A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A
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)
2 other identifiers
observational
207
13 countries
36
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jul 2020
Longer than P75 for all trials
36 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 14, 2019
CompletedFirst Posted
Study publicly available on registry
November 12, 2019
CompletedStudy Start
First participant enrolled
July 9, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 28, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
February 28, 2030
February 18, 2026
February 1, 2026
9.6 years
October 14, 2019
February 13, 2026
Conditions
Outcome Measures
Primary Outcomes (5)
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
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.
Throughout the study period (approximately up to 10 years)
Number of Participants With Adverse Events of Special Interest (AESI)
Adverse events of special interest are as follows: thromboembolic events, hypersensitivity reactions, lack of efficacy and confirmed FVIII inhibitor development.
Throughout the study period (approximately up to 10 years)
Number of Participants With Adverse Events (AE) Related to Impaired Renal Function
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.
Throughout the study period (approximately up to 10 years)
Number of Participants With Adverse Events (AE) Related to Impaired Hepatic Function
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.
Throughout the study period (approximately up to 10 years)
Number of Participants With Adverse Events (AE) Related to Impaired Neurologic Function
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.
Throughout the study period (approximately up to 10 years)
Secondary Outcomes (8)
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Specified Time Points
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
Change From Baseline in Alanine Aminotransferase (ALT) at Specified Time Points
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
Change From Baseline in Bilirubin at Specified Time Points
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
Change From Baseline in Polyethylene Glycol (PEG) Plasma Levels at Specified Time Points
Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
Number of Participants With Clinically Significant Abnormalities in Vital Signs
Throughout the study period (approximately up to 10 years)
- +3 more secondary outcomes
Study Arms (1)
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).
Interventions
Participants will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians based on their standard clinical practice and in accordance with the national SmPC.
Eligibility Criteria
Up to 200 participants with haemophilia A will be enrolled. The study will be conducted in European, North American and Asian countries.
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (36)
UF Health Shands Hospital
Gainesville, Florida, 32610, United States
SHAT of Oncohaematology Diseases
Sofia, 1527, Bulgaria
Clinical Hospital Sveti Duh
Zagreb, 10000, Croatia
University hospital centre Zagreb
Zagreb, 10000, Croatia
University hospital centre Zagreb
Zagreb, 1000, Croatia
Fakultni nemocnice v Motole
Prague, 150 06, Czechia
Krajska zdravotni a.s. - Masarykova nemocnice v Usti nad Labem o.z.
Ústí nad Labem, 40113, Czechia
SRH Kurpfalzkrankenhaus Heidelberg GmbH
Heidelberg, Baden-Wurttemberg, D-69123, Germany
Vivantes Klinikum im Friedrichshain
Berlin, 10249, Germany
Universitaetsklinikum Bonn AoeR
Bonn, 53127, Germany
Werlhof-Institut GmbH
Hanover, 30159, Germany
Heim Pal Orszagos Gyermekgyogyaszati Intezet
Budapest, 1089, Hungary
Eszak-Pesti Centrumkorhaz - Honvedkorhaz
Budapest, 1134, Hungary
Debreceni Egyetem
Debrecen, 4032, Hungary
Mohacsi Korhaz
Mohács, 7700, Hungary
Szabolcs-Szatmar-Bereg Varmegyei Oktatokorhaz
Nyíregyháza, 4400, Hungary
Pecsi Tudomanyegyetem Klinikai Kozpont
Pécs, 7624, Hungary
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza
Roma, 00161, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00168, Italy
University Medical Centre Groningen-UMCG
Groningen, 9713 GZ, Netherlands
Kyung Hee University Hospital at Gangdong
Seoul, 134-727, South Korea
Ulsan University Hospital
Ulsan, 44033, South Korea
Hospital Universitari Son Espases
Palma de Mallorca, Balearic Islands, 07120, Spain
Hospital Universitari Vall d'Hebron
Barcelona, 08035, Spain
Hospital Universitario de Gran Canaria Dr. Negrin
Las Palmas, 35010, Spain
Hospital Universitario La Paz
Madrid, 28046, Spain
Hospital Regional Universitario de Malaga
Málaga, 29010, Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, 41345, Sweden
MacKay Memorial Hospital_Taipei Branch
Taipei, 10449, Taiwan
Tri-Service General Hospital
Taipei, 11490, Taiwan
Chang Gung Memorial Hospital, Linkou
Taoyuan District, 333, Taiwan
King Chulalongkorn Memorial Hospital
Bangkok, 10330, Thailand
Phramongkutklao Hospital
Bangkok, 10400, Thailand
Siriraj Hospital
Bangkoknoi Bangkok, 10700, Thailand
Maharaj Nakorn Chiang Mai Hospital
Chiang Mai, 50200, Thailand
Related Links
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MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 14, 2019
First Posted
November 12, 2019
Study Start
July 9, 2020
Primary Completion (Estimated)
February 28, 2030
Study Completion (Estimated)
February 28, 2030
Last Updated
February 18, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Access Criteria
- IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. 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.
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.