Hemophilia ANCT04158934
A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A
An observational study of Hemophilia A, run by Baxalta now part of Shire at 36 sites in 13 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- What is being tested?
- ADYNOVI/ADYNOVATE
- How many people
- 207 participants
- Where
- 36 sitesin 13 countries
- Study dates
- 9 Jul 2020to 28 Feb 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Up to 200 participants with haemophilia A will be enrolled. The study will be conducted in European, North American and Asian countries.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
36 sites in 13 countries.
36 sites in 13 countries
Hungary6 sites
Heim Pal Orszagos Gyermekgyogyaszati Intezet
Budapest, 1089
Eszak-Pesti Centrumkorhaz - Honvedkorhaz
Budapest, 1134
Debreceni Egyetem
Debrecen, 4032
Mohacsi Korhaz
Mohács, 7700
Szabolcs-Szatmar-Bereg Varmegyei Oktatokorhaz
Nyíregyháza, 4400
Pecsi Tudomanyegyetem Klinikai Kozpont
Pécs, 7624
Spain5 sites
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Hospital Universitario de Gran Canaria Dr. Negrin
Las Palmas, 35010
Hospital Universitario La Paz
Madrid, 28046
Hospital Regional Universitario de Malaga
Málaga, 29010
Hospital Universitari Son Espases
Palma de Mallorca, Balearic Islands, 07120
Germany4 sites
Vivantes Klinikum im Friedrichshain
Berlin, 10249
Universitaetsklinikum Bonn AoeR
Bonn, 53127
Werlhof-Institut GmbH
Hanover, 30159
SRH Kurpfalzkrankenhaus Heidelberg GmbH
Heidelberg, Baden-Wurttemberg, D-69123
Thailand4 sites
King Chulalongkorn Memorial Hospital
Bangkok, 10330
Phramongkutklao Hospital
Bangkok, 10400
Siriraj Hospital
Bangkoknoi Bangkok, 10700
Maharaj Nakorn Chiang Mai Hospital
Chiang Mai, 50200
Croatia3 sites
Clinical Hospital Sveti Duh
Zagreb, 10000
University hospital centre Zagreb
Zagreb, 10000
University hospital centre Zagreb
Zagreb, 1000
Italy3 sites
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, 20122
Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza
Roma, 00161
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 00168
Taiwan3 sites
MacKay Memorial Hospital_Taipei Branch
Taipei, 10449
Tri-Service General Hospital
Taipei, 11490
Chang Gung Memorial Hospital, Linkou
Taoyuan, 333
Czechia2 sites
Fakultni nemocnice v Motole
Prague, 150 06
Krajska zdravotni a.s. - Masarykova nemocnice v Usti nad Labem o.z.
Ústí nad Labem, 40113
South Korea2 sites
Kyung Hee University Hospital at Gangdong
Seoul, 134-727
Ulsan University Hospital
Ulsan, 44033
United States1 site
UF Health Shands Hospital
Gainesville, Florida, 32610
Netherlands1 site
University Medical Centre Groningen-UMCG
Groningen, 9713 GZ
About this study
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)
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.
- Conditions
- Hemophilia A
- Being tested
- ADYNOVI/ADYNOVATE
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ADYNOVI/ADYNOVATEBiological
Also called Antihaemophilic Factor [Recombinant] PEGylated rurioctocog alfa pegol, TAK-660, BAX 855
Participants will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians based on their standard clinical practice and in accordance with the national SmPC.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Biological: ADYNOVI/ADYNOVATE |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
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.
Measured: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
Change From Baseline in Alanine Aminotransferase (ALT) at Specified Time Points
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.
Measured: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
Change From Baseline in Bilirubin at Specified Time Points
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.
Measured: 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
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.
Measured: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
Number of Participants With Clinically Significant Abnormalities in Vital Signs
Clinically significant abnormal findings in vital signs, collected as part of standard of care (SOC)/ standard clinical practice.
Measured: Throughout the study period (approximately up to 10 years)
Number of Participants With Clinically Significant Abnormalities in Physical Exam
Clinically significant abnormal findings in physical exam collected as part of standard of care (SOC)/ standard clinical practice.
Measured: Throughout the study period (approximately up to 10 years)
Number of Participants With Clinically Significant Abnormalities in Neurological Exam
Clinically significant abnormal findings in neurological exam collected as part of standard of care (SOC)/ standard clinical practice.
Measured: Throughout the study period (approximately up to 10 years)
Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters
Clinically significant abnormal findings in clinical laboratory parameters collected as part of standard of care (SOC)/ standard clinical practice.
Measured: Throughout the study period (approximately up to 10 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Nov 2019
- Study start9 Jul 2020
- Primary completion28 Feb 2030 (estimated)
- Study completion28 Feb 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 207 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04158934
- Sponsor's protocol ID
- TAK-660-403
- Other IDs
- EUPAS35698
- Status last verified
- Feb 2026
- First submitted
- 14 Oct 2019
- Last updated
- 18 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04158934
- Acronym / sponsor ID
- TAK-660-403
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 207 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 9 Jul 2020 (actual)
- Primary completion
- 28 Feb 2030 (estimated)
- Study completion
- 28 Feb 2030 (estimated)
- First posted
- 12 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Haemophilia A Group | – | Biological: ADYNOVI/ADYNOVATEParticipants 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 may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Up to 200 participants with haemophilia A will be enrolled. The study will be conducted in European, North American and Asian countries.
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ADYNOVI/ADYNOVATEAlso: Antihaemophilic Factor [Recombinant] PEGylated rurioctocog alfa pegol, TAK-660, BAX 855Participants will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians based on their standard clinical practice and in accordance with the national SmPC. | Biological | Haemophilia A Group |
Primary outcome
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.
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.
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.
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.
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.
8 secondary outcomes
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
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
Change From Baseline in Bilirubin at Specified Time Points
Time frame: 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
Time frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
Number of Participants With Clinically Significant Abnormalities in Vital Signs
Time frame: Throughout the study period (approximately up to 10 years)
Number of Participants With Clinically Significant Abnormalities in Physical Exam
Time frame: Throughout the study period (approximately up to 10 years)
Number of Participants With Clinically Significant Abnormalities in Neurological Exam
Time frame: Throughout the study period (approximately up to 10 years)
Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters
Time frame: Throughout the study period (approximately up to 10 years)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2026.
- Click here for more information about this trial in easy-to-understand language, including a Plain Language Summary of the results if the trial has been completed.Link provided by the sponsor.
- Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
36 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Baxalta now part of Shire at 36 sites in 13 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 18 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 sites in Hungary, Spain, Germany, Thailand, Croatia and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE) (time frame: Throughout the study period (approximately up to 10 years)). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Feb 2026.