A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A
- Conditions
- Hemophilia A
- Interventions
- Biological: ADYNOVI/ADYNOVATE
- Registration Number
- NCT04158934
- Lead Sponsor
- Baxalta now part of Shire
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 207
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Haemophilia A Group ADYNOVI/ADYNOVATE 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).
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE) 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) 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 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 Neurologic Function 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.
Number of Participants With Adverse Events (AE) Related to Impaired Hepatic Function 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.
- Secondary Outcome Measures
Name Time Method 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 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.
Number of Participants With Clinically Significant Abnormalities in Vital Signs 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.
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 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.
Number of Participants With Clinically Significant Abnormalities in Neurological Exam 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.
Change From Baseline in Alanine Aminotransferase (ALT) at Specified Time Points 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.
Change From Baseline in Bilirubin at Specified Time Points 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.
Number of Participants With Clinically Significant Abnormalities in Physical Exam 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.
Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters 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.
Trial Locations
- Locations (36)
UF Health Shands Hospital
🇺🇸Gainesville, Florida, United States
SHAT of Oncohaematology Diseases
🇧🇬Sofia, Bulgaria
Clinical Hospital Sveti Duh
🇭🇷Zagreb, Croatia
University hospital centre Zagreb
🇭🇷Zagreb, Croatia
Fakultni nemocnice v Motole
🇨🇿Praha 5, Czechia
Krajska zdravotni a.s. - Masarykova nemocnice v Usti nad Labem o.z.
🇨🇿Usti nad Labem, Czechia
SRH Kurpfalzkrankenhaus Heidelberg GmbH
🇩🇪Heidelberg, Baden Wuerttemberg, Germany
Vivantes Klinikum im Friedrichshain
🇩🇪Berlin, Germany
Universitaetsklinikum Bonn AoeR
🇩🇪Bonn, Germany
Werlhof-Institut GmbH
🇩🇪Hannover, Germany
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