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Hemophilia A, Prophylaxis of bleeding

Bayer Identifier:

22082

ClinicalTrials.gov Identifier:

NCT05643560

EudraCT Number:

Not Available

EU CT Number:

Not Available
An observational study called JOIHA to learn more about how well the treatment with Jivi works to prevent problems with joints in adults with hemophilia A.

Trial purpose

This is an observational study in which data from people with hemophilia A who decide on their own or by recommendation of their doctors to take Jivi are collected and studied. In observational studies, only observations are made without specified advice or interventions.
Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called “clotting factor 8” (FVIII). FVIII is naturally found in the blood where it causes the blood to clump together to help prevent and stop bleeding. People with lower levels of FVIII or with FVIII that does not work properly may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding.
The study treatment, Jivi (also called damoctocog alfa pegol), is already available for doctors to prescribe to people with hemophilia A to treat and prevent bleeding. It works by replacing the missing FVIII, or the FVIII that does not work properly.
People with hemophilia A need frequent injections of FVIII products into the vein. So called standard half-life (SHL) products need to be given 2 to 4 times a week for the prevention of bleeding. In recent years, new products like Jivi called extended half-life (EHL) products have available. These products last longer in the body so that they require to be given less often with injections up to every 7 days. Thus, these treatments may be easier and more comfortable to stick to in daily life. There is no general plan concerning the best amount of treatment and the frequency of injections for the prevention of bleeding, since the severity may be different and individual risk factors have to be considered. Doctors often decide on a treatment plan based on patient's disease and response.
Clinical studies have already shown that people with hemophilia A benefit from the treatment with Jivi. However, there are no data available coming from the real-world about how well Jivi works to support joint health, measured by ultrasound (US) examination and HEAD-US score.
In this study, researchers want to learn more about how well Jivi works if used for prolonged periods of treatment under real-world settings to prevent problems with joints in people with hemophilia A. How well it works means to find out if participants` joints status can be improved by treatment with Jivi.
To do this, researchers will collect data about participants` joints status by
• making images of participants` joints by using sound waves (ultrasound), and
• using HEAD-US score
after 24 months of treatment with Jivi.
The researchers will then compare these data to the participants` joints status before treatment start with Jivi.

Besides this data collection, no further tests or examinations are planned in this study.

Some participants in this study will already be receiving treatment with Jivi as part of their regular care no more than 12 months. And some participants will start to take Jivi in this study as prescribed by their doctors during routine practice according to the approved product information.

The researchers will collect data from each patient for a period of 26 months after initiation of the Jivi treatment.

There are no required visits or tests in this study

Key Participants Requirements

Sex

All

Age

18 - N/A
  • - Diagnosis hemophilia A with FVIII:C ≤2%.
    - Patients ≥ 18 years of age.
    - Previously treated for hemophilia A (PTPs).
    - Patients who had initiated damoctocog alfa pegol maximum 12 months prior to enrollment into the study or who initiate damoctocog alfa pegol treatment at the enrollment , where the decision to initiate damoctocog alfa pegol has been made before the decision for study participation.
    - Patients having a HEAD-US measurement available in the 2 months prior to damoctocog alfa pegol treatment initiation.
    - Patients without previous history of FVIII inhibitors or patients with previous history of FVIII inhibitors on standard prophylaxis therapy for at least 1 year prior to baseline (damoctocog alfa pegol treatment initiation).
    - No current evidence of FVIII inhibitor or clinical suspicion of FVIII inhibitor.
    - Signed informed consent/assent.
  • - Concurrent participation in an investigational program with interventions outside of routine clinical practice.
    - Diagnosis of any other bleeding/coagulation disorder other than hemophilia A.
    - Patient on immune tolerance induction (ITI) treatment at the time of enrollment.

Trial summary

Enrollment Goal
33
Trial Dates
December 2022 - June 2027
Phase
Phase 4
Could I Receive a placebo
No
Products
Jivi (Damoctocog, BAY94-9027)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Recruiting
Many LocationsMany Locations, Italy

Primary Outcome

  • Occurrence of an unchanged or decreased HEAD-US score at 24 months after treatment initiation with damoctocog alfa pegol
    The Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score is based on the three markers for the main joints (knees, elbows, ankles): synovitis (score: 0-2), cartilage (score: 0-4), subchondral bone (score: 0-2).
    date_rangeTime Frame:
    24 months

Secondary Outcome

  • Occurrence of an unchanged or decreased HEAD-US score at 12 months after treatment initiation with damoctocog alfa pegol
    date_rangeTime Frame:
    12 months
  • Occurrence of a decrease in joint ABR together with a decrease in HEAD-US score 12 and 24 months after treatment initiation with damoctocog alfa pegol
    ABR: Annualized bleeding rate.
    date_rangeTime Frame:
    12 months and 24 months
  • Change in the number of target joints (as defined by ISTH) 12 months and 24 months after treatment initiation with damoctcog alfa pegol in relation to HEAD-US score
    ISTH: International Society on Thrombosis and Hemostasis.
    date_rangeTime Frame:
    From baseline to 12 months and 24 months
  • Change in HEAD-US score and HJHS score 12 months and 24 months after treatment initiation with damoctocog alfa pegol
    The Hemophilia Joint Health Score (HJHS) measures joint health, in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in hemophilia: the knees, ankles, and elbows.
    date_rangeTime Frame:
    From baseline to 12 months and 24 months
  • Change in physical activity (IPAQ-SF) 12 months and 24 months after treatment initiation with damoctocog alfa pegol by categorized HEAD-US score change
    The International Physical Activity and sports short form (IPAQ-SF) questionnaire consists of 7 items and will ask about the time one spent being physically active in the last 7 days.
    date_rangeTime Frame:
    From baseline to 12 months and 24 months
  • Change in pain (Visual Analogue Scale) 12 months and 24 months after treatment initiation with damoctocog alfa pegol by categorized HEAD-US score change
    Pain Visual Analogue Scale (VAS) score categorized as “no pain (0-4)”, “mild pain (5-44)”, “moderate pain (45-74)” and “severe pain (75-100)” vs. HEAD-US score change categories (worsened, unchanged, improved).
    date_rangeTime Frame:
    From baseline to 12 months and 24 months

Trial design

Long-term treatment outcome of Jivi® prophylaxis on joint health in adult patients with hemophilia A
Trial Type
Observational
Intervention Type
Drug
Trial Purpose
Other
Allocation
N/A
Blinding
N/A
Assignment
N/A
Trial Arms
N/A

Additional Information

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