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NCT Number: NCT04158934

A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A

The main aim of this study is to check for long-term side effects from ADYNOVI/ADYNOVATE prophylaxis in participants with haemophilia A when used under standard clinical practice in the real-world clinical setting.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Sex eligibility

All sexes

Study type

Observational

Primary location

SHAT of Oncohaematology Diseases, Sofia, Bulgaria

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.
  • 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.

Treatment and study plan

ADYNOVI/ADYNOVATE

Biological

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 outcomes

  1. Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)

    Time frame: Throughout the study period (approximately up to 10 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. Number of Participants With Adverse Events of Special Interest (AESI)

    Time frame: Throughout the study period (approximately up to 10 years)

    Adverse events of special interest are as follows: thromboembolic events, hypersensitivity reactions, lack of efficacy and confirmed FVIII inhibitor development.

  3. Number of Participants With Adverse Events (AE) Related to Impaired Renal Function

    Time frame: Throughout the study period (approximately up to 10 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 long-term effects of PEG accumulation impaired renal function will be evaluated in this outcome.

  4. Number of Participants With Adverse Events (AE) Related to Impaired Hepatic Function

    Time frame: Throughout the study period (approximately up to 10 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 long-term effects of PEG accumulation impaired hepatic function will be evaluated in this outcome.

  5. Number of Participants With Adverse Events (AE) Related to Impaired Neurologic Function

    Time frame: Throughout the study period (approximately up to 10 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 long-term effects of PEG accumulation impaired neurologic function will be evaluated in this outcome.

Secondary outcomes

  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, 9 and 10

    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, 9 and 10

    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, 9 and 10

    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, 9 and 10

    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 10 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 10 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 Neurological Exam

    Time frame: Throughout the study period (approximately up to 10 years)

    Clinically significant abnormal findings in neurological exam collected as part of standard of care (SOC)/ standard clinical practice.

  8. Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters

    Time frame: Throughout the study period (approximately up to 10 years)

    Clinically significant abnormal findings in clinical laboratory parameters collected as part of standard of care (SOC)/ standard clinical practice.

Sponsors and collaborators

Lead sponsor

Baxalta now part of Shire

Industry

Registry information

Official study 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)

Important dates

Study start
2020
Primary completion
2030
Study completion
2030
First posted
Nov 12, 2019
Registry last updated
Feb 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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