check_circleStudy Completed

Hemophilia A, Prophylaxis of bleeding

Prophylaxis regimen for hemophilia A patients

Trial purpose

Researchers are looking for a better way to treat people who have hemophilia A.
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 become available. These products last longer in the body so that they require to be given less often with injections every 3-5 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 their experience.

The main purpose of this study is to learn how well a new scoring approach works to select a treatment plan for the prevention of bleeding in people with hemophilia A who switch their treatment from SHL products to Jivi. Different types of information are used to calculate the risk score like bleeding history, certain biological factors, and physical activity of the participant.
All participants will receive Jivi for 6 months.
In the first four weeks, all participants will receive Jivi 2 times a week at a dose level of 40 IU per kilogram body weight (also known as 40 IU/kg/dose, recommended maximum dose is 6,000 IU). Then, based on their risk score, each participant will be assigned to one of three treatment plans:
•   participants with a high risk remain on Jivi administration 2 times a week at 40 IU/kg/dose
•   participants with a medium risk will switch to Jivi administration every 5 days at 50 IU/kg/dose
•   participants with a low risk will switch to Jivi administration every 5 days at 50 IU/kg/dose and after 4 weeks to a less frequent administration (e.g., every 7 days) at 60 IU/kg/dose
To check how well the new scoring approach works for choosing the right treatment plan, researchers will look at how many participants have a favourable outcome.
This means that the participant has either fewer bleeding events vs. the pre-study treatment and takes Jivi less often or as often as the previous SHL treatment but with fewer bleeding events, or that the participant has a comparable number of bleeding events but needs to take Jivi less often than the previous treatment. Each participant will be in the study for approximately 7.5 months. During this time, 4 visits to the study site and 3 phone calls are planned. During the study, the doctors and their study team will: • do physical examinations • take blood samples • ask the participants questions about how they are feeling and what adverse events they are having. In addition, participants or their guardians are required to write down the dates of Jivi treatments and bleeding events in an electronic diary and to fill in different questionnaires on their quality of life, health status, work/ school productivity, pain, and treatment satisfaction. In addition, participants are expected to keep appointments for visits and to adhere to the assigned treatment regimen.
An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.

Key Participants Requirements

Sex

All

Age

12 - N/A

Trial summary

Enrollment Goal
21
Trial Dates
July 2022 - October 2024
Phase
Phase 4
Could I Receive a placebo
No
Products
Jivi (Damoctocog, BAY94-9027)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Completed
Aflac Cancer and Blood Disorders Center - Egleston HospitalAtlanta, 30301, United States
Completed
University of Miami HospitalMiami, 33136, United States
Completed
Gulf States Hemophilia & Thrombophilia Center- Texas Medical CenterHouston, 77030, United States
Completed
Luskin Orthopaedic Institute for ChildrenLos Angeles, 90001, United States
Completed
Rush University Medical Center - OncologyChicago, 60612, United States
Not yet recruiting
Michigan State UniversityEast Lansing, 48824, United States
Completed
Wellstar MCG Health Medical Center - GynecologyAugusta, 30912, United States
Completed
University of Colorado | Renal Research OfficeAurora, 80045, United States
Completed
Stead Family Children's Hospital - Hematology / OncologyIowa City, 52240, United States
Completed
Rutgers Robert Wood Johnson Medical School - OBGYNNew Brunswick, 08901, United States
Withdrawn
Johns Hopkins Univ School Med|Sidney Kimmel Comp Cancer CntrBaltimore, 21231, United States
Withdrawn
University of California - DavisSacramento, 95817, United States
Completed
University of Minnesota Medical CenterMinneapolis, 55455, United States
Recruiting
Dr. Akshat Jain - Loma Linda University Medical CenterLoma Linda, 92354, United States
Withdrawn
Loma Linda Children's HospitalSan Bernardino, 92408, United States
Completed
Loma Linda University Children's Hospital - Hematology / OncologyLoma Linda, 92354, United States
Withdrawn
Children's Hospital of OklahomaOklahoma City, 73104, United States

Primary Outcome

  • Occurrence of favorable outcome on the score selected dosing regimen
    To assess the effect of using a baseline risk score, based on a participant’s phenotypic and biologic variables, to select the most appropriate prophylaxis regimen for reaching a favorable outcome, when switching from a SHL product to Jivi.
    date_rangeTime Frame:
    6 months

Secondary Outcome

  • ABR (total, joint, spontaneous)
    To assess the efficacy of Jivi compared to a previous SHL treatment.
    date_rangeTime Frame:
    6 months
  • Change in total ABR from pre-study
    To assess the efficacy of Jivi compared to a previous SHL treatment.
    date_rangeTime Frame:
    6 months
  • Change in the frequency of pre-study SHL treatment to the frequency of Jivi administration (infusions/month)
    To assess the frequency of Jivi administration.
    date_rangeTime Frame:
    6 months
  • Occurrence of participants with 0 and ≤ 1 spontaneous bleeds
    To assess the proportion of participants with 0 and ≤ 1 spontaneous bleeds.
    date_rangeTime Frame:
    6 months
  • Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL)
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
    date_rangeTime Frame:
    6 months
  • Patient’s Global Impression of Change (PGI-C)
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
    date_rangeTime Frame:
    6 months
  • EuroQoL 5 Dimensions (EQ-5D-5L) questionnaire
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
    date_rangeTime Frame:
    6 months
  • Treatment Satisfaction Questionnaire for Medication (TSQM)
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
    date_rangeTime Frame:
    6 months
  • Work Productivity and Activity Impairment (WPAI) questionnaire scores
    To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
    date_rangeTime Frame:
    6 months
  • Number of target joints and change in target joint status from baseline
    To assess target joint status, per International Society on Thrombosis and Haemostasis (ISTH) guidelines
    date_rangeTime Frame:
    6 momth

Trial design

A multicenter, prospective, open-label, clinical study to assess the effect of using a new risk score approach to select the most appropriate prophylaxis regimen for reaching a favorable outcome, when hemophilia A patients switch from standard half-life products to damoctocog alfa pegol (Jivi)
Trial Type
Interventional
Intervention Type
Biological/Vaccine
Trial Purpose
Treatment
Allocation
N/A
Blinding
N/A
Assignment
Single Group Assignment
Trial Arms
1