ADYNOVI/ADYNOVATE for Hemophilia A

Not currently recruiting at 50 trial locations

Overseen By Shire Contact

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Baxalta now part of Shire

Must be taking: Adynovi/Adynovate

Disqualifiers: Hypersensitivity, Mental incapacity, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Approximately up to 10 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the long-term side effects of ADYNOVI/ADYNOVATE, a clotting factor replacement therapy, for individuals with haemophilia A. Haemophilia A prevents blood from clotting properly, causing excessive bleeding from minor injuries. Participants will use ADYNOVI/ADYNOVATE regularly to prevent bleeding, as advised by their doctors. This trial suits those with haemophilia A who are already using ADYNOVI/ADYNOVATE and have no inhibitors against the treatment. As a Phase 4 trial, the treatment has received FDA approval and proven effective, allowing researchers to understand its benefits for more patients.

Do I have to stop taking my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications. It focuses on participants already prescribed ADYNOVI/ADYNOVATE, so you may continue your current treatment unless advised otherwise by your doctor.

What is the safety track record for ADYNOVI/ADYNOVATE?

Research has shown that ADYNOVI/ADYNOVATE was tested for safety in 365 people with severe hemophilia A, including both children and adults who received at least one dose. The results indicated that most participants handled the treatment well.

Another study found ADYNOVATE to be safe during surgeries in patients with severe hemophilia A, suggesting the treatment's safety in various situations.

Overall, these findings suggest that ADYNOVI/ADYNOVATE is generally safe for people with hemophilia A. However, the current study focuses on checking for any long-term side effects. As with any treatment, participants should discuss any concerns with their doctor. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

ADYNOVI/ADYNOVATE is unique because it is designed to extend the half-life of factor VIII, which is crucial for blood clotting in people with hemophilia A. Unlike traditional factor VIII treatments that require frequent dosing, ADYNOVI/ADYNOVATE offers a longer-lasting effect, potentially reducing the need for frequent infusions. Researchers are excited about this treatment because it can provide more consistent protection against bleeding episodes, enhancing the quality of life for those living with hemophilia A.

What is the effectiveness track record for ADYNOVI/ADYNOVATE in treating haemophilia A?

Research has shown that ADYNOVI/ADYNOVATE reduces bleeding episodes in people with hemophilia A. In clinical studies, individuals using ADYNOVATE regularly to prevent bleeds experienced 2 or fewer bleeding episodes per year, applicable to both children and adults. Participants in this trial will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians according to standard clinical practice. The treatment is safe and effective for maintaining joint health and managing bleeding in everyday life. ^{3 4 5 6 7}

Who Is on the Research Team?

Study Director

Principal Investigator

Takeda

Are You a Good Fit for This Trial?

Inclusion Criteria

Signed informed consent obtained from participant and/or legally authorised representative before any study related activities (any procedure related to recording of data according to the protocol).

Participant at any age with haemophilia A prescribed ADYNOVI/ADYNOVATE prophylaxis.

Negative factor VIII (FVIII) inhibitor test at study entry.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ADYNOVI/ADYNOVATE prophylactically as prescribed by physicians based on standard clinical practice

Approximately up to 10 years

Follow-up

Participants are monitored for long-term side effects and adverse events

Approximately up to 10 years

What Are the Treatments Tested in This Trial?

Interventions

• ADYNOVI/ADYNOVATE

How Is the Trial Designed?

Group I: Haemophilia A Group Experimental Treatment 1 Intervention

Participants with haemophilia A in the study will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians based on their standard clinical practice and in accordance with the national summary of product characteristics (SmPC).

Who Is Running the Clinical Trial?

Baxalta now part of Shire

Lead Sponsor

Trials

111

Recruited

9,100+

Flemming Ornskov

Baxalta now part of Shire

Chief Executive Officer since 2013

MD, MPH

Ludwig N. Hantson

Baxalta now part of Shire

Chief Medical Officer since 2015

PhD

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04158934

NCT04158934 | A Long-term Study of ADYNOVI/ ... The main aim of this study is to check for long-term side effects from ADYNOVI/ADYNOVATE prophylaxis in participants with haemophilia A.

2. adynovate.com adynovate.com/prophylaxis

ADYNOVATE® Prophylaxis Treatment. In these ADYNOVATE clinical studies, patients receiving twice-weekly prophylaxis, ADYNOVATE demonstrated 2 bleeds or less per year in children and adults.†. † ...

3. pei.de pei.de/SharedDocs/Downloads/DE/awb/nis-0101-0200/0120-abschlussb.pdf?__blob=publicationFile&v=1

AHEAD: ADVATE / ADYNOVI Hemophilia A Outcome ... Primary objective was to determine joint health outcome in patients receiving ADVATE or ADYNOVI. The secondary objectives comprised the ...

4. adynovatepro.com adynovatepro.com/prophylaxis

Prophylaxis Data - ADYNOVATE® In clinical trials, ADYNOVATE demonstrated the ability to help prevent bleeding episodes using a prophylaxis regimen.

5. fda.gov fda.gov/media/123626/download

Adynovate 2019 Safety and Utilization Review Efficacy and safety of pegylated full- length recombinant factor VIII with extended half- life for perioperative haemostasis in hemophilia A patients.

6. adynovate.com adynovate.com/safety-information

ADYNOVATE®: Safety Information. The safety of ADYNOVATE was evaluated in 365 patients (children and adults) with severe hemophilia A who received at least 1 dose of ADYNOVATE in 6 completed ...

7. adynovatepro.com adynovatepro.com/safety-profile

Safety Profile - ADYNOVATE® The safety of ADYNOVATE was evaluated in patients (children and adults) (N=365) with severe hemophilia A who received at least 1 dose of ADYNOVATE in 6 ...