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| ID | Type | Description | Link |
|---|---|---|---|
| EUPAS35698 | Registry Identifier | EUPAS |
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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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). |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10 |
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Inclusion Criteria
Exclusion Criteria
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Up to 200 participants with haemophilia A will be enrolled. The study will be conducted in European, North American and Asian countries.
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UF Health Shands Hospital | Gainesville | Florida | 32610 | United States | ||
| SHAT of Oncohaematology Diseases |
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| Label | URL |
|---|---|
| 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. | View source |
| 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. | View source |
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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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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.
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| 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) |
| 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. |
| 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. | 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. | 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. | 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. | 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. | 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. | Throughout the study period (approximately up to 10 years) |
| 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 | 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 |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000609799 | BAX 855 |
| D005169 | Factor VIII |
| ID | Term |
|---|---|
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
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