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

Hemophilia A Clinical Trial

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04158934
• Other study ID #: TAK-660-403
• Secondary ID: EUPAS35698
• Status: Active, not recruiting
• Start date: July 9, 2020
• Est. completion date: February 28, 2030

Study information

• Verified date: March 2024
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 207
• Est. completion date: February 28, 2030
• Est. primary completion date: February 28, 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• ADYNOVI/ADYNOVATE
Participants will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians based on their standard clinical practice and in accordance with the national SmPC.

Locations

Country	Name	City	State
Bulgaria	SHAT of Oncohaematology Diseases	Sofia
Croatia	Clinical Hospital Sveti Duh	Zagreb
Croatia	University hospital centre Zagreb	Zagreb
Croatia	University hospital centre Zagreb	Zagreb
Czechia	Fakultni nemocnice v Motole	Praha 5
Czechia	Krajska zdravotni a.s. - Masarykova nemocnice v Usti nad Labem o.z.	Usti nad Labem
Germany	Vivantes Klinikum im Friedrichshain	Berlin
Germany	Universitaetsklinikum Bonn AoeR	Bonn
Germany	Werlhof-Institut GmbH	Hannover
Germany	SRH Kurpfalzkrankenhaus Heidelberg GmbH	Heidelberg	Baden Wuerttemberg
Hungary	Heim Pal Orszagos Gyermekgyogyaszati Intezet	Budapest
Hungary	Magyar Honvedseg Egeszsegugyi Kozpont	Budapest
Hungary	Debreceni Egyetem	Debrecen
Hungary	Mohacsi Korhaz	Mohacs
Hungary	SzSzB Megyei Korhazak es Egyetemi Oktatokorhaz	Nyiregyhaza
Hungary	Pecsi Tudomanyegyetem Klinikai Kozpont	Pecs
Italy	Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico	Milano
Italy	Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza	Roma
Italy	Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Roma
Italy	Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino	Torino
Korea, Republic of	Kyung Hee University Hospital at Gangdong	Seoul
Korea, Republic of	Ulsan University Hospital	Ulsan
Netherlands	University Medical Centre Groningen-UMCG	Groningen
Spain	Hospital Universitari Vall d'Hebron	Barcelona
Spain	Hospital Universitario de Gran Canaria Dr. Negrin	Las Palmas
Spain	Hospital Universitario La Paz	Madrid
Spain	Hospital Regional Universitario de Malaga	Malaga
Spain	Hospital Universitari Son Espases	Palma de Mallorca	Baleares
Sweden	Sahlgrenska Universitetssjukhuset	Göteborg
Taiwan	MacKay Memorial Hospital_Tamsui Branch	Taipei
Taiwan	Tri-Service General Hospital	Taipei
Taiwan	Chang Gung Memorial Hospital, Linkou	Taoyuan
Thailand	King Chulalongkorn Memorial Hospital	Bangkok
Thailand	Phramongkutklao Hospital	Bangkok
Thailand	Siriraj Hospital	Bangkoknoi Bangkok
Thailand	Maharaj Nakorn Chiang Mai Hospital	Chiang Mai
United States	UF Health Shands Hospital	Gainesville	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Baxalta now part of Shire

Countries where clinical trial is conducted

United States,  Bulgaria,  Croatia,  Czechia,  Germany,  Hungary,  Italy,  Korea, Republic of,  Netherlands,  Spain,  Sweden,  Taiwan,  Thailand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)

External Links

•   To obtain more information on the study, click here/on this link