as a preventive measure to help manage bleeding and protect joint health in patients with hemophilia B.

Hemophilia A – Hemophilia A is a genetic disorder caused by a deficiency of clotting factor VIII, which is essential for blood clotting. This condition leads to prolonged bleeding after injuries, surgeries, or even spontaneously. Over time, repeated bleeding episodes can cause damage to joints, muscles, and other tissues. The disease primarily affects males, as it is linked to the X chromosome. Individuals with Hemophilia A may experience frequent nosebleeds, easy bruising, and excessive bleeding from cuts or injuries. Joint bleeding is common, leading to pain and swelling.

Hemophilia B – Hemophilia B is a genetic disorder resulting from a deficiency of clotting factor IX, which is crucial for proper blood coagulation. Similar to Hemophilia A, this condition causes prolonged bleeding after injuries or surgeries and can also occur spontaneously. The disease is inherited in an X-linked recessive pattern, predominantly affecting males. Repeated bleeding episodes can lead to joint damage and muscle hemorrhages over time. Symptoms include frequent nosebleeds, easy bruising, and excessive bleeding from minor cuts or injuries. Joint bleeding is a common issue, causing pain and swelling.

Trial ID:
2022-502921-16-00
Protocol code:
Sobi.HAEM89-007
Trial Phase:
Therapeutic confirmatory (Phase III)

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