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Prophylaxis Regimen for Hemophilia A Patients (PREDICT)

Primary Purpose

Hemophilia A, Prophylaxis of Bleeding

Status
Recruiting
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Damoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG).
Sponsored by
Bayer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Participants must be ≥ 12 years of age inclusive, at the time of signing the informed consent/assent.
  • Previously treated patients (≥ 150 EDs) with congenital hemophilia A.
  • Prophylaxis with any SHL FVIII product with a stable dose/regimen for at least 12 months (including at the time of screening) before entering the study and documented in medical records.
  • Documented bleeding rate (ABR) for at least the 6 months prior to study entry.
  • No history or current evidence (≥ 0.6 BU/mL) of FVIII inhibitors.
  • If they are human immunodeficiency virus (HIV) positive, cluster of differentiation 4 (CD4) + lymphocyte count should be > 200/mm^3 within 1 year before entering the study and documented in medical records.
  • Participants who are willing to complete an electronic diary (eDiary).
  • Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
  • For adolescent participants (≥ 12 to < 18 years), a legal guardian must be available to help the study-site personnel ensure follow-up; accompany the participant to the study site on each assessment day according to the SoA (e.g. able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); consistently and consecutively be available to provide information on the participant using the PROs during the scheduled study visits; accurately and reliably dispense study intervention as directed.
  • For adolescent participants, a legal guardian must be able to accurately maintain the child's take-home record, including items of general health.

Exclusion Criteria:

  • Any other inherited or acquired bleeding disorder in addition to hemophilia A.
  • Platelet count < 100,000/mm^3
  • The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug or device within 30 days of signing informed consent.
  • The participant has a planned major surgery.
  • Documentation of missing risk score parameters

    . - Known hypersensitivity to the drug substance, excipients, or mouse or hamster protein.

  • Any other significant medical condition that the investigator feels would be a risk to the participant or would impede the study.
  • Close affiliation with the investigational site; e.g. a close relative of the investigator, dependent person (e.g. employee or student of the investigational site).
  • Otherwise vulnerable participants (e.g. participants who are in custody by order of an authority).
  • Judgment by the investigator that the participant should not participate in the study if the participant is unlikely to comply with study procedures (i.e. eDiary completion), restrictions, and requirements.

Sites / Locations

  • Dr. Akshat Jain - Loma Linda University Medical CenterRecruiting
  • Orthopaedic Institute for ChildrenRecruiting
  • University of California - DavisRecruiting
  • University of Colorado HospitalRecruiting
  • University of Miami HospitalRecruiting
  • Aflac Cancer and Blood Disorders CenterRecruiting
  • Augusta University Medical CenterRecruiting
  • Rush University Medical CenterRecruiting
  • The University of Iowa - Div of Sponsored ProgramsRecruiting
  • Johns Hopkins Univ School Med|Sidney Kimmel Comp Cancer CntrRecruiting
  • Michigan State University
  • University of Minnesota Medical CenterRecruiting
  • Rutgers Robert Wood Johnson Medical SchoolRecruiting
  • Gulf States Hemophilia & Thrombophilia Center-UT PhysiciansRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Damoctocog alfa-pegol prophylaxis regimens

Arm Description

Prophylaxis regimens: All participants will begin with prophylaxis 2x/week (40 IU/kg/dose (recommended maximum dose 6,000 IU)) Participants with a high risk score (> 4) continue on prophylaxis 2x/week (40 IU/kg/dose). Participants with a medium risk score (2 to 4) will switch after 4 weeks to prophylaxis Q5D (50 IU/kg/dose). Participants with a low risk score (< 2) will switch after 4 weeks to prophylaxis Q5D (50 IU/kg/dose) and then after 4 weeks to a less frequent (e.g. Q7D) regimen (60 IU/kg/dose).

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

ABR (total, joint, spontaneous)
To assess the efficacy of Jivi compared to a previous SHL treatment.
Change in total ABR from pre-study
To assess the efficacy of Jivi compared to a previous SHL treatment.
Change in the frequency of pre-study SHL treatment to the frequency of Jivi administration (infusions/month)
To assess the frequency of Jivi administration.
Occurrence of participants with 0 and ≤ 1 spontaneous bleeds
To assess the proportion of participants with 0 and ≤ 1 spontaneous bleeds.
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).
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).
EuroQoL 5 Dimensions (EQ-5D-5L) questionnaire
To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
Treatment Satisfaction Questionnaire for Medication (TSQM)
To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
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).
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

Full Information

First Posted
September 2, 2021
Last Updated
October 20, 2023
Sponsor
Bayer
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1. Study Identification

Unique Protocol Identification Number
NCT05036278
Brief Title
Prophylaxis Regimen for Hemophilia A Patients
Acronym
PREDICT
Official Title
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)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 28, 2022 (Actual)
Primary Completion Date
July 10, 2024 (Anticipated)
Study Completion Date
July 17, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bayer

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A, Prophylaxis of Bleeding

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Damoctocog alfa-pegol prophylaxis regimens
Arm Type
Experimental
Arm Description
Prophylaxis regimens: All participants will begin with prophylaxis 2x/week (40 IU/kg/dose (recommended maximum dose 6,000 IU)) Participants with a high risk score (> 4) continue on prophylaxis 2x/week (40 IU/kg/dose). Participants with a medium risk score (2 to 4) will switch after 4 weeks to prophylaxis Q5D (50 IU/kg/dose). Participants with a low risk score (< 2) will switch after 4 weeks to prophylaxis Q5D (50 IU/kg/dose) and then after 4 weeks to a less frequent (e.g. Q7D) regimen (60 IU/kg/dose).
Intervention Type
Biological
Intervention Name(s)
Damoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG).
Other Intervention Name(s)
Jivi, BAY94-9027
Intervention Description
Dosage Levels: 40 IU/kg/dose two times per week 50 IU/kg/dose every 5 days 60 IU/kg/dose, Less frequent dosing (e.g. every 7 days), the total recommended maximum dose/infusion is 6000 IU.
Primary Outcome Measure Information:
Title
Occurrence of favorable outcome on the score selected dosing regimen
Description
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.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
ABR (total, joint, spontaneous)
Description
To assess the efficacy of Jivi compared to a previous SHL treatment.
Time Frame
6 months
Title
Change in total ABR from pre-study
Description
To assess the efficacy of Jivi compared to a previous SHL treatment.
Time Frame
6 months
Title
Change in the frequency of pre-study SHL treatment to the frequency of Jivi administration (infusions/month)
Description
To assess the frequency of Jivi administration.
Time Frame
6 months
Title
Occurrence of participants with 0 and ≤ 1 spontaneous bleeds
Description
To assess the proportion of participants with 0 and ≤ 1 spontaneous bleeds.
Time Frame
6 months
Title
Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL)
Description
To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
Time Frame
6 months
Title
Patient's Global Impression of Change (PGI-C)
Description
To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
Time Frame
6 months
Title
EuroQoL 5 Dimensions (EQ-5D-5L) questionnaire
Description
To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
Time Frame
6 months
Title
Treatment Satisfaction Questionnaire for Medication (TSQM)
Description
To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
Time Frame
6 months
Title
Work Productivity and Activity Impairment (WPAI) questionnaire scores
Description
To assess participant quality of life (QoL) and physical activity, as measured by Patient Reported Outcomes (PROs).
Time Frame
6 months
Title
Number of target joints and change in target joint status from baseline
Description
To assess target joint status, per International Society on Thrombosis and Haemostasis (ISTH) guidelines
Time Frame
6 momth

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Participants must be ≥ 12 years of age inclusive, at the time of signing the informed consent/assent. Previously treated patients (≥ 150 EDs) with congenital hemophilia A. Prophylaxis with any SHL FVIII product with a stable frequency for at least 6 consecutive months within the last 12 months prior to screening before entering the study and documented in medical records. Stable frequency is defined as a minimum 18 weeks of treatment in a 6 (consecutive) calendar month period in the 12 months prior to screening. Patients can be on any non-Jivi EHL between the 6-month stable SHL prophylaxis period and start of study treatment. Documented bleeding rate (ABR) while on stable frequency SHL prophylaxis for at least 6 consecutive months within the last 12 months prior to screening. No current evidence (≥ 0.6 BU/mL) of FVIII inhibitors. If a participant has had a positive inhibitor titer in the past (≥ 0.6 BU/mL on two occasions) but has been tolerized for at least 1 year since the last positive titer with at least 1 negative inhibitor assay test during that period, they can be enrolled. If a participant has had a positive inhibitor titer in the past (≥ 0.6 BU/mL) but did not require tolerization and has had at least 1 negative inhibitor assay test during a minimum period of at least 1 year since the last positive titer, they can be enrolled. If they are human immunodeficiency virus (HIV) positive, cluster of differentiation 4 (CD4+) lymphocyte count should be > 200/mm^3 within 1 year before entering the study and documented in medical records. - Participants who are willing to complete an electronic diary (eDiary). Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. For adolescent participants (≥ 12 to < 18 years), a legal guardian must be available to help the study-site personnel ensure follow-up; accompany the participant to the study site on each assessment day according to the Schedule of Activities (SoA) (e.g. able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); consistently and consecutively be available to provide information on the participant using the PROs during the scheduled study visits; accurately and reliably dispense study intervention as directed. For adolescent participants, a legal guardian must be able to accurately maintain the child's take-home record, including items of general health. Exclusion Criteria: Any other inherited or acquired bleeding disorder in addition to hemophilia A. Note: von Willebrand disease should be diagnosed per local clinical practice. Participants with a diagnosis of von Willebrand disease in medical records or diagnosed at the time of screening will be excluded. Platelet count < 100,000/mm^3 Evidence of inhibitor to FVIII (≥ 0.6 BU/mL) within the last 1 year The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug or device within 30 days of signing informed consent. The participant has a planned major surgery. Documentation of missing risk score parameters other than physical activity . Known hypersensitivity to the drug substance, excipients, or mouse or hamster protein. Any other significant medical condition that the investigator feels would be a risk to the participant or would impede the study. Close affiliation with the investigational site; e.g. a close relative of the investigator, dependent person (e.g. employee or student of the investigational site). Otherwise vulnerable participants (e.g. participants who are in custody by order of an authority). Judgment by the investigator that the participant should not participate in the study if the participant is unlikely to comply with study procedures (i.e. eDiary completion, clinic visits, phone updates), restrictions, and requirements.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Bayer Clinical Trials Contact
Phone
(+)1-888-84 22937
Email
clinical-trials-contact@bayer.com
Facility Information:
Facility Name
Dr. Akshat Jain - Loma Linda University Medical Center
City
Loma Linda
State/Province
California
ZIP/Postal Code
92354
Country
United States
Individual Site Status
Recruiting
Facility Name
Orthopaedic Institute for Children
City
Los Angeles
State/Province
California
ZIP/Postal Code
90001
Country
United States
Individual Site Status
Recruiting
Facility Name
University of California - Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Colorado Hospital
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Miami Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Individual Site Status
Recruiting
Facility Name
Aflac Cancer and Blood Disorders Center
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30301
Country
United States
Individual Site Status
Recruiting
Facility Name
Augusta University Medical Center
City
Waynesboro
State/Province
Georgia
ZIP/Postal Code
30830
Country
United States
Individual Site Status
Recruiting
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Individual Site Status
Recruiting
Facility Name
The University of Iowa - Div of Sponsored Programs
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52240
Country
United States
Individual Site Status
Recruiting
Facility Name
Johns Hopkins Univ School Med|Sidney Kimmel Comp Cancer Cntr
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21231
Country
United States
Individual Site Status
Recruiting
Facility Name
Michigan State University
City
East Lansing
State/Province
Michigan
ZIP/Postal Code
48824
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
University of Minnesota Medical Center
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Recruiting
Facility Name
Rutgers Robert Wood Johnson Medical School
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Individual Site Status
Recruiting
Facility Name
Gulf States Hemophilia & Thrombophilia Center-UT Physicians
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.
Links:
URL
https://clinicaltrials.bayer.com
Description
Click here to find information for studies related to Bayer products. To find this study enter the ClinicalTrials.gov identifier (NCT) number or Bayer Study Identifier (ID) in the search field.

Learn more about this trial

Prophylaxis Regimen for Hemophilia A Patients

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