Clinical trial NCT04158934: A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A
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)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hemophilia A
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2019-11-12
Enrollment: 207
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Up to 200 participants with haemophilia A will be enrolled. The study will be conducted in European, North American and Asian countries.

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
timeFrame: Throughout the study period (approximately up to 10 years)
measure: Number of Participants With Adverse Events of Special Interest (AESI)
timeFrame: Throughout the study period (approximately up to 10 years)
measure: Number of Participants With Adverse Events (AE) Related to Impaired Renal Function
timeFrame: Throughout the study period (approximately up to 10 years)
measure: Number of Participants With Adverse Events (AE) Related to Impaired Hepatic Function
timeFrame: Throughout the study period (approximately up to 10 years)
measure: Number of Participants With Adverse Events (AE) Related to Impaired Neurologic Function
timeFrame: Throughout the study period (approximately up to 10 years)
Secondary outcomes:
measure: Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Specified Time Points
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
measure: Change From Baseline in Alanine Aminotransferase (ALT) at Specified Time Points
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
measure: Change From Baseline in Bilirubin at Specified Time Points
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
measure: Change From Baseline in Polyethylene Glycol (PEG) Plasma Levels at Specified Time Points
timeFrame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9 and 10
measure: Number of Participants With Clinically Significant Abnormalities in Vital Signs
timeFrame: Throughout the study period (approximately up to 10 years)
measure: Number of Participants With Clinically Significant Abnormalities in Physical Exam
timeFrame: Throughout the study period (approximately up to 10 years)
measure: Number of Participants With Clinically Significant Abnormalities in Neurological Exam
timeFrame: Throughout the study period (approximately up to 10 years)
measure: Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Parameters
timeFrame: Throughout the study period (approximately up to 10 years)
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: UF Health Shands Hospital
state: Florida
city: Sofia
country: Bulgaria
facility: SHAT of Oncohaematology Diseases
city: Zagreb
country: Croatia
facility: Clinical Hospital Sveti Duh
city: Zagreb
country: Croatia
facility: University hospital centre Zagreb
city: Zagreb
country: Croatia
facility: University hospital centre Zagreb
city: Praha 5
country: Czechia
facility: Fakultni nemocnice v Motole
city: Usti nad Labem
country: Czechia
facility: Krajska zdravotni a.s. - Masarykova nemocnice v Usti nad Labem o.z.
city: Heidelberg
country: Germany
facility: SRH Kurpfalzkrankenhaus Heidelberg GmbH
state: Baden Wuerttemberg
city: Berlin
country: Germany
facility: Vivantes Klinikum im Friedrichshain
city: Bonn
country: Germany
facility: Universitaetsklinikum Bonn AoeR
city: Hannover
country: Germany
facility: Werlhof-Institut GmbH
city: Budapest
country: Hungary
facility: Heim Pal Orszagos Gyermekgyogyaszati Intezet
city: Budapest
country: Hungary
facility: Magyar Honvedseg Egeszsegugyi Kozpont
city: Debrecen
country: Hungary
facility: Debreceni Egyetem
city: Mohacs
country: Hungary
facility: Mohacsi Korhaz
city: Nyiregyhaza
country: Hungary
facility: SzSzB Megyei Korhazak es Egyetemi Oktatokorhaz
city: Pecs
country: Hungary
facility: Pecsi Tudomanyegyetem Klinikai Kozpont
city: Milano
country: Italy
facility: Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
city: Roma
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza
city: Torino
country: Italy
facility: Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
city: Seoul
country: Korea, Republic of
facility: Kyung Hee University Hospital at Gangdong
city: Ulsan
country: Korea, Republic of
facility: Ulsan University Hospital
city: Groningen
country: Netherlands
facility: University Medical Centre Groningen-UMCG
city: Palma de Mallorca
country: Spain
facility: Hospital Universitari Son Espases
state: Baleares
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Las Palmas
country: Spain
facility: Hospital Universitario de Gran Canaria Dr. Negrin
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Malaga
country: Spain
facility: Hospital Regional Universitario de Malaga
city: Göteborg
country: Sweden
facility: Sahlgrenska Universitetssjukhuset
city: Taipei
country: Taiwan
facility: MacKay Memorial Hospital_Tamsui Branch
city: Taipei
country: Taiwan
facility: Tri-Service General Hospital
city: Taoyuan
country: Taiwan
facility: Chang Gung Memorial Hospital, Linkou
city: Bangkoknoi Bangkok
country: Thailand
facility: Siriraj Hospital
city: Bangkok
country: Thailand
facility: King Chulalongkorn Memorial Hospital
city: Bangkok
country: Thailand
facility: Phramongkutklao Hospital
city: Chiang Mai
country: Thailand
facility: Maharaj Nakorn Chiang Mai Hospital
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: