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Evaluation of Long-term Safety of ADYNOVI/ADYNOVATE (Antihaemophilic Factor [Recombinant] PEGylated, Rurioctocog Alfa Pegol) in Patients With Haemophilia A - An ADYNOVI/ADYNOVATE Post-Authorisation Safety Study (PASS)

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ClinicalTrials.gov Identifier: NCT04158934
Recruitment Status : Not yet recruiting
First Posted : November 12, 2019
Last Update Posted : June 24, 2020
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Brief Summary:
The main purpose is to evaluate the long-term safety of ADYNOVI/ADYNOVATE prophylaxis in patients with haemophilia A when used under standard clinical practice in the real-world setting.

Condition or disease Intervention/treatment
Hemophilia A Biological: ADYNOVI/ADYNOVATE

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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Evaluation of Long-term Safety of ADYNOVI/ADYNOVATE (Antihaemophilic Factor [Recombinant] PEGylated, Rurioctocog Alfa Pegol) in Patients With Haemophilia A - An ADYNOVI/ADYNOVATE Post-Authorisation Safety Study (PASS)
Estimated Study Start Date : July 20, 2020
Estimated Primary Completion Date : October 18, 2028
Estimated Study Completion Date : October 18, 2028


Group/Cohort Intervention/treatment
Haemophilia A Group
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).
Biological: ADYNOVI/ADYNOVATE
Participants will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians based on their standard clinical practice and in accordance with the national SmPC.
Other Names:
  • Antihaemophilic Factor [Recombinant] PEGylated rurioctocog alfa pegol
  • TAK-660
  • BAX 855




Primary Outcome Measures :
  1. Incidence of Adverse Events (AE) and Serious Adverse Events (SAE) [ Time Frame: Throughout the study period (approximately up to 9 years) ]
    An SAE is any untoward medical occurrence (whether considered to be related to study product or not) that at any dose results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality or birth defect, an important medical event. An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs and SAEs that are at least possibly related to study drug ADYNOVI/ADYNOVATE will be evaluated in this outcome.

  2. Occurrence of Adverse Events (AE) Related to Impaired Renal Function [ Time Frame: Throughout the study period (approximately up to 9 years) ]
    An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs (at least possibly related) that are potentially indicative of or related to impaired renal function will be evaluated in this outcome.

  3. Occurrence of Adverse Events (AE) Related to Impaired Hepatic Function [ Time Frame: Throughout the study period (approximately up to 9 years) ]
    An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs (at least possibly related) that are potentially indicative of or related to impaired hepatic function will be evaluated in this outcome.

  4. Occurrence of Adverse Events (AE) Related to Impaired Neurologic Function [ Time Frame: Throughout the study period (approximately up to 9 years) ]
    An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs (at least possibly related) that are potentially indicative of or related to impaired neurologic function will be evaluated in this outcome.


Secondary Outcome Measures :
  1. Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Specified Time Points [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, and 9, as per SOC ]
    eGFR levels will be assessed from baseline to end of the study at every visit. Note: all assessments are being done as per Standard of Care (SOC) at each study site/ center and are not mandatory.

  2. Change From Baseline in Alanine Aminotransferase (ALT) at Specified Time Points [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, and 9, as per SOC ]
    ALT levels will be assessed from baseline to end of the study at every visit. Note: all assessments are being done as per Standard of Care (SOC) at each study site/ center and are not mandatory.

  3. Change From Baseline in Bilirubin at Specified Time Points [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, and 9, as per SOC ]
    Bilirubin levels will be assessed from baseline to end of the study at every visit. Note: all assessments are being done as per Standard of Care (SOC) at each study site/ center and are not mandatory.

  4. Change From Baseline in Polyethylene Glycol (PEG) Plasma Levels at Specified Time Points [ Time Frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, and 9, as per SOC ]
    PEG plasma levels will be assessed from baseline to end of the study at every visit. Note: all assessments are being done as per Standard of Care (SOC) at each study site/ center and are not mandatory.

  5. Number of Participants With Clinically Significant Abnormalities in Vital Signs [ Time Frame: Throughout the study period (approximately up to 9 years) ]
    Clinically significant abnormal findings in vital signs, collected as part of standard of care (SOC)/ standard clinical practice.

  6. Number of Participants With Clinically Significant Abnormalities in Physical Exam [ Time Frame: Throughout the study period (approximately up to 9 years) ]
    Clinically significant abnormal findings in physical exam collected as part of standard of care (SOC)/ standard clinical practice.

  7. Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters [ Time Frame: Throughout the study period (approximately up to 9 years) ]
    Clinically significant abnormal findings in clinical laboratory parameters collected as part of standard of care (SOC)/ standard clinical practice.

  8. Number of Participants With Clinically Significant Abnormalities in Neurological Exam [ Time Frame: Throughout the study period (approximately up to 9 years) ]
    Clinically significant abnormal findings in neurological exam collected as part of standard of care (SOC)/ standard clinical practice.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Up to 200 patients with haemophilia A will be enrolled. The study is planned to be conducted in Europe and Asia.
Criteria

Inclusion Criteria

  • Participants of any age; except for those in the European Union (EU) must be greater than or equal to (>=) 12 years of age (per the approved EU label).
  • 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 FVIII inhibitor test at study entry.
  • Decision to initiate treatment with commercially available ADYNOVI/ADYNOVATE has been made by the participant and/or legally authorised representative and the treating physician before and independently from the decision to include the participant in this study.

Exclusion Criteria

  • Previous participation in this study. Participation is defined as signed informed consent.
  • Known or suspected hypersensitivity to ADYNOVI/ADYNOVATE or related products.
  • Mental incapacity, unwillingness or other barriers precluding adequate understanding or cooperation.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04158934


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Sponsors and Collaborators
Baxalta now part of Shire
Investigators
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Study Director: Study Director Shire
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Responsible Party: Baxalta now part of Shire
ClinicalTrials.gov Identifier: NCT04158934    
Other Study ID Numbers: TAK-660-403
EUPAS35698 ( Registry Identifier: EUPAS )
First Posted: November 12, 2019    Key Record Dates
Last Update Posted: June 24, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
URL: https://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
BAX 855
Coagulants